Category Archives: Gene Medicine

DUBLIN, Feb. 3, 2022 /PRNewswire/ -- The "Precision Medicine Software Market - Global Outlook & Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering.

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The precision medicine software market size was valued at USD 1,344.28 million in 2021 and is expected to reach USD 2,657.21 million by 2027, growing at a CAGR of 12.03% during the forecast period.

Favorable government initiatives and the adoption of big data analytics and related software continue to drive precision medicine software industry growth. Precision medicine software is one of the fast-growing healthcare systems IT industry segments, driven predominantly by genomics, drug discovery & development, clinical research, and big data analytics.

Start-ups are leveraging many software and machine learning algorithms to help solve major and complex problems such as reducing R&D activities timeline and billion dollars of expenditure during drug development processes.

PRECISION MEDICINE SOFTWARE MARKET SEGMENTATION

The on-cloud segment will witness an absolute growth of more than 100% in the forecast period. Cloud technology supports the industry with an agile and mountable provider engagement model. This provides better outcomes by pushing crucial information to clinicians while pulling vital, real-world insight back from key experts in the field.

Precision oncology has the highest share in Precision medicine practices by application. Oncology is the leading and fastest-growing therapeutic area in the life sciences industry. New treatments are being established at a remarkable pace, with more than 1100 oncology therapeutics in clinical development in the US alone.

GEOGRAPHICAL OUTLOOK

North America: North America made remarkable progress post the Human Genome Project in genome sequencing and precision medicine. The region is actively engaged in developing and commercializing cell and gene therapies with ICT and genome sequencing. This will drive demand in the precision medicine software industry.

Europe: The European Commission has been a driver for developing PM approaches to be readily implemented in healthcare practice. Its efforts started in 2010 with a series of workshops exploring different research areas that can contribute to developing precision medicine.

APAC: The region will likely witness a dramatic rise and innovation in precision medicine. China has already begun to make significant progress in genomics research, announcing its precision medicine initiative in 2016 with an investment of around USD 9 billion by 2030.

Story continues

VENDOR LANDSCAPE

The key players in the precision medicine software market are Syapse, AccessDx Laboratory, Fabric Genomics, Foundation Medicine, Intel, and International Business Machines (IBM).

Companies are resolving to inorganic growth approaches. AccessDx Holdings acquired 2bPrecise to create the industry's most advanced precision medicine enablement solution.

KEY HIGHLIGHTS

Blockchain technology, which works on shared ledgers and distributed networks, can ensure the data is secured and used ethically while prohibiting mishandling. Thus, blockchain technology has a huge scope in the precision medicine market.

Start-ups and scaleups are developing research platforms and techniques to better understand the underlying causes of cancer. For instance, US-America start-up OncXerna creates an RNA expression biomarker panel that permits clinical researchers to develop algorithms for effective treatment using RNA signature derived from biomarker panels.

AI leverages sophisticated computation and deep learning to overcome the obstacles involved in sizeable disparate data sets and generate insights to enable the system to learn and reason. Over the last few years, AI approaches have been used in neurodevelopmental disorders, specifically autism spectrum disorder, epileptic encephalopathy, intellectual disability, attention deficit hyperactivity disorder (ADHD), and rare genetic disorders.

KEY GROWTH FACTORS

Technological Advancements for Improvement of Precision Medicine Delivery

Increased Adoption of Cloud-Based Platform

The emergence of Local & Regional Start-Ups

Prevalence of Cancer, Genetic and Rare Diseases

Increased Partnership Among Software and Pharmaceutical Companies

Key Vendors

AccessDx Laboratory

Fabric Genomics

Foundation Medicine

Intel

IBM

Syapse

Other Prominent Vendors

GenomOncology

Koninklijke Philips

LifeOmic

NantHealth

PhenoTips

PierianDx

Qiagen

Roper Technologies

SOPHiA GENETICS

Translational Software

Key Topics Covered:

1 Research Methodology

2 Research Objectives

3 Research Process

4 Scope & Coverage4.1 Market Definition4.2 Base Year4.3 Scope Of The Study

5 Report Assumptions & Caveats5.1 Key Caveats5.2 Currency Conversion5.3 Market Derivation

6 Market at a Glance

7 Introduction7.1 Overview

8 Market Opportunities & Trends8.1 Technological Advances In Precision Medicine Delivery8.2 Increased Adoption Of Cloud-Based Platforms8.3 Emergence Of Local & Regional Start-UPS8.4 Artificial Intelligence In Precision Medicine

9 Market Growth Enablers9.1 High Prevalence Of Cancer, Genetic, & Rare Diseases9.2 Increased Partnerships Among Software & Pharmaceutical Companies9.3 Paradigm Shift Toward Tailored Disease Treatment

10 Market Growth Restraints10.1 High R&D & Implementation Cost Of Precision Medicine10.2 Dearth Of Skilled Professionals10.3 Lack Of Security & Storage For Large Volumes Of Sequenced Data

11 Market Landscape11.1 Market Overview11.2 Market Size & Forecast11.2.1 Geography Insights11.2.2 Deployment Insights11.2.3 Application Insights11.2.4 End-User Insights11.3 Five Forces Analysis

12 Deployment12.1 Market Snapshot & Growth Engine12.2 Market Overview12.3 On-Premise12.4 Cloud

13 Application13.1 Market Snapshot & Growth Engine13.2 Market Overview13.3 Precision Oncology13.4 Pharmacogenomics13.5 Rare Disease

14 End-User14.1 Market Snapshot & Growth Engine14.2 Market Overview14.3 Healthcare Providers14.4 Research Labs14.5 Pharma & Biotech Companies

15 Geography15.1 Market Snapshot & Growth Engine15.2 Geographic Overview

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

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

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Global Precision Medicine Software Market Report 2022: Market was Valued at $1,344.28 Million in 2021 and is Expected to Reach $2,657.21 Million by...

A new study confirms that G207, a genetically engineered virus developed at UAB, may be a beneficial therapy for brain tumors.

A new study confirms that G207, a genetically engineered virus developed at UAB, may be a beneficial therapy for brain tumors.A new study provides additional evidence of the efficacy of virotherapy for glioblastoma, the most deadly type of brain tumor. The research findings, published Feb. 1, 2022, inClinical Cancer Research, indicate that an oncolytic herpes simplex virus, G207, appears to boost immune response and that this is associated with better overall survival for patients with glioblastoma.

The study, from investigators at theUniversity of Alabama at BirminghamandNationwide Childrens Hospital, builds on previous trials of G207. The current study further examined findings from an earlier Phase 1b study of six patients with glioblastoma recurrence. The earlier study indicated G207 was well tolerated by the patients. Overall survival, a secondary outcome, was generally but not uniformly improved in the study.

In this study, we sought to identify biological differences that could describe the variable survival duration associated with G207 virotherapy, said James Markert, M.D., Ph.D., chair of theDepartment of Neurosurgeryat UABsHeersink School of Medicineand a study co-author.

We performed RNA sequencing from pre- and post-G207 treatment tumor biopsies to investigate changes in gene expression during the treatment interval. We hypothesized that gene expression indicative of an active immune response would be characteristic of patients with improved survival.

The research team found that the adaptive immune response differed between patients and indicated there was an association with this immune response and increased length of survival in patients with recurrent glioblastoma after treatment with G207.

Glioblastomas are central nervous system tumors that are uniformly fatal with median survival of 12 to 15 months from initial diagnosis and four to six months after recurrence. The standard medical care including surgical removal of the tumor, chemotherapy and radiation currently offers limited survival benefit.

Markert, who is a senior scientist in the O'Neal Comprehensive Cancer Center, says previous research has confirmed that glioblastomas have an immunosuppressive quality, meaning they inhibit the bodys immune system from attacking and destroying the tumor. This allows the tumor to evade the immune system and continue to grow and spread. Chemotherapy and radiation therapy, necessary treatments for the tumor, unfortunately exacerbate suppression of the immune response.

Immunotherapies are an evolving strategy for cancer treatment. They are designed to stimulate the immune system to attack tumor cells and have led to remarkable responses in many cancers.

Oncolytic virotherapy, in particular, involves genetically engineered viruses designed to selectively replicate in tumor cells, relieving immunosuppression in the tumor microenvironment and enhancing antitumor immune responses.

Markert first became interested in genetically engineered viruses while a neurosurgical resident in a Boston laboratory more than 30 years ago. The lab was involved in testing the first genetically engineered herpes virus as an anti-cancer drug, based on a suitable candidate initially constructed to serve as a vaccine against herpes. The realization followed that the virus might be effective as a means to destroy brain tumors.

In 2001, Markert and his colleagues published initial results of G207, indicating it was safe to use as a sole therapy as well as in combination with radiation for malignant gliomas.

For the current study, Katherine Miller, Ph.D., a research assistant professor in the Institute for Genomic Medicine at Nationwide Childrens and the Ohio State University College of Medicine, looked at RNA extracted from the patients before and after treatment with G207. Analysis of 770 immune response genes representing 24 different immune cell types revealed that G207 treatment, more than any other component, had the greatest effect on immune gene expression changes.

They also examined which immune cell types were enriched in the post-G207 samples. Looking at 109 marker genes specific to 24 major immune-cell populations to assign cell type scores, they found that nearly all post-G207 samples had a higher abundance of immune cells relative to pre-G207 samples.

Specifically, G207 treatment significantly increased the cytotoxic, T-cell, natural killer, macrophage, neutrophil and dendritic cell scores, said study co-author Kevin Cassady, M.D., professor of pediatrics at theOhio State University College of Medicineand aninfectious diseases physicianat Nationwide Childrens Hospital. We also found that G207 reduced the number of dysfunctional T cells, known as exhausted T cells in that they become tolerant of the tumor they are supposed to attack. Exhausted T cells are one contributor to a poor immune response.

The investigators also looked to see whether there was a direct correlation between the post-G207 treatment gene expression levels and survival.

We detected approximately 500 genes that significantly correlated with patient survival and demonstrated that approximately 50 percent of these genes were related to immune response pathways and functions, Markert said. Our analysis of immune-cell populations after treatment with G207 revealed associations with patient survival and identified important mechanistic events related to cellular immune infiltrate changes including an increase in the myeloid, cytotoxic and T-cell populations, suggesting a relationship between immune gene response and survival duration.

The authors suggest that the gene sets identified permit further examination of gene expression response predictors when evaluating the impact of different recombinant oncolytic viruses. Currently, there are numerous active oncolytic viral clinical trials ongoing, five of which involve the treatment of central nervous system tumors using recombinant herpes virus vectors of various designs.

Malignant glioma is one of the most devastating forms of cancer, Cassady said. Survival after diagnosis is often no more than a few years and is frequently measured in months. Our hope is that these genetically engineered viruses will ultimately extend lifespan and improve quality of life for patients with these malignant brain tumors.

This research was supported by grants from the National Cancer Institute, part of the National Institutes of Health.

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Study supports virotherapy as a potential treatment for brain tumors - The Mix

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REDWOOD CITY, Calif. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Kriya Therapeutics, Inc., a fully integrated company pioneering novel technologies and therapeutics in gene therapy, announced today that it has appointed Maan Muhsin, M.D., as President and Chief Medical Officer of Kriya Oncology, the companys oncology therapeutic area division. Dr. Muhsin will lead overall strategic, development, and partnership activities to accelerate and expand Kriyas portfolio of transformative gene therapies for cancers of high unmet need.

Maan brings a wealth of experience in the development of novel oncology therapies across a number of modalities, said Shankar Ramaswamy, M.D., Co-Founder and Chief Executive Officer of Kriya. He is uniquely positioned to accelerate the expansion and clinical translation of our growing pipeline of gene therapies that can be combined with existing and emerging standards of care in oncology. We are keen to bring forward in vivo gene therapy as a new modality to treat cancer, and Maan is a leader with an exceptional track record that will help us achieve this goal.

Dr. Muhsin previously served as Chief Medical Officer at Medicenna Therapeutics where he designed and executed clinical trials of the companys solid tumor programs. Prior to joining Medicenna, he served as Medical Lead, Oncology Clinical Development for Nektar Therapeutics where he oversaw the progression of the PIVOT-12 and REVEAL clinical studies for metastatic melanoma and advanced and local solid tumors, respectively. Dr. Muhsin has held roles of increasing responsibility at HUYA Bioscience International where he served as the Senior Vice President, Oncology Clinical Development, and at Halozyme Therapeutics where he served as Senior Medical Director, Oncology Clinical Development. He also worked in the U.S. Army Combat Support Hospitals (CSH) and held other positions within the Medical Brigade and the Medical Command under the United States Department of Defense (DoD). Dr. Muhsin completed his medical education at the Baghdad University School of Medicine and completed postgraduate education in oncology drug development at Tufts University Center for the Study of Drug Development (CSDD).

I am excited to join Kriya and look forward to advancing its promising portfolio of gene therapies in oncology, said Dr. Muhsin. While the management of cancer has come a long way in the last decade, I believe in the potential to further enhance the treatment of patients with the incorporation of rationally engineered gene therapies that can transform treatment paradigms for a wide array of cancers. I look forward to leveraging Kriyas fully-integrated gene therapy engine to deliver a pipeline of novel medicines with the potential to significantly impact the lives of cancer patients.

About Kriya

Kriya is a fully integrated company pioneering novel technologies and therapeutics in gene therapy. The company aims to revolutionize how gene therapies are designed, developed, and manufactured, improving speed to market and delivering significant reductions in cost. Kriya is organized into four principal business units: Kriya Technologies, Kriya Therapeutics, Kriya R&D, and Kriya Manufacturing. The company is advancing a deep and diversified pipeline of innovative gene therapies in multiple therapeutic area divisions, with current pipeline programs in ophthalmology, oncology, rare disease, and chronic disease. Kriya was founded by pioneers in the biopharmaceutical industry and is backed by leading life sciences and technology investors. The company has core operations in Silicon Valley, California and Research Triangle Park, North Carolina. For more information, please visit http://www.kriyatx.com and follow on LinkedIn.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220203005305/en/

Danielle CanteyCanale Communicationsdanielle.cantey@canalecomm.com(619) 826-4657

Source: Kriya Therapeutics, Inc.

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Kriya Announces the Appointment of Ma'an Muhsin, MD, as President and Chief Medical Officer of Its Oncology Therapeutic Area Division -...

Several years ago, in a lab meeting, one of Nikolaus Schultzs postdoctoral fellows posed a question: Do you think its possible to predict, based on the genomic profile of an individual tumor, which organs it might metastasize to?

And just like that, a research project was born.

Dr. Schultz is a computational oncologist in the Department of Epidemiology and Biostatistics at Memorial Sloan Kettering Cancer Center. He also serves as Head of Knowledge Systems in the Marie-Jose and Henry R. Kravis Center for Molecular Oncology. In these dual roles, he has gained a wealth of experience sequencing the DNA in patients tumors and using this information to help physicians guide treatment.

MSK computational oncologists Francisco Sanchez-Vega and Nikolaus Schultz

Every person with advanced cancer who is treated at MSK undergoes genetic sequencing of their tumor with MSK-IMPACT, a tool that looks for mutations in 400-plus cancer-associated genes. Since sequencing started in 2014, MSK has compiled genetic tumor profiles from more than 50,000 patients. This vast repository represents a goldmine for biologists interested in asking questions about the relationships between tumor mutations and cancer progression.

Dr. Schultz and Francisco Sanchez-Vega the curious postdoc who originally posed the question about metastasis and who is now an Assistant Attending Computational Oncologist at MSK realized that they could use computational techniques to search this mountain of data for clues. Specifically, they could ask whether particular mutations (or groups of mutations) correlate with the spread of cancer to particular organs, across many different types of cancer.

That was six years ago. Now, in a paper published February 3, 2022, in the journal Cell, Drs. Schultz and Sanchez-Vega and their team, including postdoctoral researchers Bastien Nguyen and Christopher Fong, and 71 other MSK scientists present the results of their investigation.

To the question of whether its possible to look at an individual tumor and, based on its genomic profile alone, predict its precise future metastatic trajectory, the answer is clearly no.

VIDEO | 01:38

Learn about metastasis, which is the spreading of cancer from its original location to a new location. Memorial Sloan Kettering researchers are making advances in understanding the three stages of metastasis.

While we found some gene mutations to be slightly more common in tumor samples with specific metastatic transitions, we found no single gene that, when mutated, will predict with absolute certainty whether or not a tumor will metastasize to a particular organ, Dr. Schultz says.

The study was revealing in other ways: At a very high level, what the data are telling us is that metastatic disease is genomically different from primary disease, Dr. Schultz says.

For example, in many cancer types, metastatic tumors have more of what geneticists call DNA copy-number changes compared with primary tumors. A DNA copy-number change is when a particular segment of DNA is present in greater or fewer than the normal number of copies. These copy-number changes, when observed in primary tumors, were an indicator of metastatic potential, Dr. Schultz points out.

In addition, he notes, some cancer-driving mutations were detected at different frequencies in metastases compared with primary tumors across a variety of cancer types.

Dr. Schultz likens the extensive dataset his team has curated to The Cancer Genome Atlas (TCGA) project, in which he was also involved. An important goal of TCGA was to assemble a valuable dataset and get it out into the world so that others could mine it, he says.

To help researchers mine these new data for insights, the team is making them publicly available through the cBioPortal for Cancer Genomics as MSK-MET (Memorial Sloan Kettering Metastatic Events and Tropisms).

The dataset includes information on genetic changes and clinical outcomes from 25,000 patients across 50 different cancer types.

Because no single mutation or set of mutations stood out as reliable predictors of metastatic behavior across tumor types, the new study may add support to an emerging framework in cancer science that views metastasis the cause of 90% of cancer deaths as not primarily driven by genetic mutations. Rather, epigenetic changes that occur in cancer cells as a consequence of their interactions with normal cells in the surrounding environment could be more to blame. (Epigenetic changes are ones that alter what genes are turned on or off in a cell without altering the DNA sequence as a mutation would.) These changes may underlie the ability of metastatic cells to adapt to otherwise hostile tissue environments.

To the question of whether its possible to look at an individual tumor and, based on its genomic profile alone, predict its precise future metastatic trajectory, the answer is clearly no.

Recent discoveries from other investigators at MSK including Joan Massagu, Karuna Ganesh, Charles Sawyers, Kat Hadjantonakis, and Dana Peer have pointed to a kind of epigenetically driven identity switching that enables cancer cells to assume more developmentally primitive states and thereby grow in new parts of the body.

Exploring the ramifications of these and other findings will be the focus of MSKs new Marie-Jose and Henry R. Kravis Cancer Ecosystems Project, which launches today.

Currently, genetic sequencing of tumors with MSK-IMPACT can inform whether or not a specific patient should receive a specific drug based on a single genomic alteration. For example, if someone is found to have a mutation in the BRAF gene, they might be a good candidate for a targeted drug called vemurafenib.

But Dr. Schultz points out that there is potentially much more information to be found in the mass of sequencing data and associated patient clinical data that could inform treatment decisions.

When properly mined, this data could tell us a lot more about prognosis of these patients, whether or not their tumors will metastasize, maybe even to what tissues, he says. This information might determine how these patients get monitored in the future. We can learn about what treatments they might be more sensitive to or less sensitive to. I think that theres enormous potential for personalized medicine that goes beyond the single biomarker, and therefore justification to keep sequencing more patients with broader sequencing panels to increase our power to make new discoveries.

Key Takeaways

See the article here:
The Mystery of Metastasis: Can a Tumors Genetic Mutations Predict Whether and Where Cancer Will Spread? - On Cancer - Memorial Sloan Kettering

DUBLIN, Feb. 1, 2022 /PRNewswire/ -- The "Cell & Gene Therapy Market - Global Outlook & Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering.

The cell and gene therapy market size was valued at USD 4.99 billion in 2021 and is expected to reach USD 36.92 billion by 2027, growing at a CAGR of 39.62 % during the forecast period

In the cell and gene therapy field, gene therapy gathered the pace last from 2 decades because of the discovery of several genes responsible for mutation in various diseases. The advancement in the cell & gene therapy field and innovative technologies give the new era for biological therapeutics. Also, PRIME Designation and marketing authorization for products provide a new opportunity for the manufacturer's financing and revenue generation.

KEY HIGHLIGHTS

As per the American Society of Cell + Gene Therapy report in 2021, increasing the number of cellular and gene therapy products, application rate and products in clinical trials drive the market growth.

As per the Dive Biopharma report 2021, biotech companies who actively engaged in regenerative medicines and therapies reported USD 14 billion funding only in six months of 2021 which was reported to be USD 19.9 billion for the overall year.

CELL AND GENE THERAPY MARKET SEGMENTATION

Increasing application of gene therapies in diseases diagnosis and rapidly growing new drugs applications will give new market space in upcoming years. In 2020, around USD 2.3 billion funding was reported only from private companies for gene therapies. By 2025, the FDA is expected to approve 10?20 products each year, driving the global cell and gene therapy manufacturing market.

In 2020, Medicine in Development Report 2020, around 176 products were reported in cancer therapies in development procedures.

GEOGRAPHICAL OUTLOOK

North America: High economic status and high expenditure on healthcare services drive the cell and gene therapy market in North America. National Health Institutes, industries, academic institutes, and hospitals are the significant contributors of sponsorship and financial funding for cell and gene therapy products.

Europe: The increasing funding for cell and gene therapy drives the cell and gene therapy market growth consistently in Europe. Around USD 2.6 billion financings were reported in Europe for CGTs in 2020, which increased by 103% compared to previous years. In the cell therapy segment, USD 1.8 billion and in gene therapy, USD 2.3 billion funding accounted in 2020, which increased by 196% and 111% growth respectively

VENDOR LANDSCAPE

The key players in the cell and gene therapy market are Gilead Sciences, Novartis, Smith Nephew, Amgen, Organogenesis, Roche (Spark Therapeutics), Dendreon, Vericel, and Bristol-Myers Squibb Company.

An increasing number of mergers and acquisitions gives new potential to market growth. Gilead Sciences acquired Kite Pharma in 2020. Also, Novartis acquired Avexis in 2018, and Smith & Nephew acquired Osiris Therapeutics.

MAJOR GROWTH FACTORS

KEY VENDORS

OTHER PROMINENT VENDORS

UPCOMING VENDORS

Key Topics Covered:

1 Research Methodology

2 Research Objectives

3 Research Process

4 Scope & Coverage4.1 Market Definition4.2 Base Year4.3 Scope of The Study

5 Report Assumptions & Caveats5.1 Key Caveats5.2 Currency Conversion5.3 Market Derivation

6 Market at a Glance

7 Introduction7.1 Overview7.1.1 Cell & Gene Therapy Approved Products 2020-20217.2 Cell & Gene Therapy Phase-III Products7.3 Road Map of Cell & Gene Therapy

8 Market Opportunities & Trends8.1 Rising Number of Mergers & Acquisitions8.2 Expansion of CGT Manufacturing Plants8.3 Expanding Applications for Cell & Gene Therapies8.4 Growing Demand for Car T-Cell Therapies

9 Market Growth Enablers9.1 New Product Approvals & Increasing Pipeline of Products9.2 Prime Designation & Funding Support For CGT9.3 Rising Use of CGT Products for Disease Care9.4 Increasing Use of CGT Products for Disease Treatment

10 Market Restraints10.1 High Cost of Cell & Gene Therapies10.2 Ethical Issues Regarding Genetical Material10.3 Stringent Regulation for CGT Approvals

11 Market Landscape11.1 Market Overview11.2 Market Size & Forecast11.3 Five Forces Analysis

12 Therapy12.1 Market Snapshot & Growth Engine12.2 Market Overview12.3 Gene Therapy12.4 Cell Therapy

13 Application13.1 Market Snapshot & Growth Engine13.2 Market Overview13.3 Oncology13.4 Genetic Disorders13.5 Dermatology13.6 Musculoskeletal Diseases13.7 Other Diseases

14 End-User14.1 Market Snapshot & Growth Engine14.2 Market Overview14.3 Hospitals14.4 Cancer Care Centers14.5 Wound Care Centers14.6 Other End-Users

15 Geography15.1 Market Snapshot & Growth Engine15.2 Geographic Overview15.2.1 Global Cell & Gene Therapy Market by Geography

For more information about this report visit https://www.researchandmarkets.com/r/z7gm5

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1904 Fax (outside U.S.): +353-1-481-1716

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Here is the original post:
Global Cell & Gene Therapy Market Research Report 2022: Expanding Application for Cell & Gene Therapies & Growing Demand for CAR T- Cell...

The authors would like to thank the participants of the individual studies contributing to the BCAC, GECCO, CORECT, CCFR, INTEGRAL-ILCCO, PRACTICAL, Finngen, BioBank Japan, Asia Colorectal Cancer Consortium, ICBP, DIAGRAM, CARDIoGRAMplusC4D, and MEGASTROKE consortia and the Genetic Epidemiology Research on Adult Health, UK Biobank, and the Korean National Cancer Center CRC Study 2. The authors would also like to acknowledge the investigators of these consortia and studies for generating the data used for this analysis. The authors would like to acknowledge the following investigators of the OncoArray and GAME-ON1KG INTEGRAL-ILCCO analyses: Maria Teresa Landi, Victoria Stevens, Ying Wang, Demetrios Albanes, Neil Caporaso, Paul Brennan, Christopher I Amos, Sanjay Shete, Rayjean J Hung, Heike Bickebller, Angela Risch, Richard Houlston, Stephen Lam, Adonina Tardon, Chu Chen, Stig E Bojesen, Mattias Johansson, H-Erich Wichmann, David Christiani, Gadi Rennert, Susanne Arnold, John K. Field, Loic Le Marchand, Olle Melander, Hans Brunnstrm, Geoffrey Liu, Angeline Andrew, Lambertus A Kiemeney, Hongbing Shen, Shan Zienolddiny, Kjell Grankvist, Mikael Johansson, M Dawn Teare, Yun-Chul Hong, Jian-Min Yuan, Philip Lazarus, Matthew B Schabath, Melinda C Aldrich. Cancer consortia-specific funding and acknowledgments is presented in S1 Text.

http://practical.icr.ac.uk/

Rosalind A. Eeles1,2, Christopher A. Haiman3, Zsofia Kote-Jarai1, Fredrick R. Schumacher4,5, Sara Benlloch6,1, Ali Amin Al Olama6,7, Kenneth Muir8,9, Sonja I. Berndt10, David V. Conti3, Fredrik Wiklund11, Stephen Chanock10, Ying Wang12, Victoria L. Stevens12, Catherine M. Tangen13, Jyotsna Batra14,15, Judith A. Clements14,15, APCB BioResource (Australian Prostate Cancer BioResource)14,15, Henrik Grnberg11, Nora Pashayan16,17, Johanna Schleutker18,19, Demetrius Albanes10, Stephanie Weinstein10, Alicja Wolk20, Catharine M. L. West21, Lorelei A. Mucci22, Graldine Cancel-Tassin23,24, Stella Koutros10, Karina Dalsgaard Srensen25,26, Eli Marie Grindedal27, David E. Neal28,29,30, Freddie C. Hamdy31,32, Jenny L. Donovan33, Ruth C. Travis34, Robert J. Hamilton35,36, Sue Ann Ingles37, Barry S. Rosenstein38,39, Yong-Jie Lu40, Graham G. Giles41,42,43, Adam S. Kibel44, Ana Vega45,46,47, Manolis Kogevinas48,49,50,51, Kathryn L. Penney52, Jong Y. Park53, Janet L. Stanford54,55, Cezary Cybulski56, Brge G. Nordestgaard57,58, Sune F. Nielsen57,58, Hermann Brenner59,60,61, Christiane Maier62, Jeri Kim63, Esther M. John64, Manuel R. Teixeira65,66,67, Susan L. Neuhausen68, Kim De Ruyck69, Azad Razack70, Lisa F. Newcomb54,71, Davor Lessel72, Radka Kaneva73, Nawaid Usmani74,75, Frank Claessens76, Paul A. Townsend77,78, Jose Esteban Castelao79, Monique J. Roobol80, Florence Menegaux81, Kay-Tee Khaw82, Lisa Cannon-Albright83,84, Hardev Pandha78, Stephen N. Thibodeau85, David J. Hunter86, Peter Kraft87, William J. Blot88,89, Elio Riboli90

1 The Institute of Cancer Research, London, SM2 5NG, UK

2 Royal Marsden NHS Foundation Trust, London, SW3 6JJ, UK

3 Center for Genetic Epidemiology, Department of Preventive Medicine, Keck School of Medicine, University of Southern California/Norris Comprehensive Cancer Center, Los Angeles, CA 90015, USA

4 Department of Population and Quantitative Health Sciences, Case Western Reserve University, Cleveland, OH 441067219, USA

5 Seidman Cancer Center, University Hospitals, Cleveland, OH 44106, USA

6 Centre for Cancer Genetic Epidemiology, Department of Public Health and Primary Care, University of Cambridge, Strangeways Research Laboratory, Cambridge CB1 8RN, UK

7 University of Cambridge, Department of Clinical Neurosciences, Stroke Research Group, R3, Box 83, Cambridge Biomedical Campus, Cambridge CB2 0QQ, UK

8 Division of Population Health, Health Services Research and Primary Care, University of Manchester, Oxford Road, Manchester, M13 9PL, UK

9 Warwick Medical School, University of Warwick, Coventry, CV4 7AL, UK

10 Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Bethesda, Maryland, 20892, USA

11 Department of Medical Epidemiology and Biostatistics, Karolinska Institute, SE-171 77 Stockholm, Sweden

12 Department of Population Science, American Cancer Society, 250 Williams Street, Atlanta, GA 30303, USA

13 SWOG Statistical Center, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA

14 Australian Prostate Cancer Research Centre-Qld, Institute of Health and Biomedical Innovation and School of Biomedical Sciences, Queensland University of Technology, Brisbane QLD 4059, Australia

15 Translational Research Institute, Brisbane, Queensland 4102, Australia

16 Department of Applied Health Research, University College London, London, WC1E 7HB, UK

17 Centre for Cancer Genetic Epidemiology, Department of Oncology, University of Cambridge, Strangeways Laboratory, Worts Causeway, Cambridge, CB1 8RN, UK

18Institute of Biomedicine, University of Turku, Finland

19 Department of Medical Genetics, Genomics, Laboratory Division, Turku University Hospital, PO Box 52, 20521 Turku, Finland

20 Department of Surgical Sciences, Uppsala University, 75185 Uppsala, Sweden

21 Division of Cancer Sciences, University of Manchester, Manchester Academic Health Science Centre, Radiotherapy Related Research, The Christie Hospital NHS Foundation Trust, Manchester, M13 9PL UK

22 Department of Epidemiology, Harvard T. H. Chan School of Public Health, Boston, MA 02115, USA

23 CeRePP, Tenon Hospital, F-75020 Paris, France

24 Sorbonne Universite, GRC n5, AP-HP, Tenon Hospital, 4 rue de la Chine, F-75020 Paris, France

25 Department of Molecular Medicine, Aarhus University Hospital, Palle Juul-Jensen Boulevard 99, 8200 Aarhus N, Denmark

26 Department of Clinical Medicine, Aarhus University, DK-8200 Aarhus N

27 Department of Medical Genetics, Oslo University Hospital, 0424 Oslo, Norway

28 Nuffield Department of Surgical Sciences, University of Oxford, Room 6603, Level 6, John Radcliffe Hospital, Headley Way, Headington, Oxford, OX3 9DU, UK

29 University of Cambridge, Department of Oncology, Box 279, Addenbrookes Hospital, Hills Road, Cambridge CB2 0QQ, UK

30 Cancer Research UK, Cambridge Research Institute, Li Ka Shing Centre, Cambridge, CB2 0RE, UK

31 Nuffield Department of Surgical Sciences, University of Oxford, Oxford, OX1 2JD, UK

32 Faculty of Medical Science, University of Oxford, John Radcliffe Hospital, Oxford, UK

33 Population Health Sciences, Bristol Medical School, University of Bristol, BS8 2PS, UK

34 Cancer Epidemiology Unit, Nuffield Department of Population Health, University of Oxford, Oxford, OX3 7LF, UK

35 Dept. of Surgical Oncology, Princess Margaret Cancer Centre, Toronto ON M5G 2M9, Canada

36 Dept. of Surgery (Urology), University of Toronto, Canada

37 Department of Preventive Medicine, Keck School of Medicine, University of Southern California/Norris Comprehensive Cancer Center, Los Angeles, CA 90015, USA

38 Department of Radiation Oncology and Department of Genetics and Genomic Sciences, Box 1236, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, New York, NY 10029, USA

39 Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 100295674, USA

40 Centre for Cancer Biomarker and Biotherapeutics, Barts Cancer Institute, Queen Mary University of London, John Vane Science Centre, Charterhouse Square, London, EC1M 6BQ, UK

41 Cancer Epidemiology Division, Cancer Council Victoria, 615 St Kilda Road, Melbourne, VIC 3004, Australia

42 Centre for Epidemiology and Biostatistics, Melbourne School of Population and Global Health, The University of Melbourne, Grattan Street, Parkville, VIC 3010, Australia

43 Precision Medicine, School of Clinical Sciences at Monash Health, Monash University, Clayton, Victoria 3168, Australia

44 Division of Urologic Surgery, Brigham and Womens Hospital, 75 Francis Street, Boston, MA 02115, USA

45 Fundacin Pblica Galega Medicina Xenmica, Santiago de Compostela, 15706, Spain

46 Instituto de Investigacin Sanitaria de Santiago de Compostela, Santiago De Compostela, 15706, Spain

47 Centro de Investigacin en Red de Enfermedades Raras (CIBERER), Spain

48 ISGlobal, Barcelona, Spain

49 IMIM (Hospital del Mar Medical Research Institute), Barcelona, Spain

50 CIBER Epidemiologa y Salud Pblica (CIBERESP), 28029 Madrid, Spain

51 Universitat Pompeu Fabra (UPF), Barcelona, Spain

52 Channing Division of Network Medicine, Department of Medicine, Brigham and Womens Hospital/Harvard Medical School, Boston, MA 02115, USA

53 Department of Cancer Epidemiology, Moffitt Cancer Center, 12902 Magnolia Drive, Tampa, FL 33612, USA

54 Division of Public Health Sciences, Fred Hutchinson Cancer Research Center, Seattle, Washington, 981091024, USA

55 Department of Epidemiology, School of Public Health, University of Washington, Seattle, Washington 98195, USA

56 International Hereditary Cancer Center, Department of Genetics and Pathology, Pomeranian Medical University, 70115 Szczecin, Poland

57 Faculty of Health and Medical Sciences, University of Copenhagen, 2200 Copenhagen, Denmark

58 Department of Clinical Biochemistry, Herlev and Gentofte Hospital, Copenhagen University Hospital, Herlev, 2200 Copenhagen, Denmark

59 Division of Clinical Epidemiology and Aging Research, German Cancer Research Center (DKFZ), D-69120, Heidelberg, Germany

60 German Cancer Consortium (DKTK), German Cancer Research Center (DKFZ), D-69120 Heidelberg, Germany

61 Division of Preventive Oncology, German Cancer Research Center (DKFZ) and National Center for Tumor Diseases (NCT), Im Neuenheimer Feld 460, 69120 Heidelberg, Germany

62 Humangenetik Tuebingen, Paul-Ehrlich-Str 23, D-72076 Tuebingen, Germany

63 The University of Texas M. D. Anderson Cancer Center, Department of Genitourinary Medical Oncology, 1515 Holcombe Blvd., Houston, TX 77030, USA

64 Departments of Epidemiology & Population Health and of Medicine, Division of Oncology, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA 94304 USA

65 Department of Genetics, Portuguese Oncology Institute of Porto (IPO-Porto), 4200072 Porto, Portugal

66 Biomedical Sciences Institute (ICBAS), University of Porto, 4050313 Porto, Portugal

67 Cancer Genetics Group, IPO-Porto Research Center (CI-IPOP), Portuguese Oncology Institute of Porto (IPO-Porto), 4200072 Porto, Portugal

68 Department of Population Sciences, Beckman Research Institute of the City of Hope, 1500 East Duarte Road, Duarte, CA 91010, 626-256-HOPE (4673)

69 Ghent University, Faculty of Medicine and Health Sciences, Basic Medical Sciences, Proeftuinstraat 86, B-9000 Gent

70 Department of Surgery, Faculty of Medicine, University of Malaya, 50603 Kuala Lumpur, Malaysia

71 Department of Urology, University of Washington, 1959 NE Pacific Street, Box 356510, Seattle, WA 98195, USA

72 Institute of Human Genetics, University Medical Center Hamburg-Eppendorf, D-20246 Hamburg, Germany

73 Molecular Medicine Center, Department of Medical Chemistry and Biochemistry, Medical University of Sofia, Sofia, 2 Zdrave Str., 1431 Sofia, Bulgaria

74 Department of Oncology, Cross Cancer Institute, University of Alberta, 11560 University Avenue, Edmonton, Alberta, Canada T6G 1Z2

75 Division of Radiation Oncology, Cross Cancer Institute, 11560 University Avenue, Edmonton, Alberta, Canada T6G 1Z2

76 Molecular Endocrinology Laboratory, Department of Cellular and Molecular Medicine, KU Leuven, BE-3000, Belgium

77 Division of Cancer Sciences, Manchester Cancer Research Centre, Faculty of Biology, Medicine and Health, Manchester Academic Health Science Centre, NIHR Manchester Biomedical Research Centre, Health Innovation Manchester, Univeristy of Manchester, M13 9WL

78 The University of Surrey, Guildford, Surrey, GU2 7XH, UK

79 Genetic Oncology Unit, CHUVI Hospital, Complexo Hospitalario Universitario de Vigo, Instituto de Investigacin Biomdica Galicia Sur (IISGS), 36204, Vigo (Pontevedra), Spain

80 Department of Urology, Erasmus University Medical Center, 3015 CE Rotterdam, The Netherlands

81 "Exposome and Heredity", CESP (UMR 1018), Facult de Mdecine, Universit Paris-Saclay, Inserm, Gustave Roussy, Villejuif

82 Clinical Gerontology Unit, University of Cambridge, Cambridge, CB2 2QQ, UK

83 Division of Epidemiology, Department of Internal Medicine, University of Utah School of Medicine, Salt Lake City, Utah 84132, USA

84 George E. Wahlen Department of Veterans Affairs Medical Center, Salt Lake City, Utah 84148, USA

85 Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN 55905, USA

86 Nuffield Department of Population Health, University of Oxford, United Kingdom

87 Program in Genetic Epidemiology and Statistical Genetics, Department of Epidemiology, Harvard School of Public Health, Boston, MA, USA

88 Division of Epidemiology, Department of Medicine, Vanderbilt University Medical Center, 2525 West End Avenue, Suite 800, Nashville, TN 37232 USA

89 International Epidemiology Institute, Rockville, MD 20850, USA

90 Department of Epidemiology and Biostatistics, School of Public Health, Imperial College London, SW7 2AZ, UK

Rainer Malik 1, Ganesh Chauhan 2, Matthew Traylor 3, Muralidharan Sargurupremraj 4,5, Yukinori Okada 6,7,8, Aniket Mishra 4,5, Loes Rutten-Jacobs 3, Anne-Katrin Giese 9, Sander W van der Laan 10, Solveig Gretarsdottir 11, Christopher D Anderson 12,13,14,14, Michael Chong 15, Hieab HH Adams 16,17, Tetsuro Ago 18, Peter Almgren 19, Philippe Amouyel 20,21, Hakan Ay 22,13, Traci M Bartz 23, Oscar R Benavente 24, Steve Bevan 25, Giorgio B Boncoraglio 26, Robert D Brown, Jr. 27, Adam S Butterworth 28,29, Caty Carrera 30,31, Cara L Carty 32,33, Daniel I Chasman 34,35, Wei-Min Chen 36, John W Cole 37, Adolfo Correa 38, Ioana Cotlarciuc 39, Carlos Cruchaga 40,41, John Danesh 28,42,43,44, Paul IW de Bakker 45,46, Anita L DeStefano 47,48, Marcel den Hoed 49, Qing Duan 50, Stefan T Engelter 51,52, Guido J Falcone 53,54, Rebecca F Gottesman 55, Raji P Grewal 56, Vilmundur Gudnason 57,58, Stefan Gustafsson 59, Jeffrey Haessler 60, Tamara B Harris 61, Ahamad Hassan 62, Aki S Havulinna 63,64, Susan R Heckbert 65, Elizabeth G Holliday 66,67, George Howard 68, Fang-Chi Hsu 69, Hyacinth I Hyacinth 70, M Arfan Ikram 16, Erik Ingelsson 71,72, Marguerite R Irvin 73, Xueqiu Jian 74, Jordi Jimnez-Conde 75, Julie A Johnson 76,77, J Wouter Jukema 78, Masahiro Kanai 6,7,79, Keith L Keene 80,81, Brett M Kissela 82, Dawn O Kleindorfer 82, Charles Kooperberg 60, Michiaki Kubo 83, Leslie A Lange 84, Carl D Langefeld 85, Claudia Langenberg 86, Lenore J Launer 87, Jin-Moo Lee 88, Robin Lemmens 89,90, Didier Leys 91, Cathryn M Lewis 92,93, Wei-Yu Lin 28,94, Arne G Lindgren 95,96, Erik Lorentzen 97, Patrik K Magnusson 98, Jane Maguire 99, Ani Manichaikul 36, Patrick F McArdle 100, James F Meschia 101, Braxton D Mitchell 100,102, Thomas H Mosley 103,104, Michael A Nalls 105,106, Toshiharu Ninomiya 107, Martin J ODonnell 15,108, Bruce M Psaty 109,110,111,112, Sara L Pulit 113,45, Kristiina Rannikme 114,115, Alexander P Reiner 65,116, Kathryn M Rexrode 117, Kenneth Rice 118, Stephen S Rich 36, Paul M Ridker 34,35, Natalia S Rost 9,13, Peter M Rothwell 119, Jerome I Rotter 120,121, Tatjana Rundek 122, Ralph L Sacco 122, Saori Sakaue 7,123, Michele M Sale 124, Veikko Salomaa 63, Bishwa R Sapkota 125, Reinhold Schmidt 126, Carsten O Schmidt 127, Ulf Schminke 128, Pankaj Sharma 39, Agnieszka Slowik 129, Cathie LM Sudlow 114,115, Christian Tanislav 130, Turgut Tatlisumak 131,132, Kent D Taylor 120,121, Vincent NS Thijs 133,134, Gudmar Thorleifsson 11, Unnur Thorsteinsdottir 11, Steffen Tiedt 1, Stella Trompet 135, Christophe Tzourio 5,136,137, Cornelia M van Duijn 138,139, Matthew Walters 140, Nicholas J Wareham 86, Sylvia Wassertheil-Smoller 141, James G Wilson 142, Kerri L Wiggins 109, Qiong Yang 47, Salim Yusuf 15, Najaf Amin 16, Hugo S Aparicio 185,48, Donna K Arnett 186, John Attia 187, Alexa S Beiser 47,48, Claudine Berr 188, Julie E Buring 34,35, Mariana Bustamante 189, Valeria Caso 190, Yu-Ching Cheng 191, Seung Hoan Choi 192,48, Ayesha Chowhan 185,48, Natalia Cullell 31, Jean-Franois Dartigues 193,194, Hossein Delavaran 95,96, Pilar Delgado 195, Marcus Drr 196,197, Gunnar Engstrm 19, Ian Ford 198, Wander S Gurpreet 199, Anders Hamsten 200,201, Laura Heitsch 202, Atsushi Hozawa 203, Laura Ibanez 204, Andreea Ilinca 95,96, Martin Ingelsson 205, Motoki Iwasaki 206, Rebecca D Jackson 207, Katarina Jood 208, Pekka Jousilahti 63, Sara Kaffashian 4,5, Lalit Kalra 209, Masahiro Kamouchi 210, Takanari Kitazono 211, Olafur Kjartansson 212, Manja Kloss 213, Peter J Koudstaal 214, Jerzy Krupinski 215, Daniel L Labovitz 216, Cathy C Laurie 118, Christopher R Levi 217, Linxin Li 218, Lars Lind 219, Cecilia M Lindgren 220,221, Vasileios Lioutas 222,48, Yong Mei Liu 223, Oscar L Lopez 224, Hirata Makoto 225, Nicolas Martinez-Majander 172, Koichi Matsuda 225, Naoko Minegishi 203, Joan Montaner 226, Andrew P Morris 227,228, Elena Muio 31, Martina Mller-Nurasyid 229,230,231, Bo Norrving 95,96, Soichi Ogishima 203, Eugenio A Parati 232, Leema Reddy Peddareddygari 56, Nancy L Pedersen 98,233, Joanna Pera 129, Markus Perola 63,234, Alessandro Pezzini 235, Silvana Pileggi 236, Raquel Rabionet 237, Iolanda Riba-Llena 30, Marta Ribass 238, Jose R Romero 185,48, Jaume Roquer 239,240, Anthony G Rudd 241,242, Antti-Pekka Sarin 243,244, Ralhan Sarju 199, Chloe Sarnowski 47,48, Makoto Sasaki 245, Claudia L Satizabal 185,48, Mamoru Satoh 245, Naveed Sattar 246, Norie Sawada 206, Gerli Sibolt 172, sgeir Sigurdsson 247, Albert Smith 248, Kenji Sobue 245, Carolina Soriano-Trraga 240, Tara Stanne 249, O Colin Stine 250, David J Stott 251, Konstantin Strauch 229,252, Takako Takai 203, Hideo Tanaka 253,254, Kozo Tanno 245, Alexander Teumer 255, Liisa Tomppo 172, Nuria P Torres-Aguila 31, Emmanuel Touze 256,257, Shoichiro Tsugane 206, Andre G Uitterlinden 258, Einar M Valdimarsson 259, Sven J van der Lee 16, Henry Vlzke 255, Kenji Wakai 253, David Weir 260, Stephen R Williams 261, Charles DA Wolfe 241,242, Quenna Wong 118, Huichun Xu 191, Taiki Yamaji 206, Dharambir K Sanghera 125,169,170, Olle Melander 19, Christina Jern 171, Daniel Strbian 172,173, Israel Fernandez-Cadenas 31,30, W T Longstreth, Jr 174,65, Arndt Rolfs 175, Jun Hata 107, Daniel Woo 82, Jonathan Rosand 12,13,14, Guillaume Pare 15, Jemma C Hopewell 176, Danish Saleheen 177, Kari Stefansson 11,178, Bradford B Worrall 179, Steven J Kittner 37, Sudha Seshadri 180,48, Myriam Fornage 74,181, Hugh S Markus 3, Joanna MM Howson 28, Yoichiro Kamatani 6,182, Stephanie Debette 4,5, Martin Dichgans 1,183,184.

1 Institute for Stroke and Dementia Research (ISD), University Hospital, LMU Munich, Munich, Germany

2 Centre for Brain Research, Indian Institute of Science, Bangalore, India

3 Stroke Research Group, Division of Clinical Neurosciences, University of Cambridge, UK

4 INSERM U1219 Bordeaux Population Health Research Center, Bordeaux, France

5 University of Bordeaux, Bordeaux, France

6 Laboratory for Statistical Analysis, RIKEN Center for Integrative Medical Sciences, Yokohama, Japan

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Genetically proxied therapeutic inhibition of antihypertensive drug targets and risk of common cancers: A mendelian randomization analysis - PLoS...

News Release

Monday, January 24, 2022

NIH-funded preclinical study identifies potential therapeutic approach to treat ALS.

Using an experimental drug, researchers were able to suppress a mutated amyotrophic lateral sclerosis (ALS) gene. Studies in mice demonstrate that the therapy could show promise in treating rare, aggressive forms of ALS caused by mutations in the fused in sarcoma (FUS) gene. The study, published in Nature Medicine, was funded in part by the National Institute for Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health.

The study models how promising gene-targeting therapies can move expeditiously from pre-clinical development to clinical testing, said Amelie Gubitz, Ph.D., program director at NINDS. There is a desperate need for innovative approaches to treating ALS.

ALS, also known as Lou Gehrigs disease, is a fatal neurological disorder that causes the degeneration of motor neurons in the brain and spinal cord. People with ALS rapidly lose muscle strength and eventually their ability to move, swallow, and breathe. Most cases of ALS are sporadic, but at least 10% of cases are familial, or due to mutations in various genes. Mutations in the gene FUS cause severe forms of ALS, referred to as FUS-ALS, including a rare type that begins in adolescence or young adulthood.

In the study, Neil Shneider, M.D., Ph.D., the Claire Tow Associate Professor of Motor Neuron Disorders and Director of the Eleanor and Lou Gehrig ALS Center at Columbia University, New York City, and his team delayed the onset of motor neuron degeneration in mice by using an antisense oligonucleotide drug designed to silence FUS by blocking cells from making specific proteins. Following encouraging results, they administered the drug to a patient with ALS.

Compared to normal mice, mice with a mutated FUS gene had higher levels of insoluble FUS and other ALS-related proteins in their brains and spinal cords. Mice with higher doses of mutant FUS in motor neurons experienced rapid neurodegeneration that began early in life, much like FUS-ALS patients.

The study establishes a mouse model that is highly disease-relevant, said Dr. Shneider. In mice, we found that FUS toxicity was due to a gain of function and was dose-dependent, suggesting that we could treat FUS-ALS by silencing the FUS gene.

In 2019 Dr. Shneider met an individual with ALS in search of therapies that may help her disease. Inspired by her story, Dr. Shneider teamed up with a pharmaceutical company to develop a personalized therapy designed to target the FUS mutation.

In mice, injecting a single dose of the drug into the ventricles, fluid-filled spaces surrounding the brain, delayed the onset of inflammation and motor neuron degeneration by six months. The drug also knocked down levels of FUS by 50% to 80% in the brain and spinal cord. Following drug administration, insoluble forms of other ALS-associated proteins were also cleared.

Under a compassionate use protocol reviewed by the U.S. Food and Drug Administration, Dr. Shneider administered the drug to the patient it had been designed for. The patient received repeated infusions of the drug into her spinal canal for 10 months. During the treatment, the patients rate of motor function deterioration slowed. The patient tolerated the treatment well and there were no medically adverse effects.

The study is an example of a precision medicine, bench-to-bedside effort, said Dr. Shneider. We began with the mouse model to establish a rationale for the drug, conducted efficacy studies in the mouse, moved the drug into a human, and collected valuable data that was ultimately used to support a larger Phase 3 clinical trial.

Treatment began more than six months after clinical onset, by which time the disease had already significantly advanced. As is typical with juvenile-onset FUS-ALS, the disease progressed rapidly, and the patient died from complications of the disease.

By studying the patients brain and spinal cord tissue, researchers found that the drug silenced FUS throughout the nervous system and reversed the toxic nature of FUS and other disease-related proteins. Compared to tissue from untreated FUS-ALS patients and healthy controls, FUS protein aggregatesa pathological hallmark of this form of ALSwere sparse, suggesting that they may have been cleared by the drug. Tissue samples were provided by the New York Brain Bank of Columbia University.

The protein made from the FUS gene has been shown to be important for various cellular processes. Prior studies in mice suggest that FUS mutations result in the production of an abnormal protein that forms clumps, or aggregates, leading to motor neuron damage. By targeting the faulty gene in a way that suppresses toxic FUS activity, gene silencing products like the antisense oligonucleotide drug could potentially reduce or prevent disease progression.

The results were used to support a clinical trial testing the drug in patients with FUS-ALS (NCT04768972).

This study was supported by grants from the NIH (NS106236), Nancy Perlman, Tom Klingenstein, and the Judith and Jean Pape Adams Charitable Foundation.

NINDSis the nations leading funder of research on the brain and nervous system.The mission of NINDS is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Korobeynikov, V.A., et al. Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis. Nature Medicine, January 24, 2022. DOI: 10.1038/s41591-021-01615-z

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Link:
Silencing a faulty gene may uncover clues to rare forms of ALS - National Institutes of Health

I was like, wait a minute. Where is this information going? How will it be utilized? she later asked. I dont know if there are any implications for life and health insurance for me or my family, but why risk it?

What should happen when researchers, while sequencing a participants DNA as part of a large study, discover gene variants that increase the risk for conditions that might be prevented with medical treatment or surveillance? Some researchers believe they have an obligation to find the participants often years after they provided a DNA sample contact them, and tell them what they have found. But, some research subjects, like Ms. Konstadt, feel they have a right not to know. Is it ethical for doctors to let them insist they can opt out of learning more without first knowing the particular risk they are facing?

For Dr. Robert Green, an investigator for the biobank with Ms. Konstadts DNA, the Mass General Brigham Biobank, and author of a recent paper about its policies, the answers are clear. The consent form for the biobank tells participants that if the researchers find a worrisome variant, and if there is an intervention that can reduce risk, the participants will be contacted. There will be seven attempts to reach participants calls and letters before the team gives up.

We are offering the information, not forcing participants to accept it, said Dr. Green, who is also a geneticist and professor of medicine at Harvard. If you dont answer the phone or decide when offered that you dont want to hear anything more, or even hang up on us when we call, then thats your choice.

Dr. Green and his colleagues point out that the possibility of being contacted was in the consent form:

While you should not expect to receive any results from your participation in this research, if experts from the Biobank decide that research results from your sample are of high medical importance, we will attempt to contact you. In some situations, follow up testing might be needed in a certified clinical lab. You and your medical insurer may be responsible for the costs of these tests and any follow up care, including deductibles and co-payments.

But some, like Ms. Konstadt, did not notice that clause when signing the form.

Out of more than 36,000 participants, whose DNA its researchers analyzed, the Mass General Brigham Biobank found 425 with worrisome gene variants whose effects could be ameliorated by depending on the genes enhanced cancer surveillance or aggressive medical treatments to lower cholesterol levels, for example.

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Their DNA Hides a Warning, but They Dont Want to Know What It Says - The New York Times

-If successful, BridgeBios investigational gene therapy BBP-631 would be the first therapy for CAH to restore the bodys hormone and steroid balance by enabling people with CAH to make their own cortisol and aldosterone

-Initial Phase 1/2 data readout anticipated in the second half of 2022

-BridgeBios gene therapy portfolio also includes a clinical stage program for Canavan disease and preclinical programs for classic galactosemia, TMC1 hearing loss, tuberous sclerosis types 1 and 2, cystinuria and a genetic dilated cardiomyopathy

PALO ALTO, Calif., Jan. 27, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first patient has been dosed in ADventure, its Phase 1/2 clinical trial of BBP-631, an investigational adeno-associated virus (AAV) 5 gene therapy for the treatment of classic congenital adrenal hyperplasia (CAH). CAH is one of the most prevalent genetic diseases, with more than 75,000 cases estimated in the United States and European Union.

Dosing the first patient in our CAH trial is a landmark milestone and we are grateful for the support from the medical and patient communities who helped us reach this moment. For more than 50 years people living with CAH have had the same limited standard of care lifelong daily steroid replacement treatment. Our investigational gene therapy offers patients a potential single-dose intervention designed to restore their bodys hormone and steroid balance by making their own cortisol and aldosterone, said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy. This is the second gene therapy trial we have initiated in less than four months, and we are excited to advance this trial and our other gene therapy programs in the hope of improving patients lives.

Adults, children and families affected by CAH experience the daily burden of the disease and often, unfortunately, the side effects and morbidities associated with the current treatment regimens. As an endocrinologist, its incredibly exciting to reimagine a new approach to treating this disease, added Adam Shaywitz, M.D., Ph.D., chief medical officer at BridgeBio Gene Therapy. Adrenas Therapeutics, the affiliate company of BridgeBio focused on developing BBP-631 for CAH, is part of BridgeBio Gene Therapys portfolio.

The Phase 1/2 open-label study is designed to evaluate the safety, tolerability and pharmacodynamic activity of the companys AAV5 gene therapy, BBP-631, in adults with classic CAH. In the initial dose-finding phase of the study, each subject will receive a single intravenous (IV) infusion. The primary outcomes of the study are safety, as well as change from baseline in endogenous cortisol levels which BBP-631 has the unique potential to restore. Change from baseline in steroid biomarkers for hydroxyprogesterone (17-OHP) levels and androstenedione (A4) levels will also be measured. Preclinical proof-of-concept data have shown the approach provides efficient and persistent delivery of functional 21-hydroxylase (21-OH) enzyme to the adrenal gland.

We are honored to be the first site to administer gene therapy in a patient with CAH as it is a potential game-changing treatment option that targets the disease at its source, said Kyriakie Sarafoglou, M.D., associate professor and director of the Center for Congenital Adrenal Hyperplasia at the University of Minnesota. We are eager to see whethergene therapy can restore endogenous cortisol production, and also look forward to exploring its effect on thephysiological mechanisms that regulate the hypothalamic-pituitary-adrenal axis including circadian and ultradian hormonal profiles.

BridgeBios investigational AAV5 gene therapy for CAH is one of the Companys 14 programs that are in the clinic or commercial setting for patients living with genetic diseases and cancers. Initial Phase 1/2 data readouts of the Companys AAV5 gene therapy for CAH and the Companys AAV9 gene therapy for Canavan disease are expected in the second half of 2022.

For more information about the ADventuretrial, visit ClinicalTrials.gov (NCT04783181).

About BBP-631BBP-631 is an AAV5 gene therapy developed to treat CAH due to 21-hydroxylase deficiency at its source. BBP-631 is designed to deliver a functional copy of the 21-hydroxylase gene and has been shown through multiple preclinical studies to result in efficient and persistent delivery to the adrenal gland, where hormones are naturally made. If successful, BBP-631 may restore the bodys hormone and steroid balance by enabling people with CAH to naturally make their own cortisol and aldosterone. It could also allow for people with CAH to eliminate or significantly reduce their daily glucocorticoid or mineralocorticoid doses, which is the current standard of care for patients.

About Congenital Adrenal Hyperplasia (CAH)Affecting approximately 75,000 people in the United States and European Union, CAH is a group of genetic disorders that affect the adrenal glands, which is caused by a mutation in the gene encoding for 21-hydroxylase, an enzyme essential for making the hormones cortisol and aldosterone which are critical for various physiologic functions. Cortisol is necessary for the body to respond to injury, stress or illness, and aldosterone is required to maintain proper blood pressure and sodium levels. Unable to produce cortisol and aldosterone, people with classic CAH cannot mount the healthy physiological response to stressors, such as illnesses, that allows their heart, lungs, kidneys and other organs to compensate for the stress, which can be life-threatening. These adrenal crises can be particularly dangerous for young children.

About BridgeBio Pharma, Inc.BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBios pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the companys first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

BridgeBio Pharma, Inc. Forward-Looking StatementsThis 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 and success of BridgeBios Phase 1/2 clinical trial of BBP-631 for the treatment of CAH, expectations, plans and prospects regarding BridgeBios regulatory approval process for BBP-631, the ability of BBP-631 to treat CAH in humans, and the timing and success of initial top-line Phase 1/2 date of BBP-631, 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, BridgeBios ability to continue and complete its Phase 1/2 clinical trial of BBP-631 for the treatment of CAH, past data from preclinical studies not being indicative of future data from clinical trials, BridgeBios ability to advance BBP-631 in clinical development according to its plans, the ability of BBP-631 to treat CAH, the ability of BBP-631 to retain Fast Track Designation, Rare Pediatric Drug Designation, and Orphan Drug Designation from the U.S. Food and Drug Administration and Orphan Drug Designation from the European Medicines Agency, risks inherent in developing therapeutic products, the success, cost, and timing of the Companys product candidate research and development activities and ongoing and planned preclinical studies and clinical trials, the success and timing of preclinical study and clinical trial results, the success of its clinical trial designs, the fact that successful preliminary preclinical study or clinical trial results may not result in future clinical trial successes and/or product approvals, trends in the industry, the legal and regulatory framework for the industry, the success of current and future agreements with third parties in connection with the development or commercialization of the Companys product candidates and FDA-approved products, the size and growth potential of the market for the Companys product candidates and FDA-approved products, the Companys ability to access additional funding upon achievement of portfolio milestones, the accuracy of the Companys estimates regarding expenses, future revenue, future expenditures and needs for and ability to obtain additional financing, the Companys ability to be a sustainable genetic medicine innovation engine and to build the next great genetic medicine company, the Companys ability to obtain and maintain intellectual property protection for its product candidates and approved products, the competitive environment and clinical and therapeutic potential of the Companys product candidates and FDA-approved products, the Companys international expansion plans, and potential adverse impacts due to the global COVID-19 pandemic such as delays in clinical trials, preclinical work, overall operations, regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; as well as those set forth in the Risk Factors section of BridgeBios most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent SEC filings, which are available on the SECs website atwww.sec.gov.Moreover, BridgeBio 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.

BridgeBio Media Contact:Grace RauhGrace.rauh@bridgebio.com(917) 232-5478

BridgeBio Investor Contact:Katherine Yaukatherine.yau@bridgebio.com(516) 554-5989

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BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH) -...

Scientists at the University of Virginia (UVA) School of Medicine have developed a promising gene therapy to target the mutation behind a severe form of epilepsy.

The new Dravet syndrome treatment could help improve and extend the lives of people with the rare but debilitating disease.

The challenge: People born with Dravet syndrome start experiencing seizures when theyre still infants. Seizures continue throughout their lives and often lead to a host of health problems severe developmental delays, speech impairments, intellectual disability, and movement difficulties.

There is no cure for Dravet syndrome, and 10-20% of children born with it die before reaching adulthood.

Certain diets, medications, and therapies can help reduce the frequency and severity of seizures, but patients still require constant, lifelong care.

Between 10% and 20% of children born with Dravet syndome die before reaching adulthood.

The idea: In more than 80% of cases, people with Dravet syndrome have a mutation in a particular gene (called SCN1A) that results in reduced production of a critical protein in the brain.

This leads to a shortage of that protein in specialized brain cells, called interneurons, that protect against seizures.

No existing Dravet syndrome treatment directly addresses the underlying cause of the disease so the UVA team decided to make one that does.

The Dravet syndrome treatment: Dravet is usually caused by a single random mutation, but people generally carry two copies of every gene. The researchers aimed to use the second intact gene to treat the disease.

The treatment completely prevented seizures and premature death in infant mouse models of Dravet.

Using an approach they call Targeted Augmentation of Nuclear Gene Output (TANGO), the scientists developed a Dravet syndrome treatment that prompts the intact gene to increase protein production.

When tested in mouse models of Dravet syndrome, the treatment completely prevented seizures and premature death in infant mice.

The animals interneurons were more responsive, more active, and better able to do their jobs, according to a press release.

The big picture: Therapies that work in mice often fail in humans, but early results in human trials suggest this Dravet syndrome treatment has a shot.

More than 70% of children with Dravet in an ongoing phase 1/2A study experienced a reduction in seizure frequency following treatment, according to Stokes Therapeutics, the company developing the drug.

That study was small, with just 21 participants so far, and these are interim results, so its still too early to say whether the therapy (called STK-001) will ultimately make it to patients.

Still, if the results hold up in larger trials, the new treatment could help people with Dravet syndrome live longer, healthier lives.

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Gene therapy shows promise at treating severe form of epilepsy - Freethink