Targeted antibiotics are designed to customize care to a particular patient, i.e. medical therapies based on symptoms, health status, patient history, body physiology, and patient physiology and disease factors related. Targeted antibiotics are often referred to as precision medicine, stratified medicine, personalized medicine or P4 medicine. Diagnostic screens are often done based on patient genetic contents or other molecular or cellular tests of targeted antibiotics to assess appropriate and effective treatment. The best response and safety margins to ensure better patient care are provided for targeted antibiotics by requiring each patient to receive the precocious diagnosis, risk assessment, and best treatment.

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The global targeted antibiotics market is expected to grow rapidly because of the rising occurrence of disease and the emphasis on medicinal products for patients who are more beneficial than other therapies. Rapid outcomes and tailored effects, the lower likeliness of therapeutic faults and adverse drug reactions are some of the other considerations that can build sufficient momentum for R&D and those drugs sales potentials. Governments favorable policies regarding personalized medicine will also create incremental opportunities to tap this market. Recent pharmacogenomics advances are expected to create an appropriate environment for the development of drugs unique to one individual or community. Developing genetic databases could provide more market boost. Opportunities for therapeutic application in fields such as cardio-renal, neurology, antiviral, pulmonary, psychiatry, among others, may create momentum in R&D across all regions. The rise in the prevalence of various cancer types, the availability of specialty therapy in cancer drugs and numerous other disease indications, the reduction in the side effects of personalized care, high penetration of developing markets and the development of new medicines are factors that drive the international customer market. Nevertheless, the market is likely to be hindered in the coming years by high competition among existing market players, strict government regulations concerning product approval and lack of awareness among rural people in developing economies.

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The detailed research study provides qualitative and quantitative analysis of targeted antibiotics market. The market has been analyzed from demand as well as supply side. The demand side analysis covers market revenue across regions and further across all the major countries. The supply side analysis covers the major market players and their regional and global presence and strategies. The geographical analysis done emphasizes on each of the major countries across North America, Europe, Asia Pacific, Middle East & Africa and Latin America

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Targeted Antibiotics Market was estimated to grow at 4.4% CAGR during the forecast period due to rising focus on providing precise medicine care to...

CARLSBAD, Calif., July 13, 2020 /PRNewswire/ --Thermo Fisher Scientific and First Genetics JCS, a manufacturer of diagnostic equipment and IVD kits intended to advance molecular genetic technologies into clinical practice, today announced a strategic partnership focused on commercializing next-generation sequencing (NGS)based diagnostics in Russia. The agreement enables First Genetics to market its F-Genetics NGS System and IVD assays to Russian labs for reproductive health testing and cancer diagnostics. The F-Genetics System is based on Thermo Fisher's Ion GeneStudio S5 System.

First Genetics has already received its first registration certificate for an IVD kit by Roszdravnadzor, the Russian medical device regulatory agency. The company's Reproline kit is now approved for detecting chromosomal abnormalities in embryos on the F-Genetics NGS System prior to IVF implantation. While IVF is now government-funded as part of Russia's policy to help boost the national birth rate, preimplantation genetic testing for aneuploidies (PGT-A) had not previously been certified in Russia. The certification now enables Russian prenatal centers, which have doubled in number over the last decade, to leverage PGT-A.

"High-throughput technologies for genetic analysis are the future of molecular diagnostics," said Nikolai Kuznetsov, CEO, First Genetics. "Today we have expanded applications of NGS into reproductive medicine in Russia, and in the future, we intend to offer clinically certified solutions for other clinical applications, such as oncology testing. This is our contribution to the development of clinical diagnostics in Russia."

Thermo Fisher has entered into a number of agreements with assay developers like First Genetics to expand the use of NGS-based molecular diagnostics in the clinic and to advance precision medicine globally. Similar partnerships with other organizations have led to approvals by regulatory agencies in China, the United States and Europe.

"It is our mission to help bring molecular testing to all parts of the world so those who need it most can benefit from the genetic information next-generation sequencing provides," said Yan Zhang, general manager, reproductive health at Thermo Fisher. "We are pleased that couples in Russia now have access to First Genetics' solutions to assist in family planning and reduce the emotional burdens they face while undergoing fertility treatment."

About Thermo Fisher ScientificThermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue exceeding $25 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, improving patient diagnostics and therapies or increasing productivity in their laboratories, we are here to support them. Our global team of more than 75,000 colleagues delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services and Patheon. For more information, please visit http://www.thermofisher.com.

About First Genetics JSCFirst Genetics JSC is a Russian manufacturer of diagnostic equipment and IVD kits based on high-performance sequencing, a partner of Thermo Fisher Scientific, a world leader in this field. The company has a licensed laboratory for conducting medical genetic tests. The company's goal is to introduce advanced molecular genetic technologies into clinical practice and improve the in vitro diagnosis of human diseases. http://www.f-genetics.com

Media Contact Information:Mauricio MinottaPhone: 760-929-2456E-mail: [emailprotected]

Jen HeadyPhone: 617-275-6547E-mail: [emailprotected]

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Thermo Fisher Scientific and First Genetics JCS Partner to Develop Next-Generation Sequencing-Based Diagnostics in Russia - PRNewswire

Published inNature,the National Lung Matrix Trial (NLMT) is the worlds largest precision medicine clinical trial for non-small-cell lung cancer (NSCLC) patients, funded by Cancer Research UK (CRUK) and supported by the charitys Stratified Medicine Programme Phase 2 (SMP2) screening platform.

A 25 million collaboration with Pfizer, AstraZeneca and other pharmaceutical companies, and with support from the NHS, the NLMT matches different treatments to different groups of patients based on genetic changes in their cancer.

Using an innovative trial design that incorporates multiple treatment arms, NLMT allows for a more flexible and informed approach than traditional clinical trials. Each arm tests a different targeted treatment matched to a different genetic subtype of NSCLC.

The trials adaptive approach makes it possible for new drugs and combinations to be added as soon as they become available or retired quickly and easily if evidence suggests they arent effective.

Patients who sign up to take part in NLMT are genetically screened by the Cancer Research UK's SMP2 to understand more about their tumour type, and whether they possess the relevant genetic signatures to be matched to a targeted treatment.

Since the trial opened to recruitment in May 2015 until November 2019, 288 patients have been recruited to the 19 targeted treatment cohorts on the trial.

Results revealed key learnings on the use of precision medicine, such as how the appropriate pre-clinical work is essential in defining appropriate biomarker-drug combinations to test in the clinic, on ensuring that the best drugs available are used to hit the genomic targets, the scale of attrition from large screening platforms, and the importance of analysing and publishing outcome data of an ongoing study.

The attrition rate was high with only a 5% of SMP2 patients receiving treatment on NLMT. As trials often come towards the end of the patient journey, cancer progression might make patients too unwell to be recruited onto trials. Targeted therapy trials should take place much earlier in the cancer journey and use blood based genomic testing for fast turn-round of the information needed to match patients to drugs.

Lead authorProfessor Gary Middleton, Medical Oncologist at the University of Birmingham, said: This study provides data on how the next wave of trialling targeted therapies to treat complex cancers should be designed. This is the first trial of its kind, the novel Bayesian design allows outcome data from open cohorts that are still recruiting to be reported alongside closed cohorts, whereas previous umbrella studies have only published the results of completed arms and cohorts.

"We found genomically complicated tumours are hard to treat with targeted therapy, especially with monotherapy. The models we test drugs on are too simplistic - they dont represent the genomic complexity of the tumour, or the trajectory of how they rapidly evolve. We need models that take into account the complexity and trajectory of a human tumour to decide if a drug is going to work.

Professor Pam Kearns, Director of Birminghams Cancer Research UK Clinical Trials Unit and University of BirminghamsInstitute of Cancer and Genomic Sciences, said: The National Lung Matrix Trial is a landmark complex innovative design trial and represents a step-change in our understanding of how to develop precision medicine in challenging to treat cancers.

Dr Ian Walker, Director of Research at Cancer Research UK, said: The National Lung Matrix trial is a flagship programme for Cancer Research UK and continues to provide significant insights into how we should treat genomically diverse cancers, such as lung. Not only will it shape the thinking for future studies delivering complex precision medicines, but it has also demonstrated how molecular diagnostic testing and clinical research can work in a truly integrated manner within the National Health Service across the UK to allow patients to access the latest new treatments.

NLMT patient Paul Inett, 72 years old, said: I was diagnosed with stage four metastatic lung cancer in late April 2015 that had spread to glands in my chest. I was referred to Professor Gary Middleton at the Queen Elizabeth Hospital Birmingham and was recruited onto the National Lung Matrix Trial. Fortunately my biopsy showed a genetic marker that matched to one of the treatment arms on the trial and received a targeted cancer drug. This ground breaking research saved my life. Im now able to live a relatively active life including spending time with my family and taking exercise classes at my local gym.

Next steps in the NLMT are to continue recruiting patients. A new combination arm has just been added to the study and opened earlier in June. There will also be further publications from the individual arms and cohorts, and translational laboratory work.

For more information please contact Sophie Belcher, Communications Manager, University of Birmingham, on +44(0) 7815607157. Alternatively, contact the Press Office out of hours on +44 (0)7789 921165.

About the University of BirminghamThe University of Birmingham is ranked amongst the worlds top 100 institutions. Its work brings people from across the world to Birmingham, including researchers, teachers and more than 6,500 international students from over 150 countries.

About Cancer Research UK

For further information about Cancer Research UK's work or to find out how to support the charity,please call 0300 123 1022 or visitwww.cancerresearchuk.org. Follow us onTwitterandFacebook

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Flagship Cancer Research UK precision medicine trial highlights the need for a new approach in treating genomically complicated cancersUntitled -...

In this world we go around once, sometimes we live for years close to a century, other times our life is cut short in our 50s, 60s or even earlier. When a person passes through this life and makes it a better world, they are remembered forever and their dedication and work to complete the task does not go unnoticed.

This is where Dr. Steve Bahrke, a Wautoma High School graduate, has made a difference. He was a husband, father, and brother, grandfather and had a plethora of friends, in the medical field and out. Steve was the youngest of four children growing up in Wautoma. He was the fourth child of Ervin Bahrke and Dorothy Roeske Bahrke. Ervin passed away at the age of 61 in 1975. His brother Jim was his soul mate and they spent many hours together on adventurous escapades and fun-loving practical jokes. His sisters, Ann and Barb, enjoyed watching their brothers raise some cane and the brothers were always there for their sisters.

While in high school at Wautoma Steve found a love for science. He attended he University of Wisconsin Oshkosh with a major in chemistry and a minor in mathematics. During a summer internship at his childhood Doctors family practice, Dr. Darby, he found a passion for medicine. He graduated from UW-Oshkosh and did research for the University of Wisconsin in genetics lab and McArdle Laboratory for Cancer Research while he prepared for medical school. He graduated from UW-Madison medical school in 1979. He was assigned to a public health hospital on the Gila River Indian Reservation in Arizona for five months.

Steve matched for his medical residency in family medicine with the Fox Valley Family Medicine Residency Program in Appleton. During his residency, he met Dr. Pete Sanderson and they agreed to start a practice together in Plover. Dr. Bahrke went on to build a new larger, state of the art clinic in Plover. Many came to study the design and replicate it in other clinics around the country. When they enlarged the clinic they welcomed Dr. Mark Felon as the third partner.

Being a doctor, one would think all is a slam-dunk and you care for your patients and Steve did that and went beyond to be a doctor that went out of his way to help others. Steve was having some health issues himself and found out that he had a genetic disease, Cadasil, and after testing the family two of his siblings, Ann and Jim, also had the disease. This sent Steve on a path that would put in him medical history as he began research on the disease and some suggested treatment that would slow the progression down. Many medical institutions have documented the research. Steve dedicated his life to the research and lost his life fighting it, but the work he did to help others is life changing.

Steve married Kathy Hartnet in 1971 and they had three children, Erin (Jeremy), Scott, and Michael (adopted at the age of 4 from Thailand). Later he married his loving wife, Debra Hopp, in Hawaii. She brought an additional two children into the family. When the affects of Cadasil made practicing medicine too difficult, Steve and Debb retired to Hernando, FL for the winter where he continued with golf, fishing, and movies. He also enjoyed refinishing antique furniture and doing lawn care. They spent their summers in Conover, WI, where they enjoyed boating, fishing, and watching eagles.

A Memorial Service for Dr. Steve Bahrke will be held on Saturday, July 25th from 11 a.m. to 2 p.m. at Pfiffner Building, 401 Franklin St., Stevens Point. Dr. Bahrke may have left this earth, but the research he did and documented on Cadasil will remain a legacy. The love he had for family and friends and for his dear mother, Dorothy Bahrke Pearsall, a resident at Heartland House, Wautoma, his wife, his children, his brother and sisters, will live forever in the hearts of those that remain on earth to cherish his memory.

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Wautoma native Dr. Steve Bahrke to be remembered for his love of family and his contribution to the genetic disease, Cadasil, that claimed his life on...

Each week, The Dailys Science & Tech section produces a roundup of the most exciting and influential research happening on campus or otherwise related to Stanford. Heres our digest for the week of July 5 July 11.

Genetic evidence links Polynesians and Native Americans

Scientists have determined from genetic evidence that ancient Polynesians and Native Americans made contact in present-day Colombia hundreds of years ago, concluding a decades-long debate in the field of archaeology, a study published on July 8 in Nature reports. Previously, there was much contention as to whether the two groups were connected, given that they were separated by thousands of miles of ocean.

The researchers conducted genetic analyses of more than 800 Indigenous individuals living across Mexico, Polynesia and various South American countries to find evidence of common ancestry.

Genomics is at a stage where it can really make useful contributions to answering some of these open questions, Alexander Ioannidis M.S. 18, Ph.D. 18, a postdoctoral research fellow in biomedical data sciences, told Stanford Medicine News. I think its really exciting that we, as data scientists and geneticists, are able to contribute in a meaningful way to our understanding of human history.

The findings suggest that there were common genetic signals between Native American and Polynesian DNA tracing back centuries.

If you think about how history is told for this time period, its almost always a story of European conquest, and you never really hear about everybody else, Ioannidis told Stanford Medicine News. I think this work helps piece together those untold stories and the fact that it can be brought to light through genetics is very exciting to me.

Phytoplankton growth increases in the Arctic Ocean

In the Arctic Ocean, the growth of phytoplankton tiny creatures that cause algae blooms has increased significantly over the past two decades, a study published on July 10 in Science found. This represents a significant regime shift as the Arctic waters become warmer due to climate change.

The team studied the net primary production (NPP), a measurement of how quickly plants and algae convert sunlight and carbon dioxide into sugars for other organisms.

The rates are really important in terms of how much food there is for the rest of the ecosystem, earth system science professor Kevin Arrigo told Stanford News. Its also important because this is one of the main ways that CO2 is pulled out of the atmosphere and into the ocean.

The findings suggest the NPP increased by 57% in the Arctic Ocean between 1998 and 2018. As a result, phytoplankton numbers grow more concentrated, potentially leading to more algae blooms.

Theres going to be winners and losers, Arrigo told Stanford News. A more productive Arctic means more food for lots of animals. But many animals that have adapted to live in a polar environment are finding life more difficult as the ice retreats.

Its taking in a lot more carbon than it used to take in but its not something were going to be able to rely on to help us out of our climate problem, he added.

Protein identified as possible target in rare genetic disorder

A newly identified protein may be an ideal drug target for Diamond Blackfan anemia, a rare genetic disease that affects red blood cell development, a study published on July 3 in Nature Communications found.

One of the difficulties of treating patients with Diamond Blackfan anemia is that the mutation which causes the disease varies across patients. The researchers approached the issue by examining which faulty cellular mechanisms protein movement or enzyme activity patients have in common.

The study identified an enzyme called Nemo-like kinase. In Diamond Blackfan anemia patients, this enzyme is overactive. The team found that slightly inhibiting the kinase activity reversed problems with red blood cell development.

The team is led by pediatrics, hematology and oncology instructor Mark Wilkes and professor Kathleen Sakamoto. Moving forward, they are working to identify potential new drugs to target the protein.

Contact Derek Chen at derekc8 at stanford.edu.

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Research Roundup: Ancient Polynesian and Native American contact, Arctic phytoplankton, protein link to rare genetic disease - The Stanford Daily

Researchers at Weill Cornell Medicine-Qatar (WCM-Q) have helped probe the genetic diversity and origins of the Arabian horse, prized all over the world for its beauty, grace and athletic endurance.

Renowned for its ability to thrive in extremely hot, arid environments, the Arabian is the oldest recorded breed of horse, with credible documentation stretching back more than 2,000 years placing its development in the Middle East.

Working in collaboration with an international team of fellow researchers, scientists at WCM-Q helped conduct a comprehensive global sampling and analysis of the genomes of 378 individual Arabian horses in Qatar, Iran, UAE, Poland, USA, Egypt, Jordan, Kuwait, the United Kingdom, Australia, Denmark and Canada. Blood and hair samples were painstakingly collected from the horses over an eight-year period.

The international team of scientists was led by the University of Floridas Samantha Brooks, a UF/IFAS assistant professor of animal sciences formerly based at Cornell University in Ithaca, New York; Doug Antczak, the Dorothy Havemeyer McConville Professor of Equine Medicine at the Baker Institute for Animal Health of Cornell University; and Andy Clark, the Jacob Gould Schurman Professor in Cornells department of molecular biology and genetics.

Researchers at WCM-Q, led by Dr. Joel Malek, Associate Professor of Genetic Medicine, used the colleges state-of-the-art equipment and expertise to assist with the sequencing of the horse DNA. The study was made possible by National Priorities Research Program (NPRP) grant 6-1303-4-023 from the Qatar National Research Fund, a member of Qatar Foundation. The paper, entitled Genome Diversity and the Origin of the Arabian Horse has now been published in Scientific Reports, a journal of the Nature series of publications

Brooks said: The Arabian horse has a special mystique due to the long recorded history of the breed. Arabian horse breeders, in particular, know their horses bloodlines many generations back. What we found was that in the area where this breed originates likely the near East region, but we dont know exactly theres a healthy level of diversity. This is particularly evident in populations from Bahrain and Syria, which suggests these are some pretty old populations.

The horse is prized for characteristics like heat tolerance and endurance, as well as its unique appearance, with a dish-shaped facial profile, wide-set eyes, an arched neck and a high tail carriage. It has been exported from its ancestral homeland for centuries, with some modern lineages drawn strictly from these smaller genetic pools, giving the breed a reputation for inbred disorders. While this was true for some groups they tested, Brooks noted, they also found remarkable diversity when considering the breed as a whole.

Brooks contrasted the discovery of more diverse populations with the samples they received from racing Arabians. Another longstanding myth says that the Arabian contributed genetically to the modern Thoroughbred, but the racing Arabians DNA told a different story.

What we found in these samples was not that much Arabian ancestry was part of the Thoroughbred line, but the opposite: that Thoroughbred DNA exists in most of the modern racing Arabian lines, indicating a more recent interbreeding within this group, Brooks said. I cant speculate on the how or why, but this is clearly the story the DNA is telling us.

Another implication of this study, Brooks said, is the potential to identify the genetic regions that determine some of the Arabians unique traits, like their facial profile. This could be expanded to identify the marker for other horse breeds head shapes, for example.

Dr. Joel Malek of WCM-Q said: It was extremely gratifying to be part of this fascinating and interesting research about the iconic Arabian horse, which is so important to this region. We are very proud to have been able to work with so many talented and dedicated researchers all over the world on this project, which underlines WCM-Q's commitment to pursuing projects at the cutting edge of science that have great local significance, in line with the goals set out in Qatar National Vision 2020.

We are also very grateful for the support this project received from Qatar Foundation through Qatar National Research Fund, which made the research possible.

The study has a long list of co-authors, with contributors from the University of Tehran, Iran; the University of Kentucky; the University of Agriculture in Krakw, Poland; the Hong Kong Jockey Club; the Equine Veterinary Medical Center at Al Shaqab (a Qatar Foundation member) in Doha, Qatar; and the University of Veterinary Medicine Vienna, Austria. Elissa Cosgrove from the Clark lab and Raheleh Sadeghi, a visiting scientist from Iran in the Antczak lab, shared first co-authorship of the study.

Dr. Khaled Machaca, Professor of Physiology and Biophysics and Senior Associate Dean for Research, Innovations, and Commercialization at WCM-Q said: It is extremely pleasing that the advanced capabilities in genomic analysis that we have developed in the Biomedical Research Program at WCM-Q allow us to contribute to ground-breaking international projects such as this one. The Arabian horse is truly a majestic creature and it is wonderful to be able to shed light on its genetic heritage in this way.

The study also had contributors from the University of Tehran, Iran; the University of Kentucky; the University of Agriculture in Krakw, Poland; the Hong Kong Jockey Club; the Equine Veterinary Medical Center (a Qatar Foundation member) in Doha, Qatar; and the University of Veterinary Medicine Vienna, Austria.

The study can be read in full at: https://www.nature.com/articles/s41598-020-66232-1

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WCM-Q Research Helps Shed Light on Genetic Heritage of the Arabian Horse - Al-Bawaba

Black men in the United States are known to suffer disproportionately from prostate cancer, but few studies have investigated whether genetic differences in prostate tumors could have anything to do with these health disparities.

Now, in the largest study of its kind to date, researchers from Boston University School of Medicine (BUSM), UC San Francisco (UCSF), and Northwestern University have identified genes that are more frequently altered in prostate tumors from men of African ancestry compared to other racial groups, though the reasons for these differences is not known, the authors say. None of the individual tumor genetic differences that were identified are likely to explain significant differences in health outcomes or to prevent Black Americans from benefiting from a new generation of precision prostate cancer therapies, the authors say, as long as the therapies are applied equitably.

The newly identified gene variants could potentially lead to precision prostate cancer therapies specifically focused on men of African ancestry, and will inform broader efforts by the National Cancer Institutes RESPOND study to link gene variants to health outcomes in an even larger cohort of Black patients nationwide.

Despite declines in mortality related to cancer in the U.S., disparities by race have persisted. One in every six Black Americans will be diagnosed with prostate cancer in their lifetime, and these men are twice as likely to die from the disease as men of other races. But it is not yet clear to researchers whether differences in prostate cancer genetics contribute to these health disparities in addition to the social and environmental inequities known to drive poorer health outcomes across the board.

To date, studies trying to figure out what genes are commonly mutated in prostate cancers often have had very few samples from racial/ethnic minority groups despite the greater burden of prostate cancer in these populations. In May, the FDA approved a class of drugs known as PARP inhibitors as a therapy for men with prostate cancers driven by specific genetic mutations, but it is not known how prevalent these mutations are in people of African descent. As more genetic health studies are performed in minority populations, it has become clear that other genetically targeted therapies that have been developed based on studies of patients of European descent are at times much less effective, and in some cases cause dangerous side-effects, in other racial and ethnic groups.

In the new study, published July 10, 2020 in Clinical Cancer Research, a journal of the American Association for Cancer Research, the research team set out to better understand differences in the mutations driving prostate cancer tumors in men with African versus European ancestry, and whether any such differences could influence disease outcomes or the effectiveness of PARP inhibitors or other targeted therapies.

The researchers collected and analyzed DNA sequencing data from previously published studies and from a commercial molecular diagnostics company. In total, they examined mutational patterns in prostate cancers from more than 600 Black men, representing the largest such study of this population to date.

The team found that the frequency of mutations in DNA repair genes and other genes that are targets of current therapeutics are similar between the two groups, suggesting that at least these classes of current precision prostate cancer therapies should be beneficial in people of both African and European ancestry, according to corresponding author Franklin Huang, MD, PhD, an assistant professor in UCSFs Division of Hematology/Oncology and member of the UCSF Helen Diller Family Comprehensive Cancer Center, UCSF Institute for Human Genetics, and UCSF Bakar Computational Health Sciences Institute.

While the researchers found no significant differences in frequencies of mutations in genes important for current prostate cancer therapies, they did identify other genes, such as ZFXH3, MYC, and ETV3, that were more frequently mutated in prostate cancers from Black men.

"These results reinforce the idea that there can be biological differences in prostate cancers between different ancestral groups and that samples from Black Americans need to be included in future molecular studies to fully understand these differences," said co-corresponding author Joshua Campbell, PhD, assistant professor of medicine at BUSM.

The poorer health outcomes we see in Black men with prostate cancer are not easily explained by any of the distinct gene mutations we identified in prostate tumors from men of African ancestry. This highlights the need to examine the environmental and social inequities that are well known to influence health outcomes across the board, Huang added. On the other hand, our tumor genomic analysis also showed that current precision medicine approaches ought to be as effective in Black Americans as they have been for other groups if we can ensure that these drugs are applied equitably going forward."

Developing a comprehensive understanding of how tumor genomics and other biological factors interact with social and environmental inequities to drive poorer clinical outcomes for Black prostate cancer patients should be an important priority for the efforts to improve precision medicine for these patients, the researchers say.

These types of studies will remain important to understand when certain therapies may preferentially benefit Black patients, who continue to remain underrepresented in clinical trials, Campbell said.

In particular, the results will inform the efforts of the NCI-funded RESPOND Study. RESPOND provided funding for the new UCSF-BUSM-Northwestern study to guide its efforts to perform targeted gene sequencing in tumors from an even larger cohort of Black prostate cancer patients, said Huang, who leads RESPONDs tumor genetics studies based at UCSF. Through partnerships with Black communities across the country, RESPOND aims to recruit 10,000 Black prostate cancer patients in an effort to better understand the drivers of the diseases outsize burden among Black Americans.

"Previous studies have looked in isolation at different biological, social and environmental drivers ofwell-knownracial disparities in prostate cancer,Huangsaid. RESPOND is a nationwide effort to integrate all these components and ultimately identify specific steps that can be taken toeliminateprostate cancers unequal burden in Black communities.

Authors: The studys lead authors are Yusuke Koga of BUSM, Hanbing Song of UCSF, and Zachary Chalmers of Northwestern University. Additional authors are Elad Ziv of UCSF; Justin Newberg and Garrett M. Frampton of Foundation Medicine, in Cambridge; Eejung Kim and Daphnee Piou of the Broad Institute of MIT and Harvard; Jian Carrot-Zhang and Matthew Meyerson of the Broad Institute and Dana-Farber Cancer Institute; Paz Polak of Mt. Sinai School of Medicine in New York; and Sarki Abdulkadir of Northwestern University.

Funding: The study was supported by the U.S. Department of Defense (W81XWH-17-PCRP-HD); the U.S. National Institutes of Health (NIH) National Cancer Institute (NCI) (P20 CA233255-01, U19 CA214253); and the Prostate Cancer Foundation.

Disclosures: The authors declare no relevant conflicting financial interests.

About UCSF:The University of California, San Francisco (UCSF) is exclusively focused on the health sciences and is dedicated to promoting health worldwidethrough advanced biomedical research, graduate-level education in the life sciences and healthprofessions, and excellence in patient care. It includes UCSF Health, which comprises three top-ranked hospitals, as well as affiliations throughout the Bay Area.

About BUSM: Originally established in 1848 as the New England Female Medical College, and incorporated into Boston University in 1873, Boston University School of Medicine (BUSM) today is a leading academic medical center with an enrollment of more than 700 medical students and 950 students pursuing degrees in graduate medical sciences.BUSM faculty contribute to more than 950 active grants and contracts, with total anticipated awards valued at more than $693 million in amyloidosis, arthritis, cardiovascular disease, cancer, infectious diseases, pulmonary disease and dermatology, among other areas. The Schools teaching affiliates include Boston Medical Center, its primary teaching hospital, the Boston VA Healthcare System, Kaiser Permanente in northern California, as well as Boston HealthNet, a network of 15 community health centers. For more information, please visithttp://www.BUSM.bu.edu/busm/.

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Largest-Ever Study of Prostate Cancer Genomics in Black Men IDs Potential Targets for Precision Therapies - UCSF News Services

SAN FRANCISCO, July 15, 2020 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate QED Therapeutics announced today that the first child with achondroplasia has been dosed with the investigational medicine infigratinib, an orally available small molecule, that targets the overactivity of fibroblast growth factor receptor 3 (FGFR3) in the PROPEL 2 Phase 2 clinical trial. Achondroplasia is the most common cause of disproportionate short stature.

With preclinical evidence showing increased growth in the long bones, spine and cranial bones, including the base of the skull, there is the potential for infigratinib to help children with achondroplasia by decreasing the impact of serious medical complications, said Professor Ravi Savarirayan, M.B., B.S., M.D., Ph.D., clinical geneticist and group leader of skeletal biology and disease at Murdoch Childrens Research Institute in Australia and the lead investigator for the PROPEL 2 trial. Additionally, infigratinib is being studied as a once-daily dose taken orally, which is an important factor for administration of therapies to children with achondroplasia.

Achondroplasia is caused by an alteration in the FGFR3 gene, which causes the FGFR3 protein to be overly active.This interferes with skeletal development and can lead to disturbances in bone growth.Infigratinib is an oral investigational medicine that is designed to reduce the activity of FGFR3.

The start of this clinical trial is the culmination of more than two years of work first to secure rights to develop infigratinib, which we pursued following the publication of data relating to its potential in achondroplasia, and then to establish preclinical data showing the efficacy and safety of very low doses of the molecule, said Michael Henderson, M.D., CEO of QED Therapeutics. Infigratinib illustrates the heart of what BridgeBio set out to do: leverage the highest quality science to identify and develop potential therapies that target genetically driven conditions at their source. Our hope is that a daily, oral dose of infigratinib, which directly targets FGFR3, can provide health benefits for children with achondroplasia.

The PROPEL 2 trial is a Phase 2 dose escalation and dose expansion trial and the first clinical trial to study infigratinib at low doses in children with achondroplasia. The goal of the study is to assess safety and measure changes from baseline in annualized height velocity and changes in other health factors. To be eligible for the trial, children must first complete a six-month period of assessment in the PROPEL prospective clinical assessment study.

About QED Therapeutics

QED Therapeutics, an affiliate of BridgeBio Pharma, is a biotechnology company focused on precision medicine for FGFR-driven diseases. Our lead investigational candidate is infigratinib (BGJ398), an orally administered, FGFR1-3 selective tyrosine kinase inhibitor that we are evaluating in clinical studies for the treatment of achondroplasia. We plan to conduct further clinical trials to evaluate the potential for infigratinib to treat patients with FGFR-driven tumor types and rare disorders. At much higher doses, infigratinib has shown activity that we believe to be meaningful in clinical measures, such as overall response rate, in patients with chemotherapy-refractory cholangiocarcinoma with FGFR2 fusions and advanced urothelial carcinoma with FGFR3 genomic alterations. QED intends to submit a New Drug Application (NDA) with the United States Food and Drug Administration (FDA) for second and later-line cholangiocarcinoma in 2020.

For more information on QED Therapeutics, please visit the companys website at http://www.qedtx.com.

About BridgeBio Pharma, Inc.

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBios pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, visit bridgebio.com.

BridgeBio Pharma Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as anticipates, believes, estimates, expects, intends, may, plans, projects, seeks, should, will, and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to the timing, expectations, plans, and potential success of QED Therapeutics Phase 2 PROPEL 2 clinical trial, the regulatory approval process, clinical trial designs, clinical development plans, clinical trial results, timing and completion of the PROPEL 2 and other clinical trials, clinical and therapeutic potential of infigratinib for the treatment of achondroplasia or other FGFR-driven diseases, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, QED Therapeutics ability to continue or complete its Phase 2 PROPEL 2 clinical trial, ongoing and planned clinical trials of infigratinib for the potential treatment of achondroplasia and other FGFR-driven, the availability of data from these trials, past data from preclinical and earlier clinical studies not being indicative of future data from clinical trials, its ability to advance infigratinib in clinical development according to its plans, and the timing of these events, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma, Inc.s most recent Quarterly Report on Form 10-Q and our other SEC filings. Moreover, QED Therapeutics operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

1. National Institutes of Health.https://ghr.nlm.nih.gov/condition/achondroplasia. Accessed June 23, 2020.

QED Contact:Carolyn HawleyCanale Communicationscarolyn@canalecomm.com858-354-3581

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BridgeBio Pharma's QED Therapeutics Doses First Child in Phase 2 Clinical Trial of the Investigational Medicine Infigratinib in Achondroplasia -...

WATERTOWN, Mass.--(BUSINESS WIRE)--Zikani Therapeutics, a company leveraging its unique TURBO-ZMTM platform to develop novel ribosome modulating agents (RMAs) for the treatment of rare, nonsense mutation-driven diseases, today announced the appointment of Vijay Modur, M.D., Ph.D., as its Chief Scientific and Medical Officer to lead the company in advancing its ribosome-modulating scientific platform into the next stage of drug development.

Dr. Modur was previously Global Project Head in Rare Disease Clinical Development at Sanofi-Genzyme where he oversaw key functions responsible for advancing venglustat for the treatment of conditions caused by lysosomal dysfunction and other investigational therapies for several rare diseases, many with the potential for accelerated approval. Before joining Sanofi-Genzyme, Dr. Modur served as Chief Medical Officer and Vice President of Translational Research for HTG Molecular. Dr. Modur previously held clinical development leadership roles at Novartis and Merck.

Zikanis top priority is to advance its current nonsense mutation readthrough programs from lead optimization to candidate selection for first in human clinical trials following the positive, pre-clinical data demonstrated in multiple disease areas, said Sumit Aggarwal, President and CEO, Zikani Therapeutics. Were optimistic about the opportunity to impact diseases with limited or no treatment options including: APC mutant colon cancer, familial adenomatous polyposis (FAP), class 1 cystic fibrosis, and recessive dystrophic and junctional epidermolysis bullosa (RDEB and JEB), and Vijays experience and leadership will strengthen our capabilities to pursue this important work, added Aggarwal.

Zikani has focused its technology on where it holds the most promise and with the addition of Vijay is building a leadership team that will advance the company into the next phases of preclinical and clinical development, said Alan Walts, Ph.D., Executive Chairman of Zikanis Board of Directors.

Dr. Modur earned his MBBS at Karnatak University, Dharwad, India and his Ph.D. in Experimental Pathology at the University of Utah. He completed his residency at Washington University, St. Louis where he served as Chief Resident, Laboratory Medicine. Dr. Modur is board certified in Clinical Pathology.

The promise of impacting rare diseases through such novel technology that builds upon the science of modulating protein translation is compelling, said Modur. The TURBO-ZMTM technology platform and the proof of cellular concept demonstrated across multiple disease states provides a compelling approach to treating rare diseases. Im excited to join the team and look forward to making an impact on behalf of patients, added Modur.

Ribosomal RNAs form the translation machinery that generates function proteins from genetic sequences. Ribosome modulation provides a therapeutic approach to addressing a number of diseases, but the development of disease-specific ribosome modulators has been a challenge.

About Zikani Therapeutics

Zikani Therapeutics is an emerging leader in the science of ribosome modulation, leveraging its innovative TURBO-ZMTM chemistry technology platform to develop novel ribosome modulating agents (RMAs) as therapeutics for people with limited treatment options. Zikanis TURBO-ZMTM platform allows rapid synthesis of novel compounds that can be optimized to modulate the ribosome in a disease specific manner. As the company evolves its focus from early-stage to clinical-stage research, Zikani is actively moving into pre-clinical development to target select rare diseases including inherited diseases and cancers caused by nonsense mutations. For more information, visit zikani.com.

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Zikani Therapeutics Appoints Chief Scientific and Medical Officer - Business Wire

Why RNA is the future of diagnostics

Weve come a long way from the early days of using medicinal leeches and mercury treatments as therapeutics. As described in the book Soonish, the path to precision medicine has been a long one, often lacking scientific rigour and sometimes, common sense.

Thankfully, on the diagnostics front, weve advanced further than posthumous autopsies and microscopic pathology developed in the nineteenth century. Today, molecular diagnostics have become commonplace in medicine. Looking into the future, how will the innovations of today shape the face of medicine ten or twenty years from now? We may see the biggest change occur in genomics, with a battle of the bases determining which biomolecule will reign supreme for biomarker discovery.

In one corner is DNA, or deoxyribonucleic acid, in the other corner is RNA, or ribonucleic acid. These two contenders are known for their respective and important roles within the central dogma of biology. And, while both are composed primarily of a sugar and base combination, major differences exist. First, the base pairs differ slightly since DNA uses the bases adenine, thymine, cytosine and guanine while RNA uses adenine, uracil, cytosine and guanine. Second, DNA most often exists as a double-stranded molecule, while RNA is usually found in its single-stranded form. RNAs bonus hydroxyl group results in decreased chemical stability relative to DNA. This, coupled with the pervading presence of RNases, results in a much shorter lifetime for RNA, and is a third defining difference between these two biomolecules.

Finally, while most organisms have only two copies of DNA per cell, even low abundance RNA transcripts often have hundreds of copies present per cell (or organism).

So, lets start off with a warm-up match: infectious diseases. RNAs ubiquitous nature affords an advantage here. Diagnostics which are developed to detect RNA species for infectious organisms could require less patient material or detect the infection earlier due to a stronger signal compared to their DNA counterparts. Further, as described by John Brunstein, if a transcript or set of transcripts is selected correctly, an RNA diagnostic could also provide a measurement of viability of the infectious agent. Looks like RNA has taken an early lead.

One more bout to get warmed up: autoimmune disease. Since the completion of the Human Genome Project and with the introduction of high-throughput sequencing, weve been able to collect massive amounts of DNA data from both healthy and diseased patients. These data sets have enabled us to determine genetic changes implicated in many diseases, including autoimmune diseases like rheumatoid arthritis (RA) and multiple sclerosis (MS). However, one challenge that remains is that DNA only reports on the risk or potential for developing one of these diseases. RNA, however, gets us one step closer to the downstream biology, and can greatly help disease diagnosis. By analysing RNA signals, its been reported that earlier, more accurate diagnosis can be achieved. This warm-up round goes to RNA, as well.

And now, for a championship match of great significance: Cancer.

This disease, or rather an assemblage of diseases, still represents the second most prevalent cause of death in the world. Despite immense improvements in understanding cancer biology and using this to develop new and impactful treatments, including targeted therapies, immunotherapies, cell therapies and cancer vaccines, we still see only a small percentage of patients benefiting with robust responses. Cancer, with its combination of hereditary, environmental and sometimes random origins, has required diagnostic approaches covering many fronts.

Round 1: genetic mutations For hereditary cancers, there are a number of mutations that have been determined to play a major role in the likelihood of developing the disease or the path of disease progression. In breast cancer, for example, these include BRCA1 and BRCA2, along with the less commonly discussed PTEN and p53 mutations.

However, for many patients, a genetic analysis delivers a number of mutations which are listed as Variants of Unknown Significance. These mutations are known to be different from a normal genome, but there is not sufficient clinical evidence to use the information to make medical decisions. However, when RNA data is layered on top of this genetic data, additional context may be used to determine whether these variants should be classified as pathogenic or benign. As described above, while were still looking at risk factors in this case, the additional clarity provided by RNA can make a big impact in determining whether a patient receives preventative surgery or more rigorous surveillance. Round 1 goes to RNA.

Round 2: gene fusions Understanding the mechanisms of cancer that impact progression and pathogenesis are imperative to improving treatment plans. Further, specific molecular events have also informed therapy selection, more commonly known as targeted therapies. One excellent example is in non-small cell lung cancer, where the EML4-ALK gene fusion, arising from chromosomal rearrangement, has become both a diagnostic marker and a target for ALK inhibitor therapies. In the diagnostic arena, fluorescence in situ hybridisation (FISH) of the two DNA sequences has been used to detect the presence of this gene fusion. However, there is significant genomic heterogeneity in these ALK rearrangements undetectable by FISH, which has been shown to be related to differences in kinase inhibitor treatment response rates. Use of RNA sequencing technologies, which look not only for expression of these fusions but also enable discovery and quantification of novel fusions, has enabled the field to move beyond the limited information provided by DNA diagnostics. RNA wins another point.

Round 3: immune response Undeniably, the most promising path to understanding and treating cancer is immuno-oncology. This approach, which harnesses the bodys immune system to fight the disease, has garnered much attention, resulting in the 2018 Nobel Prize in Medicine. While these therapies show enhanced and lasting responses, a number of challenges remain. DNA technologies, including tumour mutational burden (TMB), showed promise in predicting response to immune checkpoint inhibitors; however, the results in the clinic were disappointing. Understanding the dynamic immune composition at the site of the solid tumour is imperative, and new approaches using RNA models to achieve that are showing the promise that RNA will have in this space. It may be too early to call this round for RNA, but it is beginning to make a case.

Concluding thoughts

As it turns out, the battle of the bases was not really a fair fight. The advances in high-throughput sequencing and analysis that have bolstered our databases of DNA information have in parallel provided us with a plethora of RNA data to mine and interpret. Further contributing to this are enhanced tools for analysing chemically and enzymatically degraded RNA, removing a roadblock that previously existed. Now, instead of seeing RNAs lability as a weakness, its dynamic nature is now considered a massive advantage. We can measure the impact of therapy and disease progression in the bodys ever-changing RNA. Today, the field is focused on building and validating new tools using RNA to aid physicians in understanding what causes disease, when disease will manifest itself, how we can predict which therapies will benefit individual patients and when disease will recur or progress. From infectious diseases to oncology, and likely in areas we have not yet discovered, RNA diagnostics will certainly be a key chapter in the tome entitled precision medicine that so many of us in the industry are currently writing. And, if successful, may help us knockout some of these malevolent diseases.

Jarret Glasscock is a geneticist, computational biologist, and founder and chief executive of Cofactor Genomics

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Biomarkers and a battle of the bases - PharmaTimes

]]> Vaccines are the most effective way to end the Covid-19 pandemic. Credit: Shutterstock.

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

In the six months or so since the Covid-19 pandemic broke out in Wuhan, China, the pharma industry and university researchers from across the world have thrown themselves head-first into developing vaccines.

As of 7 July, there are 139 pre-clinical candidates and 21 clinical vaccines in development against SARS-CoV-2, the virus which causes Covid-19, according to the World Health Organization (WHO).

Of the 21 Covid-19 vaccines in clinical trials, lead candidates include Chinese biotech Sinovacs CoronaVac and the University of Oxford and AstraZenecas ChAdOx1-S both of these are in Phase III development.

Other promising candidates in Phase II trials include US-based Modernas mRNA-1273, which is being supported by the National Institute of Allergy and Infectious Diseases (NIAID), and BioNTech and Pfizers Project Lightspeed BNT162 vaccine programme.

The speed at which these companies and researchers have been able to move into and through clinical development is completely unprecedented; milestones normally achieved in years, if not decades, have been surpassed in only months.

Time is of the essence since immunity conferred through vaccines is the primary way that the world can control the spread of this deadly viral disease in the long-term, enabling the world to get back to normal.

Without taking away from this incredible achievement of science, which has been enabled by previously unseen partnerships across industry, academia and the public sector, there are already some concerns about how effective these speedily developed vaccines will be, particularly in conferring long-term immunity.

This worry has been expressed by Dr Anthony Fauci, director of NIAID and the US Governments chief health advisor; in an interview with JAMA cited by CNBC, Fauci stated that, based on previous experience with developing vaccines for other coronaviruses, its likely [the vaccine] isnt going to be a long duration of immunity.

This is also linked to that fact that most of the candidates for which there is publicly available data are focusing on inducing neutralising antibodies against SARS-CoV-2s spike protein, as noted by Nature. As a result, STAT reports it is possible that although vaccines will reduce the risk of someone contracting the disease or experiencing severe symptoms if someone is infected, they will not prevent infection entirely.

Dr Patrick Soon-Shiong, CEO and chairman of sister biotechs ImmunityBio and NantKwest, noticed this problem and his team began looking at developing a so-called second-generation Covid-19 vaccine that overcomes the short-term duration challenge of the first set of vaccines.

To this end, ImmunityBio and NantKwest are working on a Covid-19 vaccine that does not only focus on the spike protein; their vaccine also works against SARS-CoV-2s nucleocapsid.

It is our fervent belief that you cant just kill this virus from the outside in, youve got to kill it from the inside out, explains Soon-Shiong. We have created the unique construct of both nucleocapsid and spike, so that you have not just antibody, but also a memory T cell [response].

The nucleocapsid is viewed as a particularly strong stimulator of cellular immunity and improves the generation of memory T cells, explains NantKwest director of cell-mediated therapies in virology Jeffrey Safrit. T cell mediated immunity, he adds, has been shown to last significantly longer than antibody-mediated immunity.

Allowing a broader immune response involving antibody and cell-mediated immunity that is directed against more than one target lessens the ability of the virus to escape, concludes Safrit.

Another benefit of focusing on both the spike protein and nucleocapsid is linked to potential future mutations of the SARS-CoV-2 virus. Focusing on spike alone will limit vaccine effectiveness should spike mutate to avoid the immune response, explains Safrit.

Whereas the nucleocapsid gene is highly conserved among coronaviruses, meaning it mutates much less often if at all, therefore, Safrit notes, inclusion of nucleocapsid makes our approach a more universal Covid vaccine, that could even be useful against related future, yet unseen SARS infections.

Soon-Shiong was also concerned about how many leading Covid-19 candidates, such as the University of Oxford/AstraZeneca project, are relying on the adenovirus approach.

Current adenovirus vaccines are based on the first generation of this vector [Ad5] that generate significant anti-vector immunity when administered which can dampen or the even negate the immune response [conferred by the vaccine] to the Covid-19 spike protein and nucleocapsid, explains Safrit. This situation is worsened when someone has been previously exposed to an adenovirus, such as that which causes the common cold.

To overcome this challenge, NantKwest and ImmunityBio have developed a second-generation Ad5 platform. It has demonstrated safety in Phase I and II studies in cancer patients. The companies also carried out a successful proof-of-concept during the 2009 H1N1 influenza pandemic.

The platform has additional deletions of adenovirus genes that generate little to no vector-specific immunity and focuses the immune response on the inserted genes of interest, SARS-CoV-2 spike protein and nucleocapsid, says Safrit.

Because of this, the second-generation Ad5 also overcomes any adenovirus pre-existing immunity, allowing a more effective first vaccination and multiple subsequent booster vaccinations without reduced immunogenicity, he explains.

Soon-Shiong is clear that long-term duration of immunity challenges will be solved by second-generation vaccines such as Immunity Bio and NantKwests candidate.

Their vaccine, which is part of the US Governments Operation Warp Speed, is ready to launch into Phase I clinical trials this summer with early safety and preliminary efficacy results expected before the end of the year.

However, there is a need for further innovations so that the vaccine can be administered without injections to overcome access challenges, particularly in developing countries.

If we could achieve an oral form, which is not unrealistic, that would be the third generation Covid-19 vaccine, Soon-Shiong notes.

This is an avenue that ImmunityBio and NantKwest are pursuing for their Covid-19 vaccine candidate, but it is currently early in development, according to Soon-Shiong. Their oral capsule approach would avoid significant cold chain requirements of all current vaccine approaches, Safrit states.

It would also introduce the prospect of conferring mucosal immunity, generation [of] which could significantly improve the ability of the vaccine to protect against infection and disease.

Other companies slightly further along with their oral vaccines include the UKs Stabilitech and US-based Vaxart.

Stabilitechs OraPro-COVID-19, which is developed using its proprietary OraPro technology platform, aims to confer both mucosal and systemic immunity to patients in a thermally stable way. Clinical trials for this vaccine are expected to begin in August this year.

The company has recently completed a fundraising round and signed a manufacturing agreement with BioCell Corporation in New Zealand. Talking about this news, Stabilitech chairman Wayne Channon stated: It brings us closer to providing a vaccine that gives both mucosal and systemic immunity to Covid-19.

This will target the virus exactly where we catch it: in the mucous membranes of the mouth, nose, throat and even the eyes.

By taking this approach, we believe OraPro-COVID-19 could prove to provide the fastest route to widespread global immunity, turning the tide on the pandemic.

Vaxarts oral vaccine approach relies on its VAAST delivery platform. It delivers two payloads to cells in the small bowel epithelium; the second of the payloads acts like an adjuvant to further activate the immune system against the selected viral antigen.

Like ImmunityBio and NantKwests candidate, Vaxarts oral approach is one of the projects chosen to be part of the US Governments Operation Warp Speed. Following on from positive pre-clinical results in April, Vaxarts candidate is on track to move into clinical studies this summer.

Talking about this announcement, Vaxart CEO Andrei Floroiu said: We are very pleased to be one of the few companies selected by Operation Warp Speed, and that ours is the only oral vaccine being evaluated.

SARS-CoV-2, the coronavirus that causes COVID-19, is primarily transmitted by viral particles that enter through the mucosa nose, mouth or eyes strongly suggesting that mucosal immunity could serve as the first line of defence.

In addition, our vaccine is a room temperature-stable tablet, an enormous logistical advantage in large vaccination campaigns.

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Gene therapy innovations: Sarepta and Codiak partner on exosomes - Pharmaceutical Technology

The agreement allows First Genetics to bring its F-Genetics NGS system and in vitro diagnostic assays for reproductive health testing to Russian labs

Thermo Fisher Scientificand First Genetics JCS, a manufacturer of diagnostic equipment and IVD kits intended to advance molecular genetic technologies into clinical practice, today announced a strategic partnership focused on commercializing next-generation sequencing (NGS)based diagnostics inRussia. The agreement enables First Genetics to market its F-Genetics NGS System and IVD assays to Russian labs for reproductive health testing and cancer diagnostics. The F-Genetics System is based on Thermo Fisher'sIon GeneStudio S5 System.

First Genetics has already received its first registration certificate for an IVD kit by Roszdravnadzor, the Russian medical device regulatory agency. The company's Reproline kit is now approved for detecting chromosomal abnormalities in embryos on the F-Genetics NGS System prior to IVF implantation. While IVF is now government-funded as part ofRussia'spolicy to help boost the national birth rate, preimplantation genetic testing for aneuploidies (PGT-A) had not previously been certified inRussia. The certification now enables Russian prenatal centers, which have doubled in number over the last decade, to leverage PGT-A.

"High-throughput technologies for genetic analysis are the future of molecular diagnostics," saidNikolai Kuznetsov, CEO, First Genetics. "Today we have expanded applications of NGS into reproductive medicine inRussia, and in the future, we intend to offer clinically certified solutions for other clinical applications, such as oncology testing. This is our contribution to the development of clinical diagnostics inRussia."

Thermo Fisher has entered into a number of agreements with assay developers like First Genetics to expand the use of NGS-based molecular diagnostics in the clinic and to advance precision medicine globally. Similar partnerships with other organizations have led to approvals by regulatory agencies inChina,the United StatesandEurope.

"It is our mission to help bring molecular testing to all parts of the world so those who need it most can benefit from the genetic information next-generation sequencing provides," saidYan Zhang, general manager, reproductive health at Thermo Fisher. "We are pleased that couples inRussianow have access to First Genetics' solutions to assist in family planning and reduce the emotional burdens they face while undergoing fertility treatment."

Want more of the latest science news straight to your inbox?Become a SelectScience member for free today>>

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Industry News: Thermo Fisher Scientific and First Genetics JCS partner to develop next-generation sequencing-based - SelectScience

For 25 years, researchers have explored an idea that, by regulating certain genes, they could treat one of the world's most debilitating neurological diseases. That work has led to encouraging data, with the latest coming Wednesday from two studies published in the New England Journal of Medicine.

"It's a really exciting time for the field," said Orla Hardiman, clinical professor of neurology at Trinity College in Dublin and co-author to a NEJM editorial published alongside the studies.

Previously, scientists discovered the risk of developing amyotrophic lateral sclerosis,also known as ALS or Lou Gehrig's disease, is higher if a select group of genes mutate. The newly published studies each tested an experimental drug meant to silence one such gene, called SOD1, that encodes an eponymous protein. While both studies were designed to evaluate safety, researchers also looked at protein levels to see if the drugs were working as intended.

One of these drugs uses a virus to deliver a small strip of genetic material into patients' spinal areas. In theory, the material would block the gene from making its protein, but results from two patients showed that neither had a substantial change in protein levels in their cerebrospinal fluid.

However, after one of the patients died, an autopsy showed SOD1 protein levels in his spinal cord tissue were lower than in untreated patients with the same form of ALS. The study investigators concluded that additional trials with a larger number of patients are necessary to better understand the drug's effects.

The other study had more clearly positive results. It tested four doses of Biogen's tofersen against placebo, and found lower SOD1 protein levels in the cerebrospinal fluid of patients who received the drug. Compared to those in the placebo group, protein concentrations were about 20 to 25 percentage points lower for patients given the two middle tofersen doses and 33 percentage points lower for patients on the highest, 100 mg dose.

Biogen, which announced summary data from the trial last year, has since moved the high dose into a larger, efficacy-focused trial that aims to recruit around 100 patients. Enrollment has been "reasonable," albeit with slight delays due to the coronavirus pandemic, according to Toby Ferguson, head of the company's neuromuscular development unit.

Though tofersen will likely need positive late-stage results to support an approval, the currently available data offer a confidence boost for Biogen. Like other ALS drug hunters, the biotech has hit setbacks the most damaging of which came in 2013 when its small molecule medicine dexpramipexole failed a Phase 3 study.

"It's not fully shown to work yet, but at least the biology seems to be going in the right way," Ferguson said of tofersen. "It fundamentally says to me that if we pick the right targets, ALS can be a treatable disease. And we need to push forward both with genetic targets and appropriate targets for the broader population."

The tofersen study may also fuel optimism in the broader ALS research community. While two drugs are approved for ALS, there remains an urgent demand for more treatments. Most patients live just three to five years after they're diagnosed, according to the Centers for Disease Control and Prevention.

Following decades of research, genetic medicine has, in recent years, proven itself to be a valuable weapon against hard-to-treat neurological conditions. In 2016, for example, Biogen's drug Spinraza became the first ever approved treatment for spinal muscular atrophy, a rare and often life-threatening condition that impairs muscle growth. Spinraza, like tofersen, is a type of gene-silencing medicine called antisense oligonucleotides, or ASOs.

Sarepta Therapeutics also has two ASO products approved for a different muscular disorder, and research on other gene-based treatments is advancing for difficult neurological diseases like Huntington's and Rett syndrome.

In ALS, several companies are working on genetic medicines. Novartis and Voyager Therapeutics each have plans for a SOD1-targeting ALS gene therapy, while MeiraGTx and the partners Pfizer and Sangamo Therapeutics are developing gene therapies not specific to SOD1.

With tofersen, though, Biogen holds a leading and potentially tone-setting position.

As the drug progresses through late-stage testing, Hardiman said it would be "fantastic" if the drug demonstrates not just reductions in SOD1 protein levels, but also the ability to slow or stabilize the disease. Biogen's smaller study hinted that tofersen's effect on SOD1 protein levels might translate to slower functional declines, but the data aren't proof it actually does.

"If we can show that gene-silencing in SOD1 is effective, it opens the way for other gene-silencing approaches in other genetic forms of ALS," she said, pointing to several other mutations associated with ALS.

"We are in a new era now where we have a much better understanding of genomic regulation, and we're getting to a place where it's really possible to modulate these pathways in a way that's genuinely therapeutic," Hardiman added.

ALS drug research also extends beyond genes, since estimates hold that only 5% to 10% of cases are inherited and, within that fraction, SOD1 mutations account for 15% to 20% of cases.

Currently, the Sean M. Healey & AMG Center for ALS Research is running a first-of-its-kind platform trial to test five experimental therapies, including ones from Biohaven Pharmaceutical and Cambridge, Massachusetts-based Ra Pharmaceuticals, now owned by Belgium's UCB.

Privately held Amylyx Pharmaceuticals, meanwhile, is working separately with the Healey Center. The company said in December its experimental treatment slowed ALS progression in a mid-stage study, although no actual data was released.

Alexion Pharmaceuticals, a large rare disease drugmaker, also recently began exploring whether one of its approved therapies could work in ALS too.

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In the hunt for ALS treatments, researchers find promise in silencing genes - BioPharma Dive

BETHESDA, Md., July 8, 2020 /PRNewswire/ --The American College of Medical Genetics and Genomics (ACMG) announced today that the 2019 Journal Impact Factors, published by Clarivate Analytics in the latest edition of Journal Citation Reports, calculated an impact factor of 8.904 for ACMG's official journal, Genetics in Medicine (GIM). This is the second highest Impact Factor in the journal's history and ranks GIM 13th of 177 titles in the Genetics & Heredity category.

The Impact Factor is an objective measure of the world's leading journals, based on articles' cited references and is oft considered a measure of a journal's impact, overall successful performance and relevance to its field. The most highly cited article in GIM in 2019 was "Recommendations for Reporting of Secondary Findings in Clinical Exome and Genome Sequencing, 2016 Update (ACMG SF v2.0): A Policy Statement of the American College of Medical Genetics and Genomics."

"GIM's editors and editorial staff are delighted that our Impact Factor has increased from last year. This improvement in the Impact Factor once again demonstrates that the journal remains one of the most widely read and cited journals publishing clinically relevant research in the life sciences," said GIM's Editor-in-Chief Robert D. Steiner, MD, FAAP, FACMG."We are most thankful to the peer reviewers who put in countless hours to help maintain the outstanding quality of articles and the authors who trust us to disseminate their groundbreaking scholarly work. The Impact Factor is one of a number of metrics used to evaluate journals, and a journal should not be evaluated solely on that one metric. Genetics in Medicine'scontinued success and relevance is also reflected in our very high overall downloads and reads as well as a prominent social media presence."

ACMG CEO Maximilian Muenke, MD, FACMG said, "As the CEO of the ACMG, I am extremely proud of 'our' journal. As a physician-scientist who before joining ACMG worked in academic settings where publishing in high-impact factor journals was the goal, I am well aware of the importance of this metric. My congratulations and gratitude on increasing GIM's impact factor go to Bob Steiner, Jan Higgins, the GIM staff and the entire editorial team to make this success happen!"

Genetics in Medicineis published by Springer Nature. The journal, published since 1998, is supported by an expert board of editors representing all facets of genetic and genomic medicine, including biochemical and molecular genetics, cytogenetics, and the application of genetics and genomics to other medical specialties such as oncology, cardiology, neurology, pediatrics, ophthalmology and maternal-fetal medicine.

About the American College of Medical Genetics and Genomics (ACMG) and ACMG Foundation

Founded in 1991, the American College of Medical Genetics and Genomics (ACMG) is the only nationally recognized medical professional organization solely dedicated to improving health through the practice of medical genetics and genomics, and the only medical specialty society in the US that represents the full spectrum of medical genetics disciplines in a single organization. The ACMG is the largest membership organization specifically for medical geneticists, providing education, resources and a voice for more than 2,300 clinical and laboratory geneticists, genetic counselors and other healthcare professionals, nearly 80% of whom are board certified in the medical genetics specialties. ACMG's mission is to improve health through the clinical and laboratory practice of medical genetics as well as through advocacy, education and clinical research, and to guide the safe and effective integration of genetics and genomics into all of medicine and healthcare, resulting in improved personal and public health. Four overarching strategies guide ACMG's work: 1) to reinforce and expand ACMG's position as the leader and prominent authority in the field of medical genetics and genomics, including clinical research, while educating the medical community on the significant role that genetics and genomics will continue to play in understanding, preventing, treating and curing disease; 2) to secure and expand the professional workforce for medical genetics and genomics; 3) to advocate for the specialty; and 4) to provide best-in-class education to members and nonmembers. Genetics in Medicine, published monthly, is the official ACMG journal. ACMG's website (www.acmg.net) offers resources including policy statements, practice guidelines, educational programs and a 'Find a Genetic Service' tool. The educational and public health programs of the ACMG are dependent upon charitable gifts from corporations, foundations and individuals through the ACMG Foundation for Genetic and Genomic Medicine.

Kathy Moran, MBAkmoran@acmg.net

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ACMG's Genetics in Medicine Journal Receives Impact Factor of 8.904 for 2019--Journal is Ranked 13th of 177 Journals in Genetics & Heredity -...

July 08, 2020 -In the largest study of its kind, researchers discovered hundreds of novel genetic variants linked to type 2 diabetes, potentially improving care for millions living with this disease.

A team from the Perelman School of Medicine at the University of Pennsylvania and the Veterans Health Administrations (VHA) Corporal Michael J. Crescenz Veterans Affairs Medical Center (CMCVAMC) examined the genes of more than 200,000 people around the world with type 2 diabetes.

In addition to uncovering new genetic variants linked to the condition, researchers identified gene variants that vary by ethnicity, as well as variants tied to conditions related to type 2 diabetes like coronary heart disease and chronic kidney disease.

The group used data from the worlds largest biobank, the Million Veteran Program (MVP) in the VHA, as well as data from the DIAGRAM Consortium, the UK Biobank, the Penn Medicine Biobank, and Biobank Japan. Researchers analyzed a study population of 1.4 million people around the world, of whom almost 230,000 had type 2 diabetes.

The team then broke down the genetic makeup of those hundreds of thousands with type 2 diabetes and found 558 independent genetic variants that are differentially distributed between people with and without type 2 diabetes. Twenty-one of these variants were specific to European ancestry while seven were specific to African American ancestry. Of the 558 variants found, 286 had never been discovered.

Researchers set out to discover if certain genetic variants among this group of people could be linked to specific type 2 diabetes-related conditions.

Ultimately, three were linked to coronary heart disease, two to acute ischemic stroke, four to retinopathy, two to chronic kidney disease, and one to neuropathy, saidMarijana Vujkovic, PhD, a biostatistician at both the Perelman School of Medicine at the University of Pennsylvania, VHAs CMCVAMC and a co-leader for the VHAs national MVP Cardiometabolic Working Group.

Building on this research, the scientific community can assess which of the surrounding genes nearby the identified genetic variants is likely to be the causal gene that alters the risk of type-2 diabetes, and that could lead to early interventions to limit controllable risks of developing the condition.

While the researchers found many genetic variants in people with type 2 diabetes, no one variant was labeled as the worst or most dangerous.

However, just like heart disease, schizophrenia, or obesity, it is the accumulation of a large number of these variants that can add up to a considerable increase in risk, said co-senior authorBenjamin F. Voight, PhD, an associate professor of Systems Pharmacology and Translational Therapeutics at Penn, and a co-leader for the VHAs national MVP Cardiometabolic Working Group.

We hope this study can not only help find that subset of patients with substantial risk, but also to motivate new, future studies for treatments based on these findings.

Knowing more about the genetic variants linked to type 2 diabetes could help identify potential therapeutic targets for type 2 diabetes. Researchers also noted that this information could help guide treatment plans for people with the disease who may be susceptible to specific diabetes complications.

Going forward, the researchers plan to conduct a long-term examination of how genetics influence disease progression among patients with type 2 diabetes and associated metabolic disorders. The group is also leveraging the list of newly-discovered genes to investigate medication interactions.

Knowing the genetic susceptibility for diabetes complications in a patient already diagnosed with type-2 diabetes, for example through a cumulative genetic risk score, could help guide that patients care, said co-senior-authorKyong-Mi Chang, MD, a professor of Medicine at Penn, Associate Chief of Staff for Research at VHAs CMCVAMC and the Co-PI for the VHAs MVP Merit Award that supported this work.

As clinicians, we hope that these findings can ultimately be applied to improve the health outcomes for our patients including veterans.

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Researchers Discover Genetic Variants Linked to Type 2 Diabetes - HealthITAnalytics.com

NEW YORK, July 14,2020 /PRNewswire/ -- Alliance For Lupus Research(LRA) is pleased to announce the first recipients of the inaugural LRA-BMS Accelerator Award*, a collaborative project with sponsoring partner Bristol-Myers Squibb. The Award provides a collective total of $3,000,000 to support nine cutting-edge lupus research projects over two years that focus on understanding the underlying causes of systemic and cutaneous lupus, unraveling its complexity, and identifying novel biomarkers.

Nine recipients were selected from a large volume of promising applications from throughout the country and abroad. Several researchers are working to understand how the immune system overreacts, while three are testing new markers in the blood and urine that may be used to better diagnose and monitor the disease. Other projects focus on identifying the genetic causes of lupus, and one is looking at how to improve the evaluation of clinical trial results.

LRA Chief Scientific Officer Dr. Teodora Staeva commented, "The Lupus Research Alliance is delighted to be partnering with pharmaceutical leader Bristol Myers Squibb to support critical fundamental research that could lead to much-needed new therapies, biomarkers, patient-stratification approaches and outcome measures for lupus."

Peter Schafer, Executive Director, Translational Medicine at BMS, commented, "We're excited to work with the Lupus Research Alliance on this initiative, as there's a significant unmet need for people living with lupus. We believe the LRA-BMS Accelerator Award will advance critical research that will help us better understand this devastating disease."

LRA-BMS Award Recipients

Characterizing the Immune Response to Lupus

Joel Guthridge, PhD, Oklahoma Medical Research FoundationPredicting treatment responses in patients with systemic lupusDr. Guthridge's study will use emerging technology to understand the immune response of patients who respond positively to treatment with abatacept. Results of this study may help predict which patients are most likely to benefit from different lupus treatments.

Victoria Werth, MD, University of PennsylvaniaPredicting treatment responses in patients with cutaneous lupusDr. Werth's study will identify key immune cells that correlate with successful treatment, which will allow clinicians to predict which patients are likely to respond to the lupus drug hydroxychloroquine and other antimalarials before starting treatment.

J. Michelle Kahlenberg, MD, PhD, University of MichiganComparing the immune response in patients with systemic and cutaneous lupusDr. Kahlenberg's research will measure the immune response in the blood and skin samples from people with either systemic lupus erythematosus (SLE) or cutaneous lupus erythematosus (CLE) to better understand what causes each and how best to diagnose and treat each type.

Evaluating Lupus Biomarkers

Ilana Brito, PhD, Cornell UniversityLinking gut bacteria to lupusDr. Brito's research is looking at the specific effects of bacteria in the gut on the immune system of lupus patients. This research will help identify new markers of lupus that could serve as targets for treatment and possibly diagnosis.

Chandra Mohan, MD, PhD, University of HoustonMonitoring lupus nephritis in a less invasive wayDr. Mohan's research has identified and will test the effectiveness of markers in the urine of lupus patients to diagnose lupus nephritis and monitor its treatment. This approach may reduce the need for invasive surgical kidney biopsies.

Searching for the Genetic Causes of Lupus

Marta Alarcn-Riquelme, MD, PhD, Fundacin Pblica Andaluza Progreso y SaludDetermining the genetic basis of the lupus immune responseDr. Alarcon-Riquelme is looking to find the specific genes associated with differences in how the immune system functions that distinguish groups of lupus patients. Her results may help determine optimal therapies for each group of patients.

Patrick Gaffney, MD, Oklahoma Medical Research FoundationFinding race-specific predictors of lupus Dr. Gaffney's study aims to uncover how race and ethnicity are linked to differences in the type of organ damage caused by lupus and toidentify new treatment targets.

Vivian K Kawai, MD, Vanderbilt University Medical CenterIdentifying genes controlling lupus and its severityDr. Kawai is using a novel approach of combining data from different sources to determine the genetic risk factors for developing severe lupus.

Improving Clinical Trials

Kenneth C Kalunian, MD, University of California, San DiegoChoosing better endpoints for clinical trialsDr. Kalunian will develop a comprehensive tool for researchers to best understand and evaluate the effectiveness of potential therapies in clinical trials.

About the LRA-BMS Accelerator Award

The LRA-BMS Accelerator Award was created to fund cutting-edge research projects that focus on understanding the underlying mechanisms of disease, addressing lupus heterogeneity, and identifying novel biomarkers. The initiative aims to stimulate lupus research innovation, accelerate investigation on human lupus rather than relevant model organisms, and realize synergies between academia and industry. A collaboration with the pharmaceutical company Bristol Myers Squibb, the Award provides $300,000 to each awardee over two years. Close oversight is provided by a Joint Steering Committee with equal representation from LRA and BMS. The company will have first right to negotiate with the principal investigators' institutions for any intellectual property arising from the projects.

About Lupus

Lupus is a chronic, complex autoimmune disease that affects millions of people worldwide. More than 90 percent of people with lupus are women; lupus most often strikes during the childbearing years of 15-45. African Americans, Latinx, Asians and Native Americans are two to three times at greater risk than Caucasians. In lupus, the immune system, which is designed to protect against infection, creates antibodies that can attack any part of the body including the kidneys, brain, heart, lungs, blood, skin, and joints.

About Lupus Research Alliance

TheLupus Research Allianceaims to transform treatment while advancing toward a cure by funding the most innovative lupus research in the world. The organization's stringent peer review grant process fosters diverse scientific talent who are driving discovery toward better diagnostics, improved treatments and ultimately a cure for lupus.Because the Lupus Research Alliance's Board of Directors fund all administrative and fundraising costs, 100 percent of all donations goes to support lupus research programs.

*Formerly the LRA-Celgene Accelerator Award.

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Newswise The death of a twin, especially earlier in life, can increase the risk of their surviving twin being diagnosed with a psychiatric disorder, finds a new study published today in eLife.

Losing a loved one is always difficult but losing a twin may be particularly so. By virtue of being the same age, twins share many common experiences and may have strong emotional bonds. The new study suggests those who lose a co-twin may require extra support in both the short and longer term.

"Losing a co-twin by death may be a particularly devastating life stressor with considerable health implications for surviving twins, yet there have been few studies on this type of bereavement," says lead author Huan Song, a senior researcher at West China Hospital, Sichuan University, China, and also at the University of Iceland and Karolinska Institute, Sweden.

Using the Swedish health registers and the Swedish Twin Registry, Song and colleagues identified all Swedish twins who experienced the death of a co-twin between 1973 and 2013. They then compared the rates of psychiatric diagnoses in these bereaved twins with their non-twin siblings, and with 22,640 twins whose co-twin was still alive.

"We showed that the risk of being diagnosed with a psychiatric disorder increased by 55% to 65% after the death of a co-twin," Song says. This risk was highest in cases where a co-twin had died during childhood or young adulthood.

Surviving twins were most likely to receive a new psychiatric diagnosis in the first month after the death, when their risk of such a diagnosis was sevenfold higher than non-bereaved twins. But they continued to have a higher risk for more than 10 years after the loss.

The findings also revealed that the risk of being diagnosed with a psychiatric disorder after a co-twin's death was particularly high for identical twins, who share all the same genes. These individuals had about a 2.5-times higher risk compared to their non-twin siblings. Surviving fraternal twins, who are as genetically similar to their twin as their non-twin siblings, had about a 30% higher risk of a psychiatric diagnosis after the death of their twin than their non-twin siblings.

Senior author Unnur Valdimarsdttir, Professor of Epidemiology at the University of Iceland explains that because of their genetic similarities and shared experiences, twins often develop a sense of shared identity, which may compound their grief after the loss of their co-twin.

"Our results suggest that both genetic similarity and early-life attachment may contribute to the subsequent risk of psychiatric disorders among surviving twins after the death of their co-twin," Valdimarsdttir concludes.

###

Reference

The paper 'Risk of psychiatric disorders among the surviving twins after a co-twin loss' can be freely accessed online at https://doi/org/10.7554/eLife.56860. Contents, including text, figures and data, are free to reuse under a CC BY 4.0 license.

AbouteLife

eLifeis a non-profit organisation created by funders and led by researchers. Our mission is to accelerate discovery by operating a platform for research communication that encourages and recognises the most responsible behaviours. We work across three major areas: publishing, technology and research culture. We aim to publish work of the highest standards and importance in all areas of biology and medicine, including Epidemiology and Global Health, while exploring creative new ways to improve how research is assessed and published. We also invest in open-source technology innovation to modernise the infrastructure for science publishing and improve online tools for sharing, using and interacting with new results.eLifereceives financial support and strategic guidance from the Howard Hughes Medical Institute, the Knut and Alice Wallenberg Foundation, the Max Planck Society and Wellcome. Learn more athttps://elifesciences.org/about.

To read the latest Epidemiology and Global Health research published ineLife, visithttps://elifesciences.org/subjects/epidemiology-global-health.

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Loss of a co-twin linked to heightened psychiatric risk - Newswise

A large prostate cancer genomics study suggests African-American men, who are disproportionately affected by the cancer, should also benefit from current therapies that target specific genetic mutations causing the disease.

However, the researchersbased at Boston University School of Medicine, University of California San Francisco, and Northwestern Universitydid discover some genetic differences between the cancers from European Americans and African Americans, which they believe warrants further investigation.

African-American men have a 15% chance of developing prostate cancer compared with a 10% chance for European-American men. They are more likely to develop aggressive disease and are twice as likely to die from the cancer than White men. It is possible that this inequality may be due to socio-economic status and healthcare disparities, but genetic differences in cancer mutational status could also be responsible.

In what they claim is the largest such study to date, the researchers studied mutations linked to prostate cancer in 250 African American and 611 European American men from four publicly available datasets. They then compared these to prostate cancer mutations found in 436 African American and 3018 European American men with both localized and metastatic prostate cancer who contributed samples to the Foundation Medicine commercial cohort.

As reported in the journal Clinical Cancer Research, the team found no notable differences in genetic variation that would impact the efficacy of current therapies for prostate cancer, such as treatment with PARP inhibitors targeting DNA repair genes, between the two groups of men.

However, they did find some genetic differences between the two groups. For example, two genes linked to prostate cancer suppression, ETV3 and ZFHX3, were more often mutated in African-American men with prostate cancer than in European Americans. The MYC gene, which is often overexpressed in cancers, was also more often amplified in African-American men with metastatic prostate cancer.

These results reinforce the idea that there can be biological differences in prostate cancers between different ancestral groups and that samples from Black Americans need to be included in future molecular studies to fully understand these differences, said Joshua Campbell, PhD, an assistant professor at Boston University School of Medicine who is one of the study authors.

The differences in rates of onset and outcome of prostate cancer has been recognized before and in 2018 the RESPOND study was set up by the US National Institute of Health to investigate this issue further. It aims to enroll 10,000 African-American men with prostate cancer and will investigate whether environmental issues, genetic differences, or both are responsible for the higher rates and more aggressive disease seen in these men.

Previous studies have looked in isolation at different biological, social and environmental drivers of well-known racial disparities in prostate cancer, said Franklin Huang, MD, PhD, an assistant professor at UCSF and corresponding author on the current study. RESPOND is a nationwide effort to integrate all these components and ultimately identify specific steps that can be taken to eliminate prostate cancers unequal burden in Black communities.

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African American, European Ancestry Men Benefit from Same Targeted Prostate Cancer Therapies - Clinical OMICs News

CRANFORD, N.J., July 14, 2020 /PRNewswire/ --Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a specialty pharmaceutical company focused on developing and commercializing critical care drug products, announced today that Myron S. Czuczman, M.D., has joined the company as Chief Medical Officer (CMO) and Executive Vice President. Dr. Czuczman was most recently Therapeutic Area Head, Vice President, Clinical Research and Development Global Lymphoma/CLL Program at Celgene Corporation. At Celgene, he was responsible for worldwide clinical development in Lymphoma/CLL and for the development of all compounds from Proof-of-Principle through registration globally.

Myron Holubiak, Citius CEO stated, "We are honored to have a colleague as qualified as Dr. Czuczman join the Citius team. He will be enormously helpful in furthering our development program for our planned iPSC-derived mesenchymal stem cell (iMSC) for the treatment of ARDS associated with CoVid-19. This, coupled with the advanced Phase 3 trials underway for Mino-Lok and preparing an IND for Mino-Wrap, add to the importance of bringing in an executive of Dr. Czuczman's expertise, experience, and caliber to the team."

Prior to his tenure at Celgene, Dr. Czuczman served as Chief, Lymphoma/Myeloma Service in the Department of Medicine and Head of the Lymphoma Translational Research Laboratory in the Immunology Department at Roswell Park Comprehensive Cancer Center in Buffalo, NY where he attained the title of tenured Professor of Medicine and Oncology prior to joining Celgene.

Dr. Czuczman received his M.D. from Pennsylvania State University of Medicine after graduating magna cum laude in Biochemistry from the University of Pittsburgh. He completed his Internal Medicine residency training at Weill Cornell North Shore University/MSKCC Program, followed by Medical Oncology/Hematology fellowship training at Memorial Sloan-Kettering Cancer Center in New York, NY.

Dr. Czuczman was a Founding Member and reviewer forthe National Comprehensive Cancer Network (NCCN) Lymphoma Guidelines compendium panel for nearly twenty years and he has greater than 180 peer-reviewed publications. He is a Diplomate in Internal Medicine, and is Board Certified in Medical Oncology and received numerous awards and accolades during his academic career.

About Citius Pharmaceuticals, Inc.Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care. For more information, please visit http://www.citiuspharma.com.

About Mino-LokMino-Lok is an antibiotic lock solution being developed as an adjunctive therapy in patients with central line-associated bloodstream infections (CLABSIs) or catheter-related bloodstream infections (CRBSIs). CLABSIs/CRBSIs are very serious, especially in cancer patients receiving therapy through central venous catheters (CVCs) and in hemodialysis patients, for whom venous access presents a challenge. There are currently no approved therapies for salvaging infected CVCs.

About Citius iMSCCitius's planned mesenchymal stem cell therapy product is derived from a human induced pluripotent stem cell (iPSC) line generated using a proprietary mRNA-based (non-viral) reprogramming process. The iMSCs produced from this clonal technique are differentiated from adult donor-derived MSCs (bone marrow, placenta, umbilical cord, adipose tissue, or dental pulp) by providing genetic homogeneity. In in-vitro studies, iMSCs exhibit superior potency and high cell viability. The iMSCs secrete immunomodulatory proteins that may reduce or prevent pulmonary symptoms associated with acute respiratory distress syndrome (ARDS) in patients with COVID-19. The Citius iMSC is an allogeneic (unrelated donor) mesenchymal stem-cell product manufactured by expanding material from a master cell bank.

About Acute Respiratory Distress Syndrome (ARDS)ARDS is a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs. ARDS is a rapidly progressive disease that occurs in critically ill patients most notably now in those diagnosed with COVID-19. ARDS affects approximately 200,000 patients per year in the U.S., exclusive of the current COVID-19 pandemic, and has a 30% to 50% mortality rate. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain (particularly while inhaling), and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

Safe HarborThis press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price.

Factors that could cause actual results to differ materially from those currently anticipated are: our ability to attract, integrate, and retain key personnel; our need for substantial additional funds; the risk of successfully negotiating within the option period a license agreement with Novellus, Inc. for our planned Novecite therapy for ARDS; risks associated with conducting clinical trials and drug development; the estimated markets for our product candidates and the acceptance thereof by any market; risks related to our growth strategy; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; the early stage of products under development; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis; our dependence on third-party suppliers; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Contact:

Andrew Scott Vice President, Corporate Development(O) 908-967-6677 x105[emailprotected]

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Citius Pharmaceuticals Brings on Myron S. Czuczman, MD as Chief Medical Officer (CMO) and Executive Vice President - PRNewswire

SAN DIEGO, July 13, 2020 (GLOBE NEWSWIRE) -- Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, today announced the appointment of Andrew Partridge as executive vice president and chief commercial officer, effective today. Mr. Partridge has more than 20 years of global pharmaceutical sales and marketing experience leading more than 20 commercial launches across multiple indications, including oncology, hematology and rare diseases.

As we progress our global registrational study of repotrectinib and with three other earlier stage drug candidates in our pipeline, I am pleased to have a commercial veteran of Andys caliber to lead our market planning and commercialization efforts, said Dr. Athena Countouriotis, president and chief executive officer. His depth of experience preparing for and successfully executing multiple commercial launches will serve us well and makes him an ideal fit for our team.

Mr. Partridge joins the company from Centrexion Therapeutics, a privately held biotech, where he served as both chief operating officer and chief commercial officer. He was previously with Vertex Pharmaceuticals for 5 years, most recently as senior vice president and head of North American commercial operations where he built the team to successfully launch multiple cystic fibrosis therapies and new indications. Mr. Partridge had overall responsibility at Vertex across the U.S. and Canada for marketing, sales, market access and patient services and led the cross-functional North American leadership team that included medical affairs, finance, legal and human resources. In the preceding 20 years, he served in sales and marketing roles of increasing responsibility with Schering-Plough, Roche and Amgen. At Amgen, he held senior marketing and sales roles for oncology and hematology therapeutics, including strategic planning and operations of the $5 billion oncology business unit, and therapeutic area head for oncology in global strategic marketing. His global experience also includes marketing and sales management roles based in the U.K. at Roche and Schering-Plough focused on oncology and hematology. He earned a Higher National Diploma in Medical Laboratory Sciences from Birmingham City University in the U.K.

Precision medicine offers new hope to patients with genetically driven cancers, and Turning Point has an exciting pipeline of drug candidates that could potentially address multiple difficult to treat indications, said Mr. Partridge. I look forward to working closely with Athena and the team on commercial planning and launch readiness, with the goal of getting critical therapies to patients as quickly as possible.

About Turning Point Therapeutics Inc.Turning Point Therapeuticsis a clinical-stage precision oncology company with a pipeline of internally discovered investigational drugs designed to address key limitations of existing cancer therapies. The companys lead drug candidate, repotrectinib, is a next-generation kinase inhibitor targeting the ROS1 and TRK oncogenic drivers of non-small cell lung cancer and advanced solid tumors. Repotrectinib, which is being studied in a registrational Phase 2 study in adults and a Phase 1/2 study in pediatric patients, has shown antitumor activity and durable responses among kinase inhibitor treatment-nave and pre-treated patients. The companys pipeline of drug candidates also includes TPX-0022, targeting MET, CSF1R and SRC, which is being studied in a Phase 1 trial of patients with advanced or metastatic solid tumors harboring genetic alterations in MET; TPX-0046, targeting RET and SRC, which is being studied in a Phase 1/2 trial of patients with advanced or metastatic solid tumors harboring genetic alterations in RET; and TPX-0131, a next-generation ALK inhibitor in IND-enabling studies. Turning Points next-generation kinase inhibitors are designed to bind to their targets with greater precision and affinity than existing therapies, with a novel, compact structure that has demonstrated an ability to potentially overcome treatment resistance common with other kinase inhibitors. The company is driven to develop therapies that mark a turning point for patients in their cancer treatment. For more information, visit http://www.tptherapeutics.com.

Forward Looking StatementsStatements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the efficacy, safety, and therapeutic potential of Turning Point Therapeutics drug candidates the progress of Turning Point Therapeutics development programs, and plans regarding future development and commercial activities. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as plans, will, believes, anticipates, expects, intends, goal, potential and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Turning Point Therapeutics current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Turning Point Therapeutics business in general, risks and uncertainties related to the impact of the COVID-19 pandemic to Turning Points business and the other risks described in Turning Point Therapeutics filings with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Turning Point Therapeutics undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Contact:Jim Mazzolajim.mazzola@tptherapeutics.com858-342-8272

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Turning Point Therapeutics Names Andrew Partridge As Executive Vice President and Chief Commercial Officer - GlobeNewswire