Dynamically adjustable gene- and cell-based therapies are recognized as next-generation medicine. However, the translation of precision therapies into clinics is limited by lack of specific switches controlled by inducers that are safe and ready for clinical use. Ferulic acid (FA) is a phytochemical with a wide range of therapeutic effects, and its salt sodium ferulate (SF) is used as an antithrombotic drug in clinics. Here, we describe an FA/SF-adjustable transcriptional switch controlled by the clinically licensed drug SF. We demonstrated that SF-responsive switches can be engineered to control CRISPR-Cas9 systems for on-command genome/epigenome engineering. In addition, we integrated FA-controlled switches into programmable biocomputers to process logic operations. We further demonstrated the dose-dependent SF-inducible transgene expression in mice by oral administration of SF tablets. Engineered switches responsive to small-molecule clinically licensed drugs to achieve adjustable transgene expression profiles provide new opportunities for dynamic interventions in gene- and cell-based precision medicine.

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A versatile genetic control system in mammalian cells and mice responsive to clinically licensed sodium ferulate - Science Advances

PHILADELPHIA, Aug. 10, 2020 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the appointment of Gallia Levy, M.D., Ph.D., as chief medical officer. Dr. Levy will be responsible for strategic and operational leadership across all functions in the product development lifecycle, including setting the global development strategy for current and future pipeline programs.

We are thrilled to welcome Dr. Gallia Levy to our growing gene therapy company striving to create a world where no life is limited by genetic disease, said Jeffrey D. Marrazzo, chief executive officer, Spark Therapeutics. Dr. Levys passion for hematology and gene therapy research is immediately evident and exactly the perspective needed to achieve our goal of unlocking the full potential of gene therapy. Especially during this pivotal time in hemophilia research, Dr. Levys deep understanding of rare blood disorders and the community will help accelerate our ability to deliver potentially transformative gene therapies for hemophilia, while progressing potential gene therapies for other genetic disease across our pipeline.

Dr. Levy joins Spark Therapeutics from Genentech, also a member of the Roche Group, where she served as the Vice President and Global Head of the Rare Blood Disorders franchise in Product Development. In this role, she was responsible for the clinical development of HEMLIBRA for hemophilia A as well as treatments for other rare blood disorders such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). She played a key role in the evolution of gene therapy as a new modality within the Roche Group.

Ive spent my career working to find new, innovative treatment approaches for patients affected by rare, life-altering disorders, and it is with great pride that I join the Spark team to help advance novel gene therapy programs and create next-generation solutions for patients, said Dr. Levy. Spark Therapeutics shares the same affinity for breaking barriers and putting the patient first, and I look forward to what we will achieve together.

Dr. Levy first joined Genentech in 2009, where she worked in both early and late-stage clinical development. She later moved to Portola Pharmaceuticals, where she led the clinical development program for hematology and oncology indications and returned to Genentech in 2014 to lead the hemophilia program.

Dr. Levy is board-certified in hematology and holds an M.D. and Ph.D. in Molecular and Cellular Biology from the University of Michigan. She completed her residency in internal medicine at Stanford University and a fellowship in hematology and oncology at the University of California, San Francisco. She also holds an M.S. in of Molecular and Cellular Biology from the University of Paris, VI and a B.A. from the University of California, Berkeley.

About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.

Media Contact:Kevin Giordanokevin.giordano@sparktx.com(215) 294-9942

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Spark Therapeutics Deepens Drug Development Expertise in Hematology and Rare Disease with Appointment of Gallia G. Levy, MD, Ph.D., as Chief Medical...

Chemotherapeutic drug resistance is one reason cancer remains an unsolved clinical problem despite the efforts ever since President Nixon declared his "War on Cancer" in 1971. Cancer cells, due in part to the genetic destabilization characteristic of the disease, are capable of expressing genes (normal or aberrant) that permit the cell to avoid the cytotoxic effect of such drugs with the patient providing the situs of selection for and growth of resistant cells. The phenomenon is certain tumor types can have more deleterious consequences than in others, and this is particularly true for glioblastomas (and their non-malignant counterparts, gliomas), cancer of the cells that protect neurons in brain. That organ, confined to the skull, cannot accommodate tumor growth without damaging the brain with which it is confined.

The chemotherapeutic drug of choice for treating glioblastomas is temezolomide (TMZ), an oral alkylating agent that had its chemotherapeutic effect by introducing alkyl groups onto nucleotide bases (preferably at the N-7 and O-6 positions of guanine and N-3 position of adenine) in tumor cell DNA preferentially (due to the greater amount of DNA synthesis occurring in these cells) and disrupting the process leading to cell death (the O-6 methylation having the greatest capacity to induce apoptosis or programmed cell death).O-6-methylguanosnine DNA methyltransferase (MGMT) is the cellular enzyme responsible for repairing alkylated bases in DNA and reduced expression of this gene (e.g., by hypermethylation of the MGMT promoter) is a biomarker for TMZ sensitivity in gliomas and glioblastomas. Recently, a multinational team of researchers* reported genetic rearrangements associated with TMZ resistance, in a paper entitled "MGMT genomic rearrangements contribute to chemotherapy resistance in gliomas" published in Nature Communications. This paper shows a subset of gliomas with rearrangements in the MGMT gene that produce overexpression of the gene and resistance as a result. These authors screened 252 TMZ-treated recurrent gliomas by RNA sequencing and found eight different MGMT genetic fusions (designated BTRC-MGMT,CAPZB-MGMT,GLRX3-MGMT,NFYC-MGMT,RPH3A-MGMT, andSAR1A-MGMTin high-grade gliomas, HGG, andCTBP2-MGMTandFAM175B-MGMT in low-grade gliomas, LGG, in the paper) in seven patients (6 females) with recurrent disease, created by chromosomal rearrangement (see Figure 1c from paper; shown below). These individuals' tumors showed "significantly higher" expression of the rearranged MGMT gene product.

Upon further study, the authors report that five of the eight rearranged genes were located on Chromosome 10 in the vicinity of the MGMT gene itself. The breakpoint in the MGMT was uniformly found at the boundary of exon 2 of the MGMT gene, at a point 12 basepairs upstream of the ATG translation "start" codon. In three of the rearrangements, the breakpoint in the partner gene in the genetic fusion was found in the 5' untranslated region (UTR). All fusions were found to be in-frame (i.e., the reading frame of the MGMT transcript was not disrupted) and the functional regions of the MGMT protein (the methyltransferase domain and DNA-binding domain) were intact. A more fine-structure mapping experiment in the genetic rearrangement resulting in FAM175B-MGMTfound that the fusion was the consequence of a deletion of 4.8 Mb.

The effect of these rearrangements on MGMT expression was elucidated using CRIPSR-Cas9 to produce the BTRC-MGMT, NFYC-MGMT, SAR1A-MGMT, and CTBP2-MGMT rearrangements in cells of two glioblastoma cell lines, U251 and U87. When these cells and their untreated counterparts were challenged by growth in vitro with TMZ, only cells bearing the rearrangements (as confirmed by PCR analysis) were shown to be TMZ resistant. Unlike genetic rearrangements in other cancers that produce fusion proteins (such as the abl-bcr gene produced in chronic myelogenous leukemia bearing the diagnostic Philadelphia chromosome), because most of the rearrangements found involving the MGMT gene were located upstream of the initiation codon of the MGMT gene these authors reasoned that these rearrangements produce increased expression of MGMT leading to TMZ resistance because the cells were better able to repair the methylation injury and replicate functionally. This hypothesis was supported by real-time quantitative PCR analysis of MGMT transcripts in cells bearing the rearrangements, that showed a "striking" increase in expression of MGMT-encoding transcripts (an observation also found in tumors from patients whose gliomas or glioblastomas showed these rearrangements), and Western blot analysis confirmed higher expression levels of the MGMT protein. In two of the rearrangements (BTRC-MGMT and NFYC-MGMT), higher molecular weight fusion proteins were detected as predicted from the genetic data. These results were also replicated in patient tumor-derived stem cells for the BTRC-MGMTandSAR1A-MGMT rearrangements.

These results, and the researchers' conclusion that these rearrangements caused TMZ resistance by overexpression of MGMT, were confirmed by re-establishing TMZ sensitivity in these cells in the presence of O6-benzylguanine (O6-BG), an MGMT inhibitor. These results were further confirmed by detection of double-strand breaks in DNA in these cells in the presence of TMZ and O6-BG.

The relevance of these results to TMZ resistance in vivo was demonstrated using nude mouse xenograft models bearing tumors produced using BTRC-MGMT U251 cells and U251 cells without the rearrangement as control; these cells also contained a recombinant luciferase gene. Mice containing the rearrangement showed no significant prolongation of lifespan in the presence or absence of TMZ, indicating tumor cell resistance, whereas TMZ treatment of nave U251 cells showed improved survival.

While hypomethylation of the native MGMT promoter is the most frequently change associated with TMZ resistance, the results presented in this paper illustrate an alternative mechanism for glioblastomas and gliomas to acquire resistance to TMZ, the only current chemotherapeutic drugs for these maladies. Because these rearrangements were found in patients with recurrent tumors, these authors hypothesize that the rearrangements were selected or by TMZ treatment. A similar rearrangement has also been found in another cancer, medulloblastoma, after TMZ relapse. These authors also suggest that detection of these rearrangements can be used clinically to determine appropriate treatment modalities, particularly for recurrent disease.

* Seve Ballesteros Foundation Brain Tumor Group, Molecular Oncology Programme, Spanish National Cancer Research Center; Division of Life Science, Department of Chemical and Biological Engineering, Center of Systems Biology and Human Health and State Key Laboratory of Molecular Neuroscience, The Hong Kong University of Science and Technology; Beijing Neurosurgical Institute, Capital Medical University; Department of Neurosurgery, Samsung Medical Center, Sungkyunkwan University School of Medicine; Department of Systems Biology, Columbia University; The Jackson Laboratory for Genomic Medicine; and Molecular Cytogenetics Group, Human Cancer Genetics Program, Spanish National Cancer Research Center, CNIO, 28029, Madrid, Spain

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Chromosomal Rearrangements Associated with Chemotherapeutic Drug Resistance | McDonnell Boehnen Hulbert & Berghoff LLP - JD Supra

Jae Jung, Ph.D.

Cleveland Clinic has appointed Jae Jung, Ph.D., chair of Lerner Research Institutes Department of Cancer Biology. Jung will also serve as director of the new Center for Global and Emerging Pathogens Research, which will focus on public health threats ranging from the Zika virus to SARS-CoV-2 (which causes COVID-19).

Jung is an internationally renowned expert in virology and virus-induced cancers who has broken ground in the field of inflammation, immune-oncology and emerging pathogens.

As chair of Cancer Biology, he will lead the departments work in understanding the biological underpinnings of cancer ranging from genetic and molecular pathways to disease manifestation. The department is home to leaders in the field of several cancer research areas including prostate cancer, glioblastoma and stem cells. He will closely collaborate with cancer researchers across Northeast Ohio and Cleveland Clinic, including the new Center for Immunotherapy and Precision Immuno-Oncology.

Jungs cancer research focuses on virus-induced cancers, including Kaposis sarcoma, the most common tumor in patients with AIDS. For his work in this disease area, the National Cancer Institute awarded him the prestigious Outstanding Investigator Award in 2016.

Jung will also lead the Center for Global and Emerging Pathogens Research, which is focused on broadening understanding of emerging pathogens. The center spans Lerner Research Institute and the Cleveland Clinic Florida Research and Innovation Center in Port St. Lucie, Florida.

Jae Jung is a brilliant and global leader in research into the deep and complex intersections between the immune system and cancer, said Serpil Erzurum, M.D., chair of Lerner Research Institute. His work has defined how viruses induce cancers, which make up to 25% of cancers in the world. It is quite fortuitous at this time that we have recruited a world-class scientist in cancer and virus research, propelling our teams in Cleveland and Florida forward in both of these significant areas.

Jung has several research projects related to coronaviruses, including vaccine and drug development and has developed one of the first preclinical models to study SARS-CoV-2 infection and transmission to lead to development of a COVID-19 vaccine. His vaccine work utilizes nanoparticles that compel the coronavirus to use its own surface protein to produce antibodies that block viral infection. The hope is that this approach will have fewer side effects than other vaccines, especially among the older population that is particularly susceptible to COVID-19.

Jung and a multi-disciplinary team of scientists and clinicians in Ohio and Florida are collaborating to uncover the mechanisms of infectious agents and virus-induced cancers. He will lead virology, immunology and oncology researchers working to make laboratory discoveries about how pathogens spread and cause disease and will collaborate with Cleveland Clinics Center for Therapeutics Discovery. He recently received a $2.8 million grant from National Institutes of Health to develop a vaccine for a newly emerging tick-borne disease.

Jae Jung is a foremost authority in virus-related cancer and immunology who will build and grow our research programs to advance science and ultimately improve care for our patients, said Brian Bolwell, M.D., chairman of Taussig Cancer Institute and the Cleveland Clinic Cancer Center. He will bring together a team of experts to better understand the complexities of these cancers and emerging pathogens to develop critically needed treatments and vaccines.

I am excited to collaborate with Cleveland Clinics experts in immunotherapy, oncology and infectious disease to advance our knowledge of immunologic medicine, said Jung. Cleveland Clinics robust clinical and research infrastructure in Cleveland as well as at the new Florida Research and Innovation Center will enable us to develop innovative and novel approaches for new therapeutics and vaccines and make them available to people around the world.

Jung joins Cleveland Clinic from the University of Southern California, where he was chair of Molecular Microbiology and Immunology and director of the Institute of Emerging Pathogens and Immune Diseases. He earned his Ph.D. in microbiology from the University of California, Davis. He completed post-doctoral training and was later promoted to professor at Harvard Medical School. He is an elected fellow of the American Association for the Advancement of Science and the American Academy of Microbiology.

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Jae Jung, Ph.D., Appointed as Chair of Cleveland Clinic Lerner Research Institute's Department of Cancer Biology - Health Essentials from Cleveland...

The UK Government, through the British Embassy Bangkok, and the Thailand Center of Excellence for Life Sciences (TCELS, under the Ministry of Higher Education, Science, Research and Innovation) have today agreed to share knowledge, technology, experience and business information, and to support the research in health and medicines.

TCELS CEO Dr. Nares Damrongchai signed a memorandum of understanding which states that the two organisations will give financial support to the Mahidol-Oxford Research Unit, Faculty of Tropical Medicine, Mahidol University, to conduct 2 research projects: i) the implementation of RT-LAMP technology and genome evolution analysis for 2019-nCov; and ii) the development of a spatiotemporal surveillance platform with interactive user interface for real-time evaluation of the COVID-19 epidemic situation in Thailand.

The expected outputs of the projects are RT-LAMP emergency test kit for COVID-19 which have been tested and ready for mass production, and the surveillance platform for COVID-19 transmission monitoring that fits for the current situation in Thailand. The platform will be used to evaluate the disease control policy in real time, building Thailands preparedness should there be a new wave of transmission. Both projects will be conducted by researchers from the Mahidol-Oxford Research Unit which is a collaboration between the UKs Oxford University and Thailands Mahidol University.

The MOU is the first one between the British Embassy Bangkok and TCELS which will lead to further collaborations on genomic studies. This is a significant step that builds on prior medical research collaborations that the UK and Thailand continue to have for many years with an aim to sustainably better the peoples livelihood and bring prosperity to both countries.

Brian Davidson, British Ambassador to Thailand, said:

The United Kingdom has been supporting middle-income countries through our Prosperity Fund Programmes to help them achieve sustainable and inclusive economic development. The Prosperity Funds Better Health Programme aims to improve the peoples health through partnership and collaborations with our partner countries. We are excited to be working with TCELS as a part of the global effort to fight against the pandemic that has disrupted the whole world. We hope the two research projects will help Thailand in its response to the coronavirus.

Dr. Nares Damrongchai, CEO of TCELS, said:

TCELS has the mission to support and groom Thailands research and innovation that entail health and medical products and services. We aim to build in Thailand the environment, infrastructure and human resources that will enable the international-standard health and medical innovations that are relevant. We also work with our network to ready our business and investment capacities to enter the medical hub industry. One of our approaches is to give financial support to health and medical research projects under TCELS. We would like to thank the UK Government and the British Embassy Bangkok for joining us in supporting important researches that respond to global challenges.

We will continue to work with the UK on the area of health and medical research. The next phase of our collaborations will be about developing the capacities of genetic counselors for medical genomics and precision medicines, for which we hope to be able to announce some good news in the near future.

Sarinplus Leelasaowapak (Jenny)Corporate Communications ManagerMobile : 097 123 9595e-mail: sarinplus@tcels.or.th

Songsang JatupornsathienCommunications ManagerMobile: 083 988 6766 e-mail: Songsang.Jatupornsathien@fco.gov.uk

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The UK and TCELS to jointly support COVID-19 research in Thailand - GOV.UK

Alan Smith, Ph.D., Joins as Chief Technology Officer

Saira Ramasastry Joins Board of Directors; Arthur Tzianabos, Ph.D., Role Expanded to Chairman

BOSTON, Aug. 05, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. (Akouos) (Nasdaq: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced the appointment of Alan Smith, Ph.D., as chief technology officer. In addition, the company announced that Saira Ramasastry has been appointed to its board of directors as audit committee chair, and board member Arthur Tzianabos, Ph.D., has been appointed chairman of the board.

Alan brings terrific experience in the development and manufacturing of complex biologics, and a proven track record of building teams and establishing infrastructure to support in-house GMP manufacturing capabilities, said Manny Simons, Ph.D., founder, president, and chief executive officer of Akouos. Alan joins Akouos at a pivotal time as we prepare to advance our lead program, AK-OTOF, to IND submission next year. We are also excited to welcome Saira, an esteemed life science leader, to our board of directors, and delighted to expand Arthurs role to chairman of our board. Together, these appointments will be instrumental as Akouos continues to grow into a fully integrated genetic medicine company developing innovative potential therapies for a variety of inner ear disorders.

Dr. Smith joins Akouos with more than 30 years of experience in research and development, manufacturing, and quality in the areas of cell and gene therapies. He has contributed to more than 25 FDA regulatory submissions for cell therapy products and devices. Prior to Akouos, Dr. Smith was executive vice president, technical operations at Bellicum Pharmaceuticals, where he led cell product manufacturing, viral vector manufacturing, process development, assay development, GMP supply chain and logistics, worldwide facilities functions, and the design, construction, and startup of multiple GMP manufacturing facilities. Previously, Dr. Smith was vice president of research and development and cellular therapeutics for LifeNet Health and its subsidiary, The Institute of Regenerative Medicine. Earlier in his career, Dr. Smith served as president and chief executive officer for Cognate BioServices Inc. and chief operating officer and senior vice president of research and development for Osiris Therapeutics, Inc. Dr. Smith is also a former adjunct professor at Eastern Virginia Medical School, California State University, Long Beach and Utah State University. He holds a B.S. in chemistry from Southern Utah University and a Ph.D. in biochemistry from Utah State University.

Ms. Ramasastry is managing partner of Life Sciences Advisory, a firm that she founded to provide strategic advice and business development solutions for life science companies. Ms. Ramasastry is also a health innovator fellow of the Aspen Institute and a member of the Aspen Global Leadership Network. Prior to founding Life Sciences Advisory, Ms. Ramasastry was an investment banker with Merrill Lynch & Company, where she helped establish the biotechnology practice and was responsible for origination of mergers and acquisitions, and strategic and capital markets transactions. Prior to joining Merrill Lynch, she served as a financial analyst in mergers and acquisitions group at Wasserstein Perella & Co., an investment banking firm. Ms. Ramasastry currently serves on the board of directors for Vir Biotechnology Inc., Glenmark Pharmaceuticals Ltd., and Sangamo Therapeutics, Inc. She holds a B.A. in economics with honors and distinction and an M.S. in management science and engineering from Stanford University, as well as an M. Phil. in management studies from the University of Cambridge, where she is a guest lecturer for the Bioscience Enterprise Programme and previously served on the Cambridge Judge Business School Advisory Council.

Initially appointed as an independent director to Akouoss board of directors in July 2018, Dr. Tzianabos has now been appointed to serve as chairman. Dr. Tzianabos is currently the chief executive officer and president of Homology Medicines, Inc., leading the efforts to develop genetic medicines for patients with rare genetic diseases. Previously, Dr. Tzianabos spent nine years at Shire Plc, where he worked on the development and launches of multiple treatments for patients with rare genetic disorders. Prior to joining Shire, Dr. Tzianabos was an Associate Professor of Medicine at Harvard Medical School and maintained laboratories at the Channing Laboratory, Brigham and Womens Hospital and the Department of Microbiology and Molecular Genetics at Harvard Medical School. He serves on the board of directors for Stoke Therapeutics, Inc., the Alliance for Regenerative Medicine, and the development board for the University of New Hampshires College of Life Sciences and Agriculture. Dr. Tzianabos holds a B.S. in biology from Boston College and a Ph.D. in Microbiology from the University of New Hampshire.

About AkouosAkouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

Contact

Media:Katie Engleman, 1ABkatie@1abmedia.com

Investors:Courtney Turiano, Stern Investor RelationsCourtney.Turiano@sternir.com

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Akouos Announces Expansion of Executive Team and Board of Directors - BioSpace

Deletions spanning the STS (steroid sulfatase) gene at Xp22.31 are associated with X-linked ichthyosis, corneal opacities, testicular maldescent, cardiac arrhythmia, and higher rates of developmental and mood disorders/traits, possibly related to the smaller volume of some basal ganglia structures. The consequences of duplication of the same genomic region have not been systematically assessed in large or adult samples, although evidence from case reports/series has indicated high rates of developmental phenotypes. We compared multiple measures of physical and mental health, cognition and neuroanatomy in male (n=414) and female (n=938) carriers of 0.8-2.5Mb duplications spanning STS, and non-carrier male (n=192, 826) and female (n=227, 235) controls from the UK Biobank (recruited aged 40-69 from the UK general population). Clinical and self-reported diagnoses indicated a higher prevalence of inguinal hernia and mania/bipolar disorder respectively in male duplication carriers, and a higher prevalence of gastro-oesophageal reflux disease and blistering/desquamating skin disorder respectively in female duplication carriers; duplication carriers also exhibited reductions in several depression-related measures, and greater happiness. Cognitive function and academic achievement did not differ between comparison groups. Neuroanatomical analysis suggested greater lateral ventricle and putamen volume in duplication carriers. In conclusion, Xp22.31 duplications appear largely benign, but could slightly increase the likelihood of specific phenotypes (although results were only nominally-significant). In contrast to deletions, duplications might protect against depressive symptoms, possibly via higher STS expression/activity (resulting in elevated endogenous free steroid levels), and through contributing towards an enlarged putamen volume. These results should enable better genetic counselling of individuals with Xp22.31 microduplications. The Author(s) 2020. Published by Oxford University Press.

PubMed

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Medical and neurobehavioural phenotypes in male and female carriers of Xp22.31 duplications in the UK Biobank. - Physician's Weekly

The revolutionary treatment prevents the coronavirus from entering the bodys virus receptor cells while at the same time allowing immune systems to recognise the virus and learn how to fight it.

In the future it could also be used with other viruses such as seasonal influenza.

If human trials of the new therapy are successful a daily supplement or nasal spray could be ready by early next year and allow pre-pandemic life to resume without social distancing.

This would also remove the need for the conventional vaccines being developed across the globe, including at Oxford University, which experts think are likely to need annual booster injections and may only reduce the severity of Covid-19 in the same way as the existing flu jab.

The new treatment, being developed in the US and funded by British investors, is made from a synthetic protein sequence modelled on the coronavirus which acts like a cork and stops the infection from entering the bodys virus receptor cells - known as the ACE-2 receptors.

It has been assessed by leading experts at the University of California and the results of a promising laboratory study were published in the preprint journal Bio Archive on Friday.

This showed the new therapy, known as Sars-Block, stopped 95 to 100 per cent of the virus from entering coronavirus receptor cells.

Andre Watson, a bioengineer and expert in nano science who invented Sars-Block, said: This is very exciting and could revolutionise the way we treat and prevent infection.

Usually if you get infected with a virus you have to wait it out.

If all goes well this novel form of treatment would protect you from becoming ill by blocking the infection from progressing in your body while also allowing your immune system to recognise and fight it at the same time.

He added: We hope now we can fast track to human studies and have the first doses ready by early 2021.

Mr Watson, founder and chair of leading genetic medicine company Ligandal, has carried out the work with Robert Stroud, professor of biochemistry at the University of California San Francisco and member of the UK Royal Academy, together with experts in immunology, computer science and biophysics.

The work is being funded by a British syndicate of investors led by Adam Hamdy, author and medical strategy consultant.

He said: The work conducted by Andre Watson and the Ligandal team could change the way we fight lots of viral diseases. Meeting the challenge of coronavirus is the first step.

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Coronavirus vaccine breakthrough: New generation cure that stops virus developed - Express

DUBLIN, Aug. 10, 2020 /PRNewswire/ -- The "Cell & Gene Therapy Market - Global Outlook and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included

The study considers the present scenario of the cell and gene therapy market and its market dynamics for the period 2019-2025. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Key Questions Answered

1. What is the cell and gene therapy market size and growth rate during the forecast period?2. What are the factors impacting the growth of the cell and gene therapy market share?3. How is the growth of the healthcare segment affecting the growth of the cell and gene therapy market?4. Who are the leading vendors in the cell and gene therapy market, and what are their market shares?5. Which product type/ end-user type/region is generating the largest revenue in the Asia-Pacific region?

The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.9% during the period 2019-2025

The global cell and gene therapy market is one of the fastest-growing segments in the regenerative medicine market. The market is expected to grow at a faster pace during the forecast period. The demand can be attributed to the growing prevalence of several chronic diseases such as cancer, cartilage related problems, wounds, diabetic foot ulcer, genetic disorders, and other rare diseases across the globe.

The prevalence of cancer and diabetes is increasing in the global population, which is influencing the growth of the market. There is a large unmet need in the treatment available, which is filled by cell and gene therapies. The market is growing due to the increased availability of funding from various public and private institutions. Besides, there is increased support from regulatory bodies for product approval. Several governments are creating awareness of cell and gene therapies in the population.

Cell and Gene Therapy Market Segmentation

The global cell and gene therapy market research report includes a detailed segmentation by product, disease, end-user, and geography.

In 2019, the cell therapy segment accounted for a market share of over 53% in the global cell and gene therapy market. The segment is expected to grow at a steady rate during the forecast period due to the increase in the target population and the rise in the number of countries preferring cell therapies in their patients. Increased therapeutic benefits are attracting several countries to invest in this technology and conduct a high number of clinical trials. However, the lack of advanced infrastructure in developing countries is hindering the growth of the segment.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

Segmentation by Product

Segmentation by Disease

Segmentation by End-user

Insights by Geography

In 2019, North America accounted for a share of over 60% of the global cell and gene therapy market. There are more than 530 regenerative medicine companies, including cell and gene therapy manufacturing developers. The number of products approved in North America grew significantly in 2019, with developers filed for marketing authorization for 10+ regenerative medicines, many of which we expect to be approved in 2020. Within the next 1-2 years, the number of approved gene therapies is expected to double.

The US and Canada are the major contributors to the cell and gene therapy market in North America. Regulatory bodies are supporting several investigational products, fast track approvals, RMAT designation for the faster approval of the product into the market. The alliance for regenerative medicine and Medicare and Medicaid is working together to bring the structured reimbursement channels for cell and gene therapies.

Segmentation by Geography

Insights by Vendors

The global cell and gene therapy market is highly dynamic and characterized by the presence of several global, regional, and local vendors offering a wide range of therapies. Dendreon, Gilead Sciences, Novartis, Organogenesis, Osiris Therapeutics, Vericel, Amgen, and Spark Therapeutics are the leading players in the market with significant shares.

Vendors such as NuVasive, APAC Biotech, Nipro, Orthocell, bluebird bio, J-TEC, and Terumo are the other prominent players in the market with a presence, especially in the cell therapy market. Most leading players are focusing on implementing strategies such as product launches and approvals, marketing and promotional activities, acquisitions, increased R&D investments, and strengthening their distribution networks to enhance their share and presence in the market.

Prominent Vendors

Other Prominent Vendors

Market Dynamics

Opportunities & Trends

Growth Enablers

Growth Restraints

For more information about this report visit https://www.researchandmarkets.com/r/is5t27

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Global Cell and Gene Therapy Market, Forecast to 2025 by Product, Disease, End-user and Region - COVID-19 Updated - PRNewswire

For as long as Seemay Chou can remember, she has gone to bed at midnight and woken around 4:30 a.m. Chou long assumed that meant she was a bad sleeper. Not that she felt bad. In fact, sleeping just four hours a night left her feeling full of energy and with free time to get more done at her job leading a research lab that studies bacteria. It feels really good for me to sleep four hours, she says. When Im in that rhythm, thats when I feel my best.

Still, in an effort to match the slumber schedules of the rest of the world, she would sometimes drug herselfwith melatonin, alcohol or marijuana ediblesinto getting more sleep. It backfired. If I sleep seven or eight hours, I feel way worse, she says. Hung over, almost.

Although the federal government recommends that Americans sleep seven or more hours per night for optimal health and functioning, new research is challenging the assumption that sleep is a one-size-fits-all phenomenon. Scientists have found that our internal body clocks vary so greatly that they could form the next frontiers of personalized medicine. By listening more closely to the ticking of our internal clocks, researchers expect to uncover novel ways to help everybody get more out of their sleeping and waking lives.

Human sleep is largely a mystery. We know its important; getting too little is linked to heightened risk for metabolic disorders, Type 2 diabetes, psychiatric disorders, autoimmune disease, neurodegeneration and many types of cancer. Its probably true that bad sleep leads to increased risks of virtually every disorder, says Dr. Louis Ptacek, a neurology professor at the University of California, San Francisco (UCSF). But details about whats actually going on during shut-eye are sparse. We know almost nothing about sleep and how its regulated, says Ptacek.

Some people are morning larks, rising early, and others are night owls, who like staying up late. Those patterns are regulated by the bodys circadian rhythm, a 24-hour internal clock. People can manipulate their circadian rhythm through all kinds of external factors, like setting an alarm clock or exposing themselves to light. But the ideal sleep duration has long been thought to be universal. There are many people who think everyone needs eight to eight and a half hours of sleep per night and there will be health consequences if they dont get it, says Ptacek. But thats as crazy as saying everybody has to be 5 ft. 10 in. tall. Its just not true.

Ptacek and his wife Ying-Hui Fu, also a professor of neurology at UCSF, are pioneers in the relatively new field of sleep genetics. About a decade ago, Fu discovered the first human gene linked to natural short sleep; people who had a rare genetic mutation seemed to get the same benefits from six hours of sleep a night as those without the mutation got from eight hours. In 2019, Fu and Ptacek discovered two more genes connected to natural short sleep, and theyll soon submit a paper describing a fourth, providing even more evidence that functioning well on less sleep is a genetic trait.

The researchers are now collecting data on short sleepers in order to figure out just how rare these mutations are. If we can get a better understanding of why their sleep is more efficient, we can then come back and help everybody sleep more efficiently, Fu says. Among the participants is Chou, who also happens to work at UCSF. One day at a faculty meeting, she and Ptacek chatted about his work. She immediately recognized herself when he described short sleepers. I had never heard of this. But once I started reading about it, it was sort of an epiphany.

Chou doesnt know yet if she has the identified genetic variants. But after the researchers interviewed her about her familys sleeping patterns, she realized her mom is also a short sleeper. I have memories of when I was younger, and my dad being frustrated with her for staying up really late, but she always seemed fine, she says. The researchers took blood samples from both women.

Doctors once dismissed short sleepers like Chou as depressed or suffering from insomnia. Yet short sleepers may actually have an edge over everyone else. Research is still early, but Fu has found that besides being more efficient at sleep, they tend to be more energetic and optimistic and have a higher tolerance for pain than people who need to spend more time in bed. They also tend to live longer. Chou says the first three hold true for her; by nature, she is sunny and positive, and though she often finds bruises on her body, she usually doesnt remember getting them. I find it annoying how much people complain about little physical pains, she says.

So far, these are just intriguing observations. But by studying genetic short sleepers, Fu and Ptacek believe theyll eventually learn lessons for the rest of us. As we identify more and more genes and we think about the pathways in which they function, at some point, a picture is going to emerge, and we will begin to have an understanding of how sleep is regulated in greater detail, Ptacek says. This, they hope, will lead to targeted treatments, like pills or vitamins, to improve sleep efficiency in everyone.

Researchers are also looking beyond sleep to other circadian bodily processes that might benefit from a personalized or targeted approach. While a master clock in the brain acts like a conductor, setting time for the whole body, the rest of the body is like orchestra players with clocks of their own. All your organs have rhythms, says Steven Lockley, an associate professor of medicine at Harvard Medical School who studies circadian rhythms and sleep. Theres a clock in your heart, a clock in the lungs, a clock in the kidneys. Just about everything in the bodymetabolism, hormones, the immune system, reproductive function and the way DNA is translatedis influenced by a circadian rhythm, he says.

And not everybodys is the same. Peoples internal clocks are often hours off from one another, Lockley says. The range of individual differences is much bigger than anyone really understands yet.

The bodys complex clock system has implications for both healthy people and those with medical conditions, and scientists are already seeing glimpses of how they can time certain tests and treatments to get more accurate or potent results. A cholesterol reading, for example, might be affected by what time of day you go to the doctors office, because the liver (which makes cholesterol) has a circadian rhythm. The time of day at which you measure something could make someone look clinically abnormal, even though theyre not, Lockley says.

Medicine might also be more effective if taken at a certain time. Because theyre metabolized in the liver, drugs change their effects throughout the day, Lockley says. Other circadian bodily processes, like cell function, can also affect how medication acts. Early research suggests certain drugsincluding some for colorectal cancer, pain and asthmaperform better or are less toxic when taken at different times of day.

Exercise, which can be as powerful as medicine for some conditions, is good for you whenever you do it. But I do think that the time of day may have an influence, on top of the effects of exercise, on our metabolic health, says Juleen Zierath, professor of physiology at Karolinska Institute in Sweden. In one small study published in 2018 in the journal Diabetologia, Zierath and her team started 11 men with Type 2 diabetes on a high-intensity interval training program. The men exercised either in the morning (around 8 a.m.) or the afternoon (4 p.m.) for two weeks, then switched schedules. The researchers expected that regardless of the time of day, men in both groups would see improvements in blood-sugar levels. But when they exercised in the morning, they actually had slightly higher levels of blood sugar [than baseline], which we didnt expect at all, Zierath says. Its not clear to what extent the type of exercise and other variables matter, but the study provides an intriguing hint that time of day might make a difference for exercise.

Scientific knowledge is nascent when it comes to optimizing testing and treatment by the clock. Our understanding of individual circadian time is even more primitive. But Lockley believes its the key to personalized medicine; he and others are exploring ways to measure a persons internal circadian time through simple clinical tests. Hopefully in the next five to 10 years, youd go to the doctor, give a breath test or a pee sample, and the doctor would know your biological time, he says. Then all your test results and treatments could be based on your real internal time, which is going to be very different between you and me based on our internal clocks.

For now, even the understanding that our bodies often operate according to different clocks is a big scientific advance. Its already changed the way Chou sleeps, lives and works. Ive just become more comfortable with accepting my sleep, she says. She now asks her employees about their sleep schedules to anticipate when each will be at their peak. She also informs everyone about her own abbreviated schedule, so they know she doesnt expect an immediate response to an email she sends at 4:05 a.m. Thats just when my brain is working, she says.

This appears in the August 17, 2020 issue of TIME.

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Write to Mandy Oaklander at mandy.oaklander@time.com.

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Not Everyone Needs 8 Hours of Sleep, New Research Reveals | Time - TIME

Lindsey A. Criswell, MD, MPH, DSc

Lindsey A. Criswell, MD, MPH, DSc, vice chancellor of research at UC San Francisco, has been selected as the next director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), one of 27 institutes and centers that comprise the National Institutes of Health (NIH). She will assume the role in early 2021.

Having dedicated much of my career to studying and treating autoimmune disorders such as lupus and rheumatoid arthritis, I am tremendously honored to serve as director of NIAMS, said Criswell, professor of medicine in the School of Medicine, professor of orofacial sciences in the School of Dentistry and co-director of the UCSF Clinical & Translational Sciences Institute.

As NIAMS director, Criswell will oversee a federal institution with an annual budget of nearly $625 million and a mission to support scientific research, training and career development in the fields of rheumatology, muscle biology, orthopedics, bone and mineral metabolism, and dermatology, among others. Criswell was selected by NIH Director Francis Collins, MD, PhD, and will succeed Robert H. Carter, MD, who has served as acting NIAMS director since 2018 after long-time director Stephen I. Katz, MD, PhD, passed away unexpectedly in December 2018.

Dr. Criswell has rich experience as a clinician, researcher and administrator. Her ability to oversee the research program of one of the countrys top research-intensive medical schools, and her expertise in autoimmune diseases, including rheumatoid arthritis and lupus, make her well-positioned to direct NIAMS, said Collins. I look forward to having her join the NIH leadership team early next year.

Criswell will join NIAMS after having spent nearly the entirety of her career at UCSF. She arrived at UCSF in 1982 to pursue her medical education, and received her MD from the School of Medicine in 1986. After completing an internship and residency in Southern California, she rejoined UCSF in 1989 to begin a fellowship in rheumatology. She has been at the University ever since. Criswell was made full professor in 2007 and has served as vice chancellor of research since 2017.

Ive had the pleasure of working with Lindsey for nearly four decades. She has distinguished herself as one of UCSFs most accomplished clinician-scientists, as well as a devoted mentor and skillful administrator. Her incredible character and her talent as a leader make her an excellent choice for NIAMS director, said Dan Lowenstein, MD, executive vice chancellor and provost at UCSF.

In her role as vice chancellor of research, Criswell has helped define UCSFs overall research priorities and goals, and has also overseen research infrastructure, with a view to ensuring seamless and superior service to researchers across the University. As a scientist and clinician, Criswell has worked to understand and treat autoimmune disorders like rheumatoid arthritis and lupus, with a particular focus on the genetics, epigenetics and epidemiology of these diseases. She has authored more than 200 peer-reviewed scientific papers, and her efforts have contributed to the identification of more than 30 genes linked to these and other autoimmune disorders. Criswell is also a dedicated educator, who has trained and mentored dozens of students, medical residents, postdoctoral fellows and junior faculty.

Lindseys contributions to science have been tremendous, said Talmadge E. King Jr., MD, dean of the School of Medicine and vice chancellor for medical affairs. Her efforts have led to significant advances in our understanding of the underlying causes of and potential treatments for rheumatoid arthritis, lupus and other debilitating autoimmune disorders. Her influence can also be seen in the many talented young scientists and clinicians whom she has trained, and who are now establishing themselves as leaders in the field, thanks in large part to Lindseys mentorship.

Criswell has received many awards and honors, including the 2014 Resident Clinical and Translational Research Mentor of the Year; a Kirkland Scholar Award from the Mary Kirkland Center for Lupus Research; the Henry Kunkel Young Investigator Award from the American College of Rheumatology; a UCSF Faculty Development Award; and a Pfizer Scholars Award.

Criswell joins the ranks of other distinguished UCSF faculty who have served in leadership roles with the NIH. Eliseo Perez-Stable, MD, was a professor of medicine, chief of the Division of General Internal Medicine, and director of the Center for Aging in Diverse Communities (CADC) before assuming his current role as director of the National Institute on Minority Health and Health Disparities (NIMHD). Zach Hall, PhD, joined UCSF in 1976 and remained on the faculty until appointed director of the National Institute of Neurological Disorders and Stroke (NINDS) in 1994. Nobel laureate Harold Varmus, MD, was a UCSF faculty member for two decades before being appointed NIH director in 1993, and later serving as director of the National Cancer Institute (NCI).

Criswell earned her bachelors degree in genetics and her masters degree in public health from UC Berkeley. She later received a DSc in genetic epidemiology from the Netherlands Institute of Environmental Health Sciences in Rotterdam. Criswell is board certified in internal medicine and as a wilderness medicine first responder. Upon leaving UCSF, Criswell will be granted emeritus status.

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Lindsey Criswell Named Director of National Institute of Arthritis and Musculoskeletal and Skin Diseases - UCSF News Services

Newswise DALLAS Aug. 7, 2020 UT Southwestern scientists have developed a genetically engineered mouse and imaging system that lets them visualize fluctuations in the circadian clocks of cell types in mice. The method, described online in the journal Neuron, gives new insight into which brain cells are important in maintaining the bodys master circadian clock. But they say the approach will also be broadly useful for answering questions about the daily rhythms of cells throughout the body.

This is a really important technical resource for advancing the study of circadian rhythms, says study leader Joseph Takahashi, Ph.D., chair of the department of neuroscience at UT Southwestern Medical Center, a member of UT Southwesterns Peter ODonnell Jr. Brain Institute, and an investigator with the Howard Hughes Medical Institute (HHMI). You can use these mice for many different applications.

Nearly every cell in humans and mice has an internal circadian clock that fluctuates on a roughly 24-hour cycle. These cells help dictate not only hunger and sleep cycles, but biological functions such as immunity and metabolism. Defects in the circadian clock have been linked to diseases including cancer, diabetes, and Alzheimers, as well as sleep disorders. Scientists have long known that a small part of the brain called the suprachiasmatic nucleus (SCN) integrates information from the eyes about environmental light and dark cycles with the bodys master clock. In turn, the SCN helps keep the rest of the cells in the body in sync with each other.

What makes the SCN a very special kind of clock is that its both robust and flexible, says Takahashi. Its a very strong pacemaker that doesnt lose track of time, but at the same time can shift to adapt to seasons, changing day lengths, or travel between time zones.

To study the circadian clock in both the SCN and the rest of the body, Takahashis research group previously developed a mouse that had a bioluminescent version of PER2 one of the key circadian proteins whose levels fluctuate over the course of a day. By watching the bioluminescence levels wax and wane, the researchers could see how PER2 cycled throughout the animals bodies during the day. But the protein is present in nearly every part of the body, sometimes making it difficult to distinguish the difference in circadian cycles between different cell types mixed together in the same tissue.

If you observe a brain slice, for instance, almost every single cell has a PER2 signal, so you cant really distinguish where any particular PER2 signal is coming from, says Takahashi.

In the new work, the scientists overcame this problem by turning to a new bioluminescence system that changed color from red to green only in cells that expressed a particular gene known as Cre. Then, the researchers could engineer mice so that Cre, which is not naturally found in mouse cells, was only present in one cell type at a time.

To test the utility of the approach, Takahashi and his colleagues studied two types of cells that make up the brains SCN arginine vasopressin (AVP) and vasoactive intestinal polypeptide (VIP) cells. In the past, scientists have hypothesized that VIP neurons hold the key to keeping the rest of the SCN synchronized.

When the research team looked at VIP neurons expressing Cre in just those cells, so that PER2 luminesced green in VIP cells, while red elsewhere they found that removing circadian genes from the neurons had little overall effect on the circadian rhythms of the VIP neurons, or the rest of the SCN. Even when VIP neurons no longer had a functioning clock, the rest of the SCN behaved essentially the same, explains Yongli Shan, Ph.D., a UTSW research scientist and lead author of the study. Nearby cells were able to signal to the VIP neurons to keep them in sync with the rest of the SCN, he says.

When they repeated the same experiment on AVP neurons, however removing key clock genes not only did AVP neurons themselves show disrupted rhythms, but the entire SCN stopped synchronously cycling on its usual 24-hour rhythm.

What this showed us was that the clock in AVP neurons is really essential for the synchrony of the whole SCN network, says Shan. Thats a surprising result and somewhat counterintuitive, so we hope it leads to more work on AVP neurons going forward.

Takahashi says other researchers who study circadian rhythms have already requested the mouse line from his lab to study the daily cycles of other cells. The mice might allow scientists to hone in on the differences in circadian rhythms between cell types within a single organ, or how tumor cells cycle differently than healthy cells, he says.

In all sorts of complex or diseased tissues, this can let you see which cells have rhythms and how they might be similar or different from the rhythms of other cell types.

Takahashi holds the Loyd B. Sands Distinguished Chair in Neuroscience.

Other UTSW researchers who contributed to this study were Yan Li, Mariko Izumo, Kimberly Cox, Byeongha Jeong, and Seung-Hee Yoo, who is now at UT Health Science Center in Houston. Collaborators were John Abel and Francis Doyle of Harvard Medical School and David Olson of the University of Michigan.

This research was supported by funds from the HHMI, the National Institutes of Health (R01 NS106657, R01 GM114424, T32-HLO9701, F32-AG064886), and The Welch Foundation (AU-1971-20180324).

About UTSouthwestern Medical Center

UT Southwestern, one of the premier academic medical centers in the nation, integrates pioneering biomedical research with exceptional clinical care and education. The institutions faculty has received six Nobel Prizes, and includes 24 members of the National Academy of Sciences, 16 members of the National Academy of Medicine, and 13 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,500 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UT Southwestern physicians provide care in about 80 specialties to more than 105,000 hospitalized patients, nearly 370,000 emergency room cases, and oversee approximately 3 million outpatient visits a year.

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Pinpointing The Cells That Keep The Bodys Master Circadian Clock Ticking - Newswise

The US FDA has awarded RPDD to paxalisib for the treatment of Diffuse Intrinsic Pontine Glioma, a rare and highly aggressive childhood brain cancer.

() () has surged on being granted Rare Pediatric Disease Designation (RPDD) by the US FDA for its paxalisib for the treatment of DIPG, a rare and highly aggressive childhood brain cancer.

With RPDD granted, Kazia may now be eligible to receive a rare pediatric disease priority review voucher (PRV) if paxalisib is approved for Diffuse Intrinsic Pontine Glioma (DIPG).

A PRV grants the holder an expedited six-month review of a new drug application by FDA.

PRVs can be sold to other companies and have historically commanded prices between US$68 million and US$350 million.

Shares have been more than 78% higher in early trade to a new 3.5 year high of A$1.00.

Kazia CEO Dr James Garner said: Although glioblastoma remains our primary focus for paxalisib, we have been devoting increasing energy to developing the drug in childhood brain cancer as well.

"For patients diagnosed with DIPG, there are currently no FDA-approved drug treatments, and the average survival from diagnosis is around 9.5 months.

Garner said: "The granting of RPDD by the FDA recognises our efforts and achievements so far and leaves us well placed to move paxalisib forward as a potential therapy for DIPG.

"We continue to be inspired by the dedication of our collaborators in this field and are committed to understanding whether paxalisib may be able to help in this enormously challenging paediatric disease.

RPDD has been awarded following positive emerging preclinical data in DIPG and with initial clinical efficacy data expected in the current half-year, positive clinical data may substantially enhance the likelihood of a potential future PRV.

The US Food and Drug Administrations (FDA) RPDD program is intended to advance the development of drugs and biologics for certain serious and life-threatening rare pediatric diseases by providing incentives to industry.

Most significant among these incentives is the potential access of a priority review voucher at the time of a marketing authorisation for the rare pediatric disease.

RPDD may be granted to drugs in development for diseases which primarily affect children under the age of 18, have an incidence of less than 200,000 new cases per annum in the US and which are serious or life-threatening.

In October 2018, St Jude Childrens Research Hospital in Memphis, Tennessee, began a phase I clinical trial of paxalisib in DIPG (NCT03696355).

This study reported favourable top-line safety data in September 2019 and established 27 mg/m2 as the maximum tolerated dose for paediatric use.

The study has completed recruitment and initial efficacy data is anticipated during the second half of 2020.

This data will be used to guide future development of paxalisib (formerly GDC-0084) in this disease.

Dr Matt Dun and colleagues at the University of Newcastle, Australia have conducted extensive laboratory research with paxalisib, focused on phosphoproteomic analysis of its activity in DIPG cell lines.

Phosphoproteomics is a new approach in cancer research that attempts to discern how complex signalling pathways are modified in tumours.

Work at the Dun laboratory has shown paxalisib to be broadly active in DIPG and has identified a number of potential combination strategies which may enhance its activity.

Initial data was presented at the Society for Neuro-Oncology (SNO) Pediatric Neuro-Oncology Basic and Translational Research Conference in San Francisco, CA, in May 2019.

Further ongoing work in animal models is expected to provide additional insight.

In parallel, related laboratory research is underway in the DMG Research Center at the University of Zurich, Switzerland, under the leadership of Dr Javad Nazarian.

Dr Nazarian is also the principal investigator at Center for Genetic Medicine within the Childrens National Medical Center, Washington DC with a focus on DIPG.

Laboratory research is also being conducted at St Jude Childrens Research Hospital by Dr Chris Tinkle and Dr Suzanne Baker and colleagues, in parallel to the ongoing phase I clinical trial at that centre.

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Kazia Therapeutics hits A$1 on grant of Rare Pediatric Disease Designation to paxalisib for DIPG - Proactive Investors USA & Canada

(UroToday.com) In conjunction with the Scientific Congress held as part of the American Society of Clinical Oncologys (ASCO) Annual Meeting in May 2020, an Educational Symposium was convened on August 8 to 10th. In a session entitled Sorting Through the Maze of Treatment Options for Metastatic Castration-Sensitive Prostate Cancer, Dr. Alicia Morgans from Northwestern University presented a plenary talk discussing the treatment considerations for patients who received combination therapy for metastatic castration sensitive prostate cancer (mCSPC). This followed prior presentations from Dr. Carmel Pezaro and Dr. Neal Shore regarding the role of chemotherapy and novel androgen axis inhibitors in patients with mCSPC.

Dr. Morgans began by highlighting that, as per National Comprehensive Cancer Network (NCCN) guidelines, combination therapy with androgen deprivation therapy and another agent (apalutamide, abiraterone, docetaxel, or enzalutamide) is recommended.

While there are a number of treatment options for patients with mCSPC, there is an even greater plethora of treatment options for patients with metastatic castration-resistant prostate cancer (mCRPC), with no less than seven different agents available on the basis of prior therapies received, patient characteristics, and tumor characteristics.

Clearly, prior lines of therapy (including those used in the mCSPC space) influence future treatment choices when the patient progresses to mCRPC. In the mCRPC disease space, treatment choices may be guided by the following factors, among others:

1. prior treatment received, with a preference for novel mechanisms of action2. treatment availability in practice setting/location3. presence of visceral metastasis or restriction to bone-only metastasis4. candidacy for chemotherapy, on the basis of patient comorbidity and frailty5. histologic characteristics, including the presence of small cell/neuroendocrine differentiation6. targetable genetic or genomic characteristics including actionable DNA damage repair mutations or presence of microsatellite instability high status7. availability of clinical trials

Dr. Morgans highlighted that resistance mechanisms can spread through metastasis-to-metastasis seeding and similar resistance patterns are often seen in geographic proximity suggesting an element of interclonal cooperativity. As a result, at the time of disease progression, the use of therapy with a novel treatment mechanism is necessary to counter to acquired resistance.

As a general principle, Dr. Morgans outlined that sequencing of androgen axis inhibitors (including abiraterone acetate, enzalutamide, apalutamide, and darolutamide) is generally poorly effective. To drive home this point, Dr. Morgans reviewed data from the CARD trial which randomized patients who had progression on one androgen axis targeting agent to a switch or to cabazitaxel. Only 14% of patients who switched to the other androgen axis targeting agent had a PSA response of 50% or more and median progression-free survival was only 2.7 months (95% confidence interval 2.4 to 2.8 months) in contrast to median progression-free survival of 4.4 months (95% confidence interval 3.6 to 5.4 months) among those randomized to cabazitaxel (hazard ratio 0.52, 95% confidence interval 0.40 to 0.68). Similarly, in PROfound, patients who had disease progression on a novel androgen axis inhibitor were randomized to physicians choice of therapy (often including a switch to another androgen axis inhibitor) or Olaparib. As with the CARD trial, patients randomized to a second androgen axis inhibitor had median progression-free survival of 3.55 months (hazard ratio 0.34, 95% confidence interval 0.25 to 0.47).

Apart from these randomized data, there are a number of other studies that have assessed response rates for sequencing of androgen axis targeting agents demonstrates generally short progression-free survival.

Dr. Morgans then highlighted data that may guide specific treatment considerations in particular populations. First, she reviewed data from the PROCEED observational registry of patients treated with sipuleucel-T. In a prostate-specific antigen (PSA) matched analysis, median overall survival was significantly longer for African American men (35.3 months) as compared to Caucasian men (25.8 months) (hazard ratio 0.70, 95% confidence interval 0.57 to 0.86). Further, baseline PSA at the time of initiating sipuleucel-T therapy was associated with survival patients with PSA below the median (29.5 ng/mL) had improved survival (hazard ratio 0.52, 95% confidence interval 0.37 to 0.72).

In an international early access program, observational data suggested that asymptomatic patients receiving radium-223 had significantly improved overall survival compared to those who were symptomatic at the initiation of therapy (hazard ratio 0.49, 95% confidence interval 0.33 to 0.73).

Beyond clinicodemographic factors, Dr. Morgans discussed genetic and genomically targeted approaches. She first examined data from TRITON2 in which patients with mCRPC and deleterious mutations in DNA defect repair (DDR) genes who had progressed on androgen axis targeting agents received rucaparib in a single-arm design. The vast preponderance of patients had a beneficial PSA response. Subsequently, the PROfound study reported a randomized comparison of Olaparib versus physicians choice among a similar patient population. They demonstrated significantly improved radiographic progression-free survival among those receiving olaparib (median 5.8 months) versus physicians choice of therapy (median 3.5 months; hazard ratio 0.49, 95% confidence interval 0.38 to 0.63). Great detail regarding the data for PARP inhibitors in prostate cancer is provided in a UroToday Center of Excellence article.

On the basis of these data, rucaparib has been approved for men with BRCA1/2 mutations who have progressed following androgen receptor-targeted therapy and taxane chemotherapy while olaparib has been approved for men with Homologous Recombination Repair (HRR) mutations following androgen receptor-targeted therapy, with or without prior taxane treatment.

Beyondpoly ADP ribose polymerase (PARP) inhibitors, pembrolizumab is another targeted option for men with advanced prostate cancer. In this case, patients must have microsatellite instability-high status to be eligible, as the United States Federal Drug Administration (FDA) has approved pembrolizumab in a tissue agnostic fashion for patients with microsatellite instability-high tumors. Dr. Morgans reported that approximately 2-3% of men with prostate cancer will have microsatellite instability-high tumors, and are therefore expected to have radiographic responses to pembrolizumab.

Dr. Morgans closed by highlighting the importance of shared decision making for patients with advanced prostate cancer. This is particularly important where there are so many treatment options. Patient preferences may be driven by home or work obligations, by past or vicarious experiences or fears, by insurance restrictions, and other factors.

Moving forward, Dr. Morgans highlighted the potential for theranostics, including the therapeutic role of Lutetium-177-prostate-specific membrane antigen (LuPSMA). A number of ongoing trials will assess the role of Lu-PSMA, including in a comparison against cabazitaxel in patients who have progressed following docetaxel and in comparison against best supportive or standard of care in patients who have progressed following both taxane chemotherapy and an androgen axis inhibitor.

Presented by: Alicia Morgans, MD, MPH, Associate Professor of Medicine in the Division of Hematology/Oncology at the Northwestern University Feinberg School of Medicine in Chicago, Illinois

Written by:Christopher J.D. Wallis, Urologic Oncology Fellow, Vanderbilt University Medical Center,@WallisCJD on Twitteratthe ASCO20 Virtual Education Program, #ASCO20, August 8-10, 2020.

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Treatment Considerations for Prostate Cancer Progressing After Combination Therapy for mCSPC - UroToday

MENLO PARK Aug 8, 2020 (Thomson StreetEvents) -- Edited Transcript of Personalis Inc earnings conference call or presentation Thursday, August 6, 2020 at 9:00:00pm GMT

* Aaron L. Tachibana

Personalis, Inc. - CFO

Personalis, Inc. - Co-Founder, CEO, President & Director

Oppenheimer & Co. Inc., Research Division - MD & Senior Analyst

* Subhalaxmi T. Nambi

H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst

* Caroline V. Corner

Good afternoon. My name is Jerome, and I'll be your conference operator today. At this time, I would like to welcome everyone to the Personalis Q2 2020 Earnings Conference Call. (Operator Instructions)

Thank you. I would now like to turn the call over to Ms. Caroline Corner. Please go ahead.

Caroline V. Corner, Westwicke Partners, LLC - MD [2]

Thank you, operator. Welcome to Personalis' Second Quarter 2020 Earnings Call. Joining me on today's call are John West, President and Chief Executive Officer; and Aaron Tachibana, Chief Financial Officer.

This call will include forward-looking statements, including statements regarding the markets in which we operate, including potential market sizes; trends and expectations for products, services and technology; trends and demand for our products; Personalis' expected financial performance, expenses and position in the market; and the impact of the COVID-19 pandemic on our operations and our customers' operations. These statements are subject to risks and uncertainties that could cause actual results to differ materially from our current expectations. We encourage you to review our most recent filings with the SEC, particularly the risk factors described in our 10-K filing for fiscal year 2019 and in our 10-Q filing for our second quarter ended June 30, 2020.

The forward-looking statements we provide during this call, including expectations for future performance, are based on our reasonable beliefs and expectations as of today. Personalis undertakes no obligation to update these statements, except as required by applicable law.

Our press release with our second quarter 2020 results is available on our website, http://www.personalis.com under the Investors section and includes additional details about our financial results. Our website also has our latest SEC filings, which we encourage you to review. A recording of today's call will be available on our website by 5:00 p.m. Pacific Time today.

Now I'd like to turn the call over to John for his comments on second quarter 2020 business highlights.

John Stephen West, Personalis, Inc. - Co-Founder, CEO, President & Director [3]

Thank you, Caroline. Personalis has been responding to COVID-19 in -- began responding to COVID-19 in Q1 before government's shelter orders. That early response helped us have a record quarter in Q1. Our continued response and execution has now led us to another record in Q2.

In April, we coordinated with the VA MVP program to take the largest shipment of samples from them that we have ever had just before their lab in Massachusetts was closed in response to the pandemic. The automation that we've put in place over the last 3 years then allowed us to efficiently maintain our whole genome sequencing work, even when we had a reduced workforce in our lab as a result of operational changes we implemented to comply with state and local orders related to COVID-19 and to protect the health and safety of our employees. As a result, in Q2, we were again able to sequence about 14,000 whole human genomes, up 70% from Q2 last year and matching the Personalis population sequencing revenue record we had set in Q1.

On the oncology side of our business, we also had to adapt rapidly. Some prospective clinical trials that our biotech customers are involved with were either slowed or stoped due to the pandemic. And also revenue from our leading biobank customer fell 98% from Q1. We were able to overcome these challenges by beginning to ramp revenue from our next platform. This allowed us to achieve a sequential revenue increase for biopharma and all other customers. Most of our biopharma business is for retrospective projects, which have been resilient during this pandemic. Over the last 4 quarters, biopharma customer orders have significantly exceeded revenue, driven by adoption of our NeXT platform and predominantly by large pharmaceutical customers. As we saw in Q2, these orders are now beginning to convert to revenue.

Another challenge we faced during the quarter was the timing of biopharma sample receipts. Many were delivered to us later in the quarter than expected so we were not able to process all of them for revenue. This does, however, put us in a good position heading into Q3.

Our laboratory operations team executed well during Q2 and carefully arranged the sample processing schedules to focus on VA MVP samples early within the quarter when labor capacity was lower due to shelter-in-place guidelines. That allowed us to utilize more capacity later in the quarter for biopharma sample processing. As a result, our revenue from pharmaceutical customers increased by more than 20% sequentially. This more than offset the dip in revenue from biobank and biotech customers and helped us achieve quarter-on-quarter growth in our oncology-focused business.

Driven by this growth of NeXT, our overall revenue increased for a 16th consecutive quarter to $19.5 million, up 23% from Q2 last year. All of this was accomplished, while more than 80% of our employees were working from home.

Shifting focus to our progress this quarter, there are several factors which give us confidence in the long-term growth of our business. In the month of June, we were able to bring our liquid biopsy development team members back into the lab. You may have seen our recent press release launching NeXT Liquid Biopsy, which is now available for customer orders. Both our tissue and liquid biopsy-based products have been designed specifically to meet the needs of our pharmaceutical customers and provide data on all 20,000 human genes. With the addition of our liquid biopsy product, we expect to enable comprehensive monitoring of patients across 20,000 genes at multiple time points.

Many new cancer drugs extend the lives of patients but do not eradicate the disease. So a growing segment of the cancer survivor population consists of patients who are still undergoing active disease management. Our first liquid biopsy product tracks the evolution of what can be hundreds or even thousands of cancer mutations in a single tumor. Importantly, we can also detect new mutations as they emerge under therapeutic pressure. Note that some cancer patients develop a second genetically independent cancer while being treated for the first. Our liquid biopsy product's ability to see new mutations may also provide early detection of some so-called second cancers.

I'm extremely proud of our team. We pulled together during this challenging time to get this exciting product into the marketplace. We expect to receive first orders in the coming months. And while we expect that liquid biopsy revenues will be modest initially, we think this new product puts us in an even stronger competitive position as we go forward.

Our liquid biopsy product is designed to be used together with our tissue biopsy product, not instead of it. We expect initial sales to be to customers using our tissue-based testing, who are also seeking liquid biopsy capabilities to monitor the same patients over time. We believe that our capabilities to offer both tissue and liquid biopsy-based products and to leverage the synergy between them, positions us favorably relative to companies who only offer one or the other.

In the future, we will be broadening our liquid biopsy product line to include personalized tests designed for a specific patient's tumors. The team is hard at work here and barring any COVID-19-related interruptions or other unexpected delays, we expect to launch this additional offering in 2021.

We have continued to gain traction with biopharma customers, realizing strong order levels once again in Q2. To frame the pace of customer orders in a different way, in the first half of 2020, we received about the same dollar value of biopharma orders as we received for the full year of 2019. As we've explained before, it takes time for orders to convert to revenue and the actual revenue recognized from an order may be less than expected due to failure of individual customer samples to meet our sample quality requirements and other factors. But this growth in orders gives us confidence in our future revenue stream.

We are winning business based on the value proposition of our NeXT platform. Our NeXT platform can identify biomarkers, including neoantigens across all types of cancer, providing comprehensive data across all 20,000 human genes, all from a small tissue sample.

Our customer base has also grown substantially over the last year. While we can't disclose all of our customer names, I can tell you that we have now received orders from a majority of the top 10 oncology-focused pharma companies. As of the end of Q2, we had 32 different customers who have placed orders for NeXT, up from 26 at the end of Q1. In addition, you may recall that new customers typically evaluate or run a small pilot before growing into larger contracts. Up until the last few quarters, a pilot order could be around $50,000. But with our recent progress, we are now receiving initial orders that can be several hundred thousands of dollars. And in a few cases, approaching $1 million. We believe that this growing adoption of NeXT further highlights the power of our platform and that the comprehensiveness of our platform is important to our customers.

In June, we announced a research collaboration with Sarepta Therapeutics, a leader in precision genetic medicine for rare disease. As part of the collaboration, Sarepta is working with us to characterize immune response precision genetic therapeutics, utilizing our advanced proprietary neoantigen analytics. We are excited that the value of our platform is now being recognized in areas beyond cancer.

We also announced in June that we are establishing a lab and commercial operations in the People's Republic of China. Several global pharmaceutical companies have asked us about China, underscoring our belief that it's a good time to expand there.

Additionally, we recently announced a partnership with Berry Genomics, a Chinese company focused on the development and commercialization of genetic test technologies in clinical applications with approximately 1,500 employees and 7 different clinical laboratories. We believe that our partnership with Berry Genomics will be complementary since our customer focus is pharma and theirs is clinics and hospitals.

We have continued to expand our commercial team in both the U.S. and Europe. In particular, we have added business development staff with extensive commercial experience in companion diagnostic development. And as a result, we are now engaging with an increasing number of biopharma companies for potential companion diagnostic development programs. We've also expanded our quality and regulatory team to support these efforts. And in June, we had our first FDA pre-submission meeting for a single-site PMA for NeXT, which went well.

I'd now like to update you on the population sequencing part of our business, which is sometimes referred to in the field as population genomics. As you may have seen in our June press release, our population sequencing business hit a big milestone with the 75,000th full human genome sample sequence, and we are well on track to reach 100,000 by the end of this calendar year. This could make Personalis the first for-profit company ever to sequence 100,000 whole human genomes in the United States, and we are really looking forward to achieving this milestone.

Our work with the VA MVP represents the largest population sequencing effort within the United States. The VA now targets enrollment of 2 million veterans and over 825,000 veterans have enrolled so far. Personalis has been contracted so far to sequence over 116,000 VA MVP samples, with approximately 41,000 remaining to be sequenced.

Having already received population sequencing orders totaling over $145 million from the VA MVP, we are now expanding our commercial team to address and extend our reach into what is projected to be a multibillion-dollar population health market. We expect our unparalleled experience with the VA MVP program to position us well for new opportunities in population sequencing. Given our clinical experience and work with pharma, we also see an opportunity to help transition population research to population health and to involve pharma in the future.

Our population sequencing and biopharma businesses share a unifying theme in the comprehensive and large-scale genomic characterization of human samples. Both our whole genome sequencing and our NeXT platform cover all 20,000 human genes. Because of their shared underlying technologies and operational implementation, we achieved considerable synergy between the 2 businesses.

I would now like to expand on the synergy between the population sequencing and oncology parts of our business, in particular, synergies that go beyond the operational and cost synergies already achieved. Our extensive experience with whole genome sequencing, combined with our deep expertise in cancer, has allowed us to launch whole genome sequencing from cancer samples. We believe that this will be increasingly important in the future, particularly in cancers such as breast and prostate, which have relatively low mutational burdens.

Using our cancer whole genome technology will identify up to 20x somatic variants to serve as the basis for personalized cancer assays. We believe this will let us achieve high sensitivity, even in cancers which have low mutation rates, which have been surgically resected or which had relatively low amounts of cell-free DNA into the blood. We believe this can be a leading technology in some very large market opportunities, and we'll have more to say about this as these product developments progress.

Before I wrap up, I'd like to give you an update on another pipeline product. As many of you know, we have been working for over 3 years on a combined laboratory and informatics project to advance our neoantigen characterization capabilities. We believe that neoantigens are the crucial centerpiece of a new generation of companion diagnostic biomarkers. We expect our biopharma customers will apply this capability to mainstream cancer drugs such as checkpoint inhibitors and also to experimental personalized cancer therapies, which explicitly target neoantigens.

Our new advanced capability may also be used for development of drugs that are designed for use outside of cancer, such as gene therapies. This Personalis R&D project has required multiple proprietary technologies, including genetic engineering of proprietary human cell lines, mass spectrometry to identify and quantify peptides binding to HLA and the training of novel machine learning algorithms. Our data shows that this project has achieved a leapfrog advance in this field. We expect to launch this capability as part of our NeXT platform in the fourth quarter of this year.

In summary, I'm very proud that our combined business has shown strong resilience throughout the pandemic. Customer adoption for NeXT has been excellent, and our pipeline of compelling new products is rich. We believe these factors, among others, put us in a strong position for long-term growth.

With that, I will now hand it over to Aaron for our financial results.

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Aaron L. Tachibana, Personalis, Inc. - CFO [4]

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Thank you, John, and good afternoon, everyone. Revenues for the second quarter of 2020 were $19.5 million, up 2% from $19.2 million for the prior quarter and up 23% from $15.8 million for the same period of the prior year. The $19.5 million was a new record high for quarterly revenues. The quarter-on-quarter revenue growth was driven by an increase in volume for genomic testing services provided to biopharma customers. Biopharma and all other customers accounted for revenues of $4.7 million for the second quarter, an increase of 8% from last quarter.

In the second quarter, revenues from our NeXT platform began to ramp and exceeded $2 million. Also to note, the revenue increase from NeXT more than offset the sequential decline of over $1 million from both our biobank customer that had sample collection and shipment delays due to the pandemic and also biotech customers that were impacted by the slowdown of clinical trials.

For the second quarter, the VA MVP revenue of $14.8 million was flat from last quarter and was 73% higher compared with $8.5 million for the same period of the prior year. The VA MVP unfulfilled orders at the end of the second quarter were $39.3 million and based upon current estimates, we expect the unfulfilled orders to convert to revenue over approximately the next 2 to 3 quarters. The balance of the unfulfilled orders will decline from the fourth quarter of the prior year through the second quarter of the current year since the annual new order is typically received late in the third quarter.

Gross margin was 24% for the second quarter compared with 21.1% for the prior quarter. The VA MVP gross margins continued to be solid and once again were higher than the corporate gross margin reported for the second quarter. The VA MVP being higher volume and a single-service offering has been automated and does not require a significant amount of labor and has a very efficient sample test process.

In the second quarter, the negative impact to gross margin from COVID-19 was approximately 80 basis points from higher labor costs related to overtime pay. Also, we had a 100 basis point impact from higher facility costs related to an increase in lease rates. As a reminder, we may see gross margin variability in the future as there are a few moving parts, such as sample receipt linearity from customers, the mix of customer projects and capacity utilization of labor and equipment.

During our last conference call, we mentioned our initiative to build out a lab operation in China this year, which will add start-up expenses beginning in 2020, and most of these expenses will be classified as SG&A this year. We expect revenue from China to begin ramping throughout 2021. And during this ramp-up period, we expect to have gross margin headwinds from the under-absorbed labor and overhead.

Operating expenses were $14.2 million in the second quarter compared with $10 million for the same period of the prior year. R&D expense was $6.5 million for the second quarter compared with $4.5 million for the same period last year, and SG&A expense was $7.7 million in the second quarter compared with $5.5 million for the same period last year.

Net loss for the second quarter was $9.3 million compared with a net loss of $5.9 million for the same period of the prior year. The net loss per share for the second quarter was $0.29, and the weighted average basic and diluted share count was 31.7 million compared with a net loss per share of $0.89 and a weighted average basic and diluted share count of 6.6 million for the same period of the prior year.

Now on to the balance sheet. We exited the second quarter with a strong balance sheet with cash and short-term investments of $105.2 million. Second quarter cash flow from operations was a usage of approximately $15 million, primarily due to the net loss and working capital needs. During the quarter, we maintained our buffer inventory level of approximately $1.5 million to help mitigate potential supply chain disruption.

Now for discussion about our guidance. During our May conference call, we did not provide any 2020 revenue guidance due to the uncertainty from the pandemic. And although we had a solid second quarter, too much uncertainty remains about whether or not work conditions for us, our customers and suppliers will remain the same as today or change in the near future. Potential changes could have an adverse effect on our financial results. And therefore, we are not providing revenue guidance for fiscal 2020 at this time. We plan to provide an update to this information during our next earnings call.

Now I will turn the call back over to the operator, Jerome, to begin the Q&A session. Operator?

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Questions and Answers

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Operator [1]

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(Operator Instructions) Your first question comes from the line of Doug Schenkel from Cowen.

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Subhalaxmi T. Nambi, Cowen and Company, LLC, Research Division - Research Associate [2]

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This is Subbu on for Doug Schenkel. You posted a great update intra-quarter on the VA update. Given that you're on track to complete the project, how long -- so most of the database, given that you're on track to finish 100 case sequences, most often, we notice that database that has integrated electronic health record data has more value. Do you envision that to happen with the VA MVP project?

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John Stephen West, Personalis, Inc. - Co-Founder, CEO, President & Director [3]

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Yes. This is John West. I'm happy to answer that. Actually, a part of -- key part of the advantage that the Million Veterans Program has is that veterans have all been on the same electronic medical record system for years. And so they already have that in electronic format. And unlike other population sequencing efforts, they don't have to try to harmonize results from many different electronic medical record systems. All of the patients that are handled by the VA MVP are patients who would be -- have been on the same electronic medical record system during their time as patients with the VA. So it's actually really a major advantage that they have. I would also say that we're coming up on the 100,000 patients. That's nowhere near finishing the project. The project is the sequence -- to enroll 2 million patients. There are actually 825,000 samples that have already been collected. So actually, we're just beginning to scratch the surface of the project. We've been contracted to sequence 116,000, and we anticipate that there can be an additional order still this year that will extend the program even further.

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Subhalaxmi T. Nambi, Cowen and Company, LLC, Research Division - Research Associate [4]

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So you -- so if I heard it right, you're already doing that. You already have data with the -- the electronic health record data of all these individuals. Or do you plan to do that in the future?

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Aaron L. Tachibana, Personalis, Inc. - CFO [5]

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Let's see, our customer already has that information in the VA. They're the ones who have that health record data. They're the ones sharing it. It will be their database. They're the ones paying for it. But they do already have all of that health record data.

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Subhalaxmi T. Nambi, Cowen and Company, LLC, Research Division - Research Associate [6]

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Got it. And you recently partnered with Berry Genomics, and you also said that, that will serve as your local subsidiary opportunity. How do you plan to strategize that, given that NGS testing has just begun gaining traction there, and there are already a few high-profile local players? So what would be the key advantage? Would you call it your core technology? Or is there some other strategy that you're working on?

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John Stephen West, Personalis, Inc. - Co-Founder, CEO, President & Director [7]

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Yes. So thank you for the question. So the key reason that we are expanding to China is that there are international pharmaceutical companies that we already work with in the U.S. and Europe who are conducting international clinical trials. And so they have patients who are being enrolled into those clinical trials from China now in addition to other countries. And we've already been sequencing samples from patients that are from countries outside of China, but our pharmaceutical customers came to us and said, we're not allowed to export the samples of patients from China. We can't send them to you in California. They have to be sequenced inside China because of the regulations of China. And so they've asked us to set up this additional capability using our proprietary technology, our NeXT platform, to be able to run that inside China.

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Operator [8]

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Your next question comes from the line of Kevin DeGeeter from Oppenheimer.

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Kevin Michael DeGeeter, Oppenheimer & Co. Inc., Research Division - MD & Senior Analyst [9]

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Congrats on a really nice quarter. Can you just talk a little bit about the recently launched liquid biopsy program? Specifically, in addition to breadth of coverage, how else do you envision differentiating the product in the market, at least initially? And then in terms of the, what I'll call, incremental or second-generation product that would provide additional functionality for really kind of personalized precision oncology programs, just kind of walk us through the development steps that may be necessary to have that functionality.

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John Stephen West, Personalis, Inc. - Co-Founder, CEO, President & Director [10]

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Read the original here:

Edited Transcript of PSNL.OQ earnings conference call or presentation 6-Aug-20 9:00pm GMT - Yahoo Finance

]]]]]]>]]]]>]]> Verona will use the financing to support further development of its nebulised COPD drug ensifentrine. Credit: Shutterstock.

Sign up here for GlobalData's free bi-weekly Covid-19 report on the latest information your industry needs to know.

Chronic obstructive pulmonary disease (COPD) specialist Verona Pharma has closed an oversubscribed private placement and subscription with $200m raised from existing and new investors.

Once agency fees and other expenses are dealt with, the net proceeds of the placement are estimated to be around $183m.

The financing was led by new investors RA Capital Management, Access Biotechnology, Perceptive Advisors, Acorn Bioventures, PBM Capital, Samsara BioCapital, Foresite Capital, Sphera, Fairmount and Soleus Capital. Novo Holdings, Vivo Capital and other existing investors also participated in the placement.

Commenting on this financing, Verona CEO David Zaccardelli says: It is great to retain this expertise and to expand on it by adding additional support from new investors who also have deep knowledge of investing in drug development.

Verona is a public company listed on Nasdaq and the London Stock Exchanges AIM, which means it is more typical to raise funds through private placements of debt or stock, rather than through series rounds, explains Zaccardelli. He adds that the company is very pleased to have raised almost four times its market capitalisation, and calls this raise unprecedented.

Zaccardelli links the oversubscribed nature of this private placement to investors seeing value in our compelling and comprehensive data package for lead product ensifentrine for COPD, compared to placebo. Attracting such support provides a validation ofVeronaPharmas team, strategy and the potential opportunity for ensifentrine in respiratory disease, he adds.

Ensifentrine is a first-in-class drug, which combines a bronchodilator and anti-inflammatory agent into one molecule. Verona believes it has the potential to revolutionise COPD treatment, which currently involves dual or triple therapy with long-acting muscarinic antagonists (LAMAs), long-acting beta adrenoceptor agonists (LABAs) and inhaled corticosteroids, explains Zaccardelli. Not only is this approach burdensome on the patient, the combination fails to relieve the symptoms of millions of COPD patients.

New therapeutic options are urgently needed since the World Health Organization estimates the incidence of COPD is growing, and the lung disease is likely to be the third biggest killer in the world by 2030.

Verona is planning to use this $200m financing to fund Phase III trials of ensifentrine in COPD. These trials are expected to start later this year, and the funding will support the studies through to 2023. One of the trials will focus on ensifentrine as a monotherapy, and another will assess its efficacy as an add-on to either a LAMA or a LABA.

Zaccardelli concludes: The financing is a significant milestone and brings us closer to our goal of ensuring ensifentrine is available for the millions of COPD patients who urgently need better treatments.

Veronas Phase III programme for ensifentrine known as ENHANCE is being launched on the back of very promising results for ensifentrine in a Phase II programme that involved 16 clinical trials in a total of 1,300 patients.

In a statement, Zaccardelli noted: We continue to be very encouraged by the Phase II results that have demonstrated ensifentrines effects on lung function, COPD symptoms and quality of life as well as its favourable safety profile.

Although Verona has focused on studying ensifentrine in a nebulised formulation so far and this will be focus of the ENHANCE trials the company recognises patients may prefer handheld inhaler formats. Therefore, it has developed formulations of ensifentrine in dry powder inhaler and pressurised metered dose inhaler formats, according to Zaccardelli.

Verona has undertaken two successful Phase II trials of both dry power inhaler and pressurised metered dose inhaler formulations of ensifentrine in the last year. Following the publication of interim results in March 2020, the second half of the pressurised metered dose formulation trial has been postponed due to the Covid-19 pandemic.

To date, there have been no announcements from Verona about progressing these two formulations into Phase III studies.

View original post here:

Ovid and Angelini partner on treating Angelman syndrome in Europe - Pharmaceutical Technology

The University of Helsinki as research-oriented university offers a stimulating and international environment. The Faculty of Medicine of the University of Helsinki, together with Helsinki University Central Hospital, The Helsinki Institute of Life Science, and the Institute for Molecular Medicine Finland forms the Academic Medical Center Helsinki. This medical campus ranks among the 10 best medical centers at European level and the 50 best centers in the world. More info on the Faculty or Medicine and AMCH can be found here:

https://www.helsinki.fi/en/faculty-of-medicinehttps://www.helsinki.fi/en/meilahti-campus

Faculty of Medicine, Jackson laboratory, invites applications for a position of Doctoral Student / Research Assistant in mitochondrial biology and signalling for a fixed-term

The focus of the lab is to unravel fundamental signalling pathways and mechanisms controlling mitochondrial function. We are particularly interested in how mitochondria adapt signalling to metabolic cues in models of dysfunction including primary mitochondrial patient cell lines, cancer and associated disorders. We aim to exploit this knowledge on basic principles of mitochondrial signalling and subsequent metabolic susceptibilities in cellular models to help devise novel treatments of diseases that stem from misregulated mitochondrial function.

In this role the candidate will have the unique opportunity to drive an exciting project addressing limiting metabolic pathways in models of mitochondrial dysfunction by genome-wide CRISPR screening and mitochondrial structural analysis. The successful candidate will have access to a wide range of methodologies with work consisting in preparation of cell models, construction of libraries, and analysis of metabolic, proteomic and microscopic data at the cellular level.

We are looking for a highly motivated independent researcher with prime analytical skills driven by a passion for science. We expect you to formulate your own hypotheses, propose experimental design to address them, and take advantage of the laboratory and research communitys intellectual and skill resources to advance the knowledge of the field. The appointee is expected to publish in top-level international peer reviewed scientific journals. In addition, she/he is also expected to potentially take part on the other research tasks such as ones related to ongoing studies and preparation of grant applications. The specific responsibilities will vary according to the level of expertise of the candidate.

The ideal candidate has an extensive background in standard molecular cell biological methods, ideally encompassing genetic and microscopic techniques and/or bioinformatics (NGS data). Expertise in biochemistry and molecular biology is a pre-requisite for this position. Additional expertise for research projects involving human subjects, primary cell culture would be highly desirable. The ideal candidate is an adaptable team-player and has excellent communication and organizational skills and a strong command in written and spoken English and should be familiar with the constraints related to translational research.

This position will provide multiple opportunities for collaborations with potential lab visits abroad and cross-disciplinary scientific exchange, where the candidate will receive extensive training in cutting-edge technology. An aptitude to drive and maintain intra and inter team collaborations will be highly appreciated. In return, the candidate is offered access to an extensive variety of methodology and advanced techniques, appropriate supervision and help in career development.

More information about the lab, including the latest research and news, can be found here:http://www.jacksonlab.org

The position is initially limited to 2 years with a possibility of extension starting from September 2020. The contract of employment includes a probationary period of 6 months. Salary will be based on the Universities salary scheme for teaching and research personnel composed of both task specific and personal performance components.

The position will remain open until a suitable candidate will be recruited.

Please submit: CV, list of publications, motivation letter including a description of your research interests, and the names and telephone numbers of at least two referees.

Please submit your application, together with the required attachments, through the University of Helsinki electronic recruitment system by clicking on the Apply for job button. Internal applicants (i.e., current employees of the University of Helsinki) submit their applications through the SAP HR portal.

For further information, please contact Christopher B. Jackson, Ph.D., docent:christopher.jackson@helsinki.fi

Due date

31.08.2020 23:59 EEST

View original post here:

Doctoral Student/Research Assistant in Mitochondrial Biology and Signalling job with UNIVERSITY OF HELSINKI | 218596 - Times Higher Education (THE)

Associate research specialist Miranda Stauss and senior scientist Roger Wiseman process small vials of spit collected from volunteers.

At far right, Tom Friedrich, professor in the UW School of Veterinary Medicine, explains a consent form as his colleague Matt Reynolds, center, assistant professor in the UW School of Veterinary Medicine, hands a volunteer small vial for collecting spit as part of a trial of a new COVID-19 saliva test.

A volunteer spits in a small vial as part of a trial of a new COVID-19 saliva test.

A vial with saliva is processed as part of a trial of a new COVID-19 saliva test.

Wearing an N95 face masks, associate research specialist Miranda Stauss and senior scientist Roger Wiseman process small vials of spit collected from volunteers as part of a trial.

Researchers sort small vials of spit collected from volunteers.

Stauss processes small vials of spit.

Vials of spit are collected from volunteers as part of the trial.

Volunteers at four sites in Madison are being tested for the virus that causes COVID-19 by spitting in a vial, which may prove faster, cheaper and less complicated than other common tests, according to University of WisconsinMadison researchers.

Scientists from UWMadisons AIDS Vaccine Research Laboratory, a team that in recent years has also turned its attention to COVID-19 and Zika virus outbreaks as need arose, have tuned a relatively simple genetic testing process to find evidence of the novel coronavirus in saliva.

With support from a National Institutes of Health grant program that hopes to expand testing in the United States by fall, the researchers have collected hundreds of samples from volunteers at three UWMadison sites and a local elementary school. The tests were completed in hours, a stark contrast to common wait times of several days or even weeks for results from other kinds of COVID-19 tests.

Tom Friedrich, professor in the UW School of Veterinary Medicine, explains a consent form and the process of volunteers spitting in a small vial as part of a trial of a new COVID-19 saliva test.

This sort of testing, if it is successful and can be expanded, offers hope that schools and workplaces could receive rapid turnaround testing to assist in the complex decision of managing education during the outbreak with a test that is still sensitive enough to catch the people who are contagious, but exceptional in terms of accessibility, cost, and turnaround time says David OConnor, professor at the UW School of Medicine and Public Health.

They made their early findings available in late July in a brief study posted on medRxiv, a website for health sciences research that has not yet been peer-reviewed and published in a scientific journal. The test has not been approved for clinical diagnosis. The UWMadison researchers are studying whether this type of test can be administered frequently and efficiently.

Recent studies show that frequent, repeated testing is key to detecting infected people quickly, says Tom Friedrich, professor in the UW School of Veterinary Medicine. Because people can be contagious before they show symptoms of COVID-19, rapid testing can allow them to isolate and protect others before they even realize they are infected.

The project started in February even before the first COVID-19 cases appeared in Madison when OConnor and Friedrich were working with UW Hospital and Clinics to see if recent flu-like illnesses were actually the new virus.

We were interested in knowing whether there was silent spread of the virus in Madison, says OConnor. Fortunately, diagnostic testing became available very quickly. We shifted gears to adapting an alternative type of nucleic acid testing.

Most testing for SARS-CoV-2, the virus that causes COVID-19, uses a chemical process called polymerase chain reaction, or PCR, to make copies of the genetic material in a small sample so they are easier to identify. The Madison group employs a different method, called reverse-transcriptase loop-mediated isothermal amplification (RT-LAMP) to amplify the identifiable parts of virus available in saliva samples.

The advantage of RT-LAMP is that it is easier to set up than PCR, and doesnt require specialized instrumentation, OConnor says. We realized that this sort of testing might be more appropriate for places like workplaces, schools and nursing homes that might require on-site, frequent, repeated testing.

RT-LAMP also uses different chemicals than the PCR process, which has become so vital to pandemic testing that supply chains and manufacturing capacity have been stretched thin. And RT-LAMP requires fewer steps, using simpler and less expensive instrumentation than PCR.

I set up an (RT-LAMP kit) one Saturday afternoon and confirmed that indeed we could do the assay, says AVRL scientist Dawn Dudley. However, it soon became clear that this technique was not as sensitive as PCR especially in its easiest form.

Both PCR and RT-LAMP processes work better if the genetic material the nucleic acids that make up DNA and RNA are separated from the rest of the stuff in saliva, but the simplest version of RT-LAMP skipped that step. David Beebe, a UWMadison pathology professor with experience in putting lab tests on small, reproducible chips, and Salus, the Madison-based spinoff company he helped create in 2013, joined the group to design and produce an extraction process that would work outside lab settings and make the RT-LAMP test much more accurate with a small saliva sample.

Dudley and scientist Christina Newman spent months adapting the test for saliva, because the group expected people would get pretty tired of the common sampling method, a swab (now also in short supply) run sometimes deep into the nose.

Scientists Roger Wiseman and Miranda Stauss process small vials of spit collected from volunteers.

Collection is more comfortable, which is especially important if you are getting tested twice a week and important for children, Dudley says. Swabs can be quite invasive and somewhat damaging over time. Spitting into a tube? Not so bad.

Newman also set up the testing sites including AVRL and the Wisconsin National Primate Research Center where the researchers unload their gear from a minivan twice a week and collect samples across a folding table from as many as 60 people on some days.

Basically, people come, sign the consent, and spit into a tube that is left in a cooler, Dudley says. It takes less than five minutes.

With small groups, processing can be finished, results read via a color change in the test tube and delivered in a matter of hours and probably without a team of lab-trained scientists.

Other groups are also testing the effectiveness of the new test. Chris Mason, a UWMadison alum who is now a professor at Cornell University, is running a trial of his labs LAMP-based test with city workers in Racine, Wisconsin, where the collecting and processing is done mostly by firefighters. Salus is working on a commercial version of the test that can be deployed in small, mobile labs that Newman says could be straightforward enough to be operated by people without lengthy lab science training.

The researchers have run more than 400 tests, finding two positive cases and one that may be a false positive. Each result positive and negative, save the potential false positive has been confirmed laterby checking the saliva sample with the clinical-lab-standard PCR testing. Because the RT-LAMP test is not yet approved for clinical diagnosis of COVID-19 infection, the researchers have UW Hospital and Clinics doctors contact volunteers who tested positive and advise them to get a PCR test as soon as possible.

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Simpler COVID-19 test could provide results in hours from saliva - University of Wisconsin-Madison

In some parts of the U.S. right now, it can take weeks to get results for a simple Covid-19 test, a delay that renders the results largely useless.

So a handful of city governments and schools are turning to an entirely different type of Covid-19 test that they say is simpler, easier, and most importantly faster and therefore more meaningful.

The tests are like a streamlined version of the far more common PCR tests used to diagnose Covid-19. The tests simpler process requires fewer materials and less equipment; the results can be read by eye within an hour. But the technique known as LAMP can also, in some situations, be less sensitive and less accurate than PCR tests. And it can be hard to run many of the tests at once outside of a central laboratory; people can only work so quickly.

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Enter Color Genomics, a California-based, genetic testing company that says it has solved many of the problems associated with the technique. Its Covid-19 tests are just as accurate as PCR tests, according to the documents it filed to the Food and Drug Administration for an emergency authorization. And its automated the process enough to run thousands of tests each day. Already, Color is processing about half of all the daily tests run in San Francisco and returning results in one to three days.

But Colors improvements come with tradeoffs. Experts told STAT that an additional step in the companys process means it costs more and takes more time than others. Colors automation setup might not be cheap to replicate around the country. And since Color uses the same swabs and some of the same chemicals that so many PCR tests rely on, it could also face some of the same supply chain issues that have plagued other testing efforts.

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It opens up the flexibility of the supply chain to this PCR alternative, which is nice. But it doesnt overcome some of the common barriers to testing, said Matthew Lalli, a researcher studying genomics technologies at Washington University in St. Louis.

The technology behind the tests is known as LAMP, or loop-mediated isothermal amplification. Its been around since the 1990s, when a Japanese scientist developed it as a less-intensive alternative to PCR, or polymerase chain reaction-based tests.

Both PCR-based and LAMP-based tests look for genetic strands of a given virus in a sample collected from a patient. But PCR requires that a sample be repeatedly heated up and cooled down in precise intervals using an expensive machine known as a thermocycler.

LAMP does not require a thermocycler. The reaction can be run at a constant temperature about 65 or 70 degrees Celsius, or 150 degrees Fahrenheit. That makes it far simpler to run, and far less equipment-intensive. The reaction also creates a change in the acidity level of a sample; often, a compound that changes color from pink to yellow in different pH conditions is added to the tube, which means anyone can read the results.

Before this year, LAMP-based tests have been used for screening animal food for Salmonella as well as diagnosing C. difficile infections in humans and chlamydia in koalas.

And several groups around the world are developing new Covid-19 testing programs around LAMP. Many are relatively basic: In Racine, Wis., for example, researchers are running LAMP tests in an unused corner of city hall with minimal equipment and supplies, as Wired reported last month. Another Wisconsin group is running tests in elementary school parking lots. And a hospital in Germany is experimenting with a LAMP-based surveillance protocol developed at the Broad Institute.

But many of those point-of-care LAMP tests have an admitted shortcoming: Though they are simple, cheap, and fast, they are also less sensitive than PCR tests. Because of that, its often used for screening groups to try to catch most of the people who might have a disease not to formally diagnose someone.

Theres a type of PCR, for example, that has a nearly perfect sensitivity. You cannot beat that, said Jonathan Schmid-Burgk, a professor at University Hospital Bonn who has also developed a test that relies on LAMP technology and who is not affiliated with Color.

Colors LAMP-based tests solve that problem by adding a little complexity. Before Color actually runs the LAMP protocol on a swab, it extracts and purifies the RNA. This step, which is done on a particular machine that uses magnetic beads, concentrates the RNA.

This [step] directly translates to sensitivity, said Schmid-Burgk that is, it can make it more likely that a test will give accurate negative results.

The companys EUA paperwork indicates that for more than 500 samples, its test gave the same results as the Covid-19 diagnostic test developed by the Centers for Disease Control and Prevention.

Colors been using the technology for Covid-19 tests since April; the company got the first-ever FDA emergency authorization for the test in May.

The 7-year-old company is far better known for selling genetic tests work that investors and governments alike have been willing to fund. It raised $215 million from venture capitalists and received grants worth millions through the NIHs All of Us program, which intended to sequence q million Americans genomes.

When the pandemic came to the U.S., Color expected that it would be helping labs figure out how to automate certain processes to take some of the human effort out of running Covid-19 tests.

The deciding factor for us was the realization that no one we were talking to was taking an integrated approach similar to what had made us successful in genetics, said Color spokesperson Benjamin Kobren. Lab people were trying to build a lab. Logistics people were trying to set up drive-through sites, and so on.

But in the early days of the pandemic, supplies to run PCR tests were in very short supply. Colors team worried that competing with other labs for limited supplies might exacerbate the testing problem. So they looked at alternate protocols, like LAMP, that wouldnt add stress to some of the PCR supply chains.

The thing that made it good for point-of-care testing was actually the thing that we thought would make it really good for super high-throughput labs which is that its a relatively simple process, said Colors CEO, Othman Laraki. Were able to run almost the entire process on a single robot.

San Franciscos city government began working with Color in early April, according to a spokesperson. Color is one of a handful of labs behind the CityTestSF program, which offers testing by appointment for San Francisco residents and essential workers. The company charges about $100 per test, a San Francisco city representative told STAT; that figure is about the same as Medicares current reimbursement rate for Covid-19 PCR tests.

Kobren, the companys spokesman, noted that the price of a test can vary, depending on how many tests are included in a particular contract and what kind of group that contract covers.

Since the program began, Color says it has been able to deliver results far more quickly than many other laboratories. Results from Color are usually available within one to three days, the companys website states.

That kind of turnaround time has been critical, said Sarah Owens, the deputy press director for San Franciscos mayor.

Effective contact tracing depends on Covid-19 results being received in a timely manner, Owens said. Getting results back quickly allows us to contact people who test positive and begin the process of reaching out to their close contacts more quickly, thereby slowing the spread of COVID-19 in the community.

Colors LAMP tests are also the foundation of the University of Southern Californias testing program; the company said in a press release that it expects to run at least 500 tests per day on samples collected at three different sites.

Experts caution Colors test isnt suddenly going to replace PCR tests around the country.

For one, its product is proprietary; only Colors lab can process the tests, necessarily limiting how many can be run in a day. Colors worked hard to increase the number its automated process can now handle 10,000 tests per day.

That RNA purification step that Color added comes with a price.

Purifying RNA is really tedious, said Schmid-Burgk. Adding purification may increase a tests sensitivity. However, it also increases the time a test needs to process and can ultimately cuts down on the number of tests a lab can process each day.

You kind of lose the speed advantage by adding an RNA extraction step, said Lalli, the Washington University in St. Louis researcher.

Colors automated setup is also expensive. Replicating Colors automation would require about $500,000 worth of equipment, according to Chris Mason, a computational genomics specialist at Weill Cornell Medicine.

Even existing clinical laboratories with deep pockets might hesitate before ordering equipment and supplies to set up an entirely new protocol when PCR machines are already sitting in their facilities.

Most labs have PCR equipment, but many do not have LAMP, so it logistically makes it more difficult to bring up in the scale needed for widespread testing, a Quest spokesperson noted.

Another potential challenge: While some parts of Colors LAMP reaction uses machines and chemicals that are completely distinct from those needed for PCR tests, Colors process still requires some of the same supplies like swabs, RNA extraction kits and pipette tips.

The New York Times reported that those supplies may become scarce again soon which means that even Color itself could ultimately be unable to use its labs full capacity or unable to process tests as quickly as it is now.

But Colors team is optimistic that its prepared for that possibility. The company is hiring more people, adding additional equipment, and partnering with other labs that can run PCR tests in order to handle potential increases in demand. And Kobren, the company spokesman, told STAT that the company believes its automation enables us to reduce per-test consumption of scarce resources such as pipette tips and tubes.

We continue to invest resources and R&D to make our processes as efficient as possible.

Correction: A previous version of this story mistakenly identified Color as a direct-to-consumer genetic testing company. Its tests were never available directly to consumers.

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This company has a better version of a simpler, faster Covid-19 test - STAT

By Jennifer Plant Johnston

When Amy Ladd first began taking her 5-year-old son, Wesley, to SeRenade, a music class at Vanderbilt providing parent training and peer inclusion for kids with autism spectrum disorder, he had difficulty doing everything from putting on clothes in the morning tosettling down to sleep at night.

But by the end of the 12-week programa keystone project of the Vanderbilt Music Cognition Lab, supported through an award from the National Endowment for the Arts Research LabsLadd had acquired a musical toolbox of skills to help Wesley move more easily through the day.

We could add a song to getting ready, and things would go so much more smoothly, Ladd says. He would ask to listen to the CD from the class every time we got in the car. When my husband puts him to bed, he still asks for one of the calming songs to be played.

When behavioral challenges did emerge for Wesley, the researchers worked with the family to devise solutions. We were able to change our goals to be able to use music for him, Ladd says. Their support was huge for us.

Miriam Lense, MS10, PhD14, assistant professor of otolaryngology and psychology at Vanderbilt, and Sara Beck, BS01, PhD18, a psychology professor at Randolph College who is also an accomplished singer and songwriter, with input from music therapists, behavior analysts, and caregivers of young children with ASD. In addition to providing parent training, the classes allow researchers to explore the impact of community-based parentchild music programs on families of preschool-aged children with ASD, as well as the ways music intervention could impact children with the disorder.

The positive effects of music training on brain development, especially in children, have been well documented, and scientists in recent years have become increasingly interested in neural entrainmentthe use of rhythmic activity to stimulate sensory gain. The past decade in particular has been marked by a dramatic increase in music cognition inquiry, as about 100 laboratory groups around the world, including at Vanderbilt, are working across disciplines to understand musics relationship to the brain, behavior and health, and to develop effective intervention strategies.

Lense co-directs Vanderbilts Music Cognition Lab with Reyna Gordon, a fellow assistant professor of otolaryngology and psychology. Currently housed in Vanderbilt University Medical Center and the Curb Center for Art, Enterprise and Public Policy, as well as a new space on Nashvilles famed Music Row, Lenses and Gordons work is a central part of a campuswide network of music research originally seeded by funding from a Trans-Institutional Programs, or TIPs, grant.

Music and rhythm are a ubiquitous part of everyday life and are present in cultures around the world, says Lense, pointing out that rhythms also occur through speech and movement. Were excited to get to study music scientifically to understand the mechanisms by which musical experiences may impact development, as well as to try to harness music through clinical or applied studies.

Vanderbilt is a natural environment for the music lab, and not just because of its location in Music City. Lense was drawn to graduate school at Vanderbilt by the opportunity to work under the tutelage of Elisabeth Dykens, professor of psychology and human development, pediatrics, and psychiatry and behavioral sciences. Among other areas, Dykens has expertise in the study of Williams syndrome, a rare genetic neurodevelopmental condition often associated with special musical abilities. Lense first became interested in Williams syndrome as a Harvard undergraduate.

Dykens began the annual Academy of Country Musics Lifting Lives Music Camp in partnership with the Vanderbilt Kennedy Center for Research on Human Development, which also hosts the event, for people with Williams syndrome. Each year campers participate in research and have a really phenomenal music-making experience, Lense says. They write a song with a Nashville songwriter, record it in the studio, and perform on the Grand Ole Opry within a week of being here.

Lense and Gordon first began collaborating at the Kennedy Center in 2011 when Lense was a clinical psychology Ph.D. student and Gordon was a postdoctoral fellow researching intellectual disabilities. After Gordon joined Vanderbilts faculty, she helped lure Lense back to the university from a postdoctoral position at Emory Universitys School of Medicine.

In addition to collaborating in the Music Cognition Lab, Lense and Gordon are good friends who talk every day. And like most of the students in the lab, they both march to a musical beat. Lense studied classical composition, violin and oboe. Gordon is a classically trained singer who majored in music as an undergraduate, with a minor in Italian.

I have always been interested in music and language and the brain, and it took a few years for me to find a way to combine those interests, says Gordon. She completed her masters-level work in neuroscience at Universit de Provence in a French-speaking lab in Marseille doing cutting-edge language and music research using EEG (electroencephalogram) and fMRI (functional magnetic resonance imaging) methods to measure how the brain responds to singing.

Lense and Gordon continue to draw on their musical training in their daily work. In both composition and research, Lense says, youre building upon knowledge and experiences to create something novel and true.

Lense has a special interest in the relationship between music and social communication in individuals with and without developmental disorders. In addition to rhythm in social communication and parentchild musical engagement experiences, she continues to explore her interest in the musical abilities of people with Williams syndrome and studies the potential for musical activities that can scaffold other skills in infants and toddlers with ASD.

We know that children with autism, for example, have difficulty with eye gaze and also with the timing of social interaction, Lense says. We conduct studies using rhythm and music to assess how sensitive children are to the timing of social cues. We may then be able to use these rhythmic cues to help support their interactions.

In one such study, the lab has partnered with the Vanderbilt Bill Wilkerson Center for Otolaryngology and Communication Sciences and the Marcus Autism Center at Emory to study musical rhythm synchronization in hopes of developing musical interventions to improve social communication in children with ASD. The study is part of the Sound Health Initiative, a partnership of the National Institutes of Health and the John F. Kennedy Center for the Performing Arts, in association with the National Endowment for the Arts.

Preliminary data suggest that when typically developing infants see videos of someone singing, they are more likely to fixate on the eyes of the singer during predictable, rhythmically important moments. Singers also are particularly expressive during these moments. This suggests that the rhythm of communication really can be helpful for transferring social information, Lense explains.

Gordon, the recipient of a 2018 National Institutes of Health Directors New Innovator Award (DP2HD098859), leads a research team focused on exploring the relationship between rhythm and grammar skills, especially in children. She utilizes a variety of methods, including genetics, EEG and cognitivebehavioral assessments to understand the biology underlying language and music, with a special interest in populations who face hurdles to learning, such as dyslexia or developmental language disorder.

A new National Science Foundation grant, awarded to the lab in 2019 in partnership with researchers at Middle Tennessee State University, provides funding for a series of studies that looks at the relationship between rhythm and reading skills at behavioral, neural and genetic levels. The grants main co-investigators are Vanderbilts Assistant Professor of Biological Sciences Nicole Creanza and Associate Professor of Medicine Piper Below, as well as MTSUs Cyrille Magne.

Individuals who have dyslexia are more likely to have poor rhythm sensitivity in both language and music, Gordon says. The research focuses on discerning individual differences in sensitivity to rhythmic and intonational fluctuations in speech and determining how those differences relate to reading skills. The team is hopeful that results of the project eventually could be applied by using rhythm in speech and music to help individuals improve reading proficiency, closing the gap in college readiness among young adults.

Even when outside the lab, Gordon is always attuned to the profound impact that music can have on those listening to itand to the neuroscientific processes that explain that experience.

Every time I go to a Nashville Symphony concert, Im absolutely floored by the incredible skill of the musicians: They are true experts in auditorymotor integration, she says. But Im also struck by how our brains as audience members are so good at tracking the beat, predicting what comes next, and processing information on a millisecond time-scale. Im so fortunate that I get to devote my professional life to studying how and why this occurs.

Gordon and Lense believe strongly in creating multidisciplinary teams, particularly when they offer training opportunities for musicianscientists at the undergraduate level. Both undergraduate and graduate students are fully engaged in the labs work, from chronicling study interactions and conducting literature searches to using sophisticated technology, such as EEG.

For example, on a day at the lab earlier this spring, Mine Muezzinoglu, BA20, a recent graduate from Istanbul who majored in neuroscience and French, viewed previous SeRenade classes on a computer monitor, taking detailed notes to track how parents and caregivers such as Ladd integrate the tools they learn as part of the music class. She also noted the childrens engagement in the class.

Meanwhile, Peyton Boyd, a senior from St. Louis majoring in neuroscience and medicine, health and society, was at work on a project in which adult participants tap along to various stimuli to see how well they do in conditions where the rhythm of the stimuli has been disrupted, with the aim of someday extending this research to other populations, such as to children with ASD. Teaming with Boyd on that project was Brett Koolik, a biomedical engineering major and aspiring musician from Boca Raton, Florida, who runs the nearby recording studio in the Curb Center.

I just love how collaborative the lab is, says Koolik, who also is working on a study examining the effects of songwriting on caregivers, using the creative process to facilitate coping skills for managing stress and mental health concerns.

The Music Cognition Lab trains students and faculty from across the university and Vanderbilt University Medical Center, encompassing academic disciplines as diverse as auditory processing, childhood language development, music performance, sensorymotor systems, comparative ethology, genomics, developmental psychology, special education and computational modeling. Different perspectives and viewpoints strengthen the outcomes, says Lense. It challenges you to do really rigorous research.

The close proximity of the university campus to the Medical Center was one reason Anna Kasdan chose Vanderbilt for her neuroscience Ph.D. training. That doesnt exist in a lot of places, she says.

A trained classical pianist, Kasdan works both with Lense, on a study of individuals with Williams syndrome, and with Gordon and Stephen Wilson, associate professor of hearing and speech sciences, on understanding rhythm processing in individuals with aphasia, an acquired language disorder due to brain injury.

Because of her interest in the socialemotional benefits of music in aphasia, Kasdan received starter funds from the Curb Center to host a music and arts program for individuals with aphasia, ranging from 20 to 70 years old. The program was organized in conjunction with Deborah Levy, a Ph.D. student in hearing and speech sciences, through the Aphasia Group of Middle Tennessee, directed by Dominique Herrington, assistant manager of rehab services for the Vanderbilt Department of Hearing and Speech Sciences Pi Beta Phi Rehabilitation Institute.

I did not imagine I would get to be involved with so many different populations so early in my graduate career, says Kasdan, a third-year graduate student in neuroscience. Im getting to work with the community and learn several methodologies, including EEG. I got to jump into projects that Im excited about early on, which are already branching off into other projects.

Such training and community outreach opportunities are needed to provide musicianscientists such as Kasdan the ability to pursue and develop seemingly disparate interests, the researchers say.

Meanwhile, two years after Ladd and Wesley participated in SeRenade, she continues to draw from her musical toolbox to tackle daily behavioral and learning challenges. Wesley has learned to make up songs when enduring long wait times that used to upend his composure, his mother says.

He is learning to spell words right now, Ladd says, and when there is a harder word, like purple, we add a little singing and clapping to help him remember how to spell it. I never realized how useful music could be until we took the class.

Jennifer Plant Johnston is a Nashville-based freelance writer with more than 30 years of experience, many of them writing for Vanderbilt. A former Associated Press reporter, she was executive director of the Vanderbilt Center for Nashville Studies before retiring in 2016 to travel and write.

Miriam Lense, MS10, PhD14, assistant professor of otolaryngology and psychology at Vanderbilt, and Sara Beck, BS01, PhD18, a psychology professor at Randolph College, recently were featured guests on the new webinar series Music and Mind Live with Rene Fleming.

The series, which is hosted by celebrated opera singer Rene Fleming in conjunction with the John F. Kennedy Center for the Performing Arts, explores the intersection of music, health and neuroscience. Lense and Beck appeared on an episode titled At Home with Children: Musical Toolkit, in which they discussed the SeRenade parentchild music program they co-developed at Vanderbilt.

Parents naturally develop relationships and patterns with their children that provide meaningful and rewarding social experiencesand one of those ways is through music, Lense said during the interview. Our social interactions already have a musicality to them. Theres a rhythm to them in terms of how we interact with other individuals, and children are very responsive to this.

Added Beck, As humans we have this history of music-making as this very active, physical thing we do together in a shared space. And it has these qualitiesa shared attention and a shared intentionality around itas well as this quality of moving together. Theres a lot of interesting research showing that just moving together acts as a cue for social bonding. It makes us feel closer to one another.

To watch the interview in its entirety, visit vu.edu/fleming-webinar.

SETH ROBERTSON

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Mental Notes: Music Cognition Lab is dedicated to the scientific study of how music affects the brain and behavior - Vanderbilt University News