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Archive for the ‘Gene therapy’ Category

The PBS documentary The Gene showcases genetics promise and pitfalls – Science News

Saturday, April 11th, 2020

The genetic code to alllife on Earth, both simple and complex, comes down to four basic letters: A, C,T and G.

Untangling the role thatthese letters play in lifes blueprint has allowed scientists to understandwhat makes everything from bacteria to people the way they are. But as researchershave learned more, they have also sought ways to tinker with this blueprint,bringing ethical dilemmas into the spotlight. The Gene, a two-part PBS documentary from executive producer Ken Burnsairing April 7 and 14, explores the benefits and risks that come withdeciphering lifes code.

The film begins with oneof those ethical challenges. The opening moments describe how biophysicist HeJiankui used the gene-editing tool CRISPR/Cas9 to alter the embryos of twin girls who were born in China in 2018 (SN: 12/17/18). Worldwide, criticscondemned the move, claiming it was irresponsible to change the girls DNA, asexperts dont yet fully understand the consequences.

This moment heraldedthe arrival of a new era, narrator David Costabile says. An era in whichhumans are no longer at the mercy of their genes, but can control and evenchange them.

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The story sets the stagefor a prominent theme throughout the documentary: While genetics holdsincredible potential to improve the lives of people with genetic diseases,there are always those who will push science to its ethical limits. But thedriving force in the film is the inquisitive nature of the scientistsdetermined to uncover what makes us human.

The Gene, based on the book of the same name by Siddhartha Mukherjee (SN:12/18/16), one of the documentarys executive producers, highlights many ofthe most famous discoveries in genetics. The film chronicles Gregor Mendels classicpea experiments describing inheritance and how experts ultimately revealed inthe 1940s that DNA a so-called stupid molecule composed of just four chemicalbases, adenine (A), thymine (T),cytosine (C) and guanine (G) is responsible for storing geneticinformation. Historical footage, inBurns typical style, brings to life stories describing the discovery of DNAshelical structure in the 1950s and the success of the Human Genome Project indecoding the human genetic blueprint in 2003.

The film also touches ona few of the ethical violations that came from these discoveries. The eugenicsmovement in both Nazi Germany and the United States in the early 20th century aswell as the story of the first person to die in a clinical trial for genetherapy, in 1999, cast a morbid shadow on the narrative.

Interwoven into thistimeline are personal stories from people who suffer from genetic diseases.These vignettes help viewers grasp the hope new advances can give patients asexperts continue to wrangle with DNA in efforts to make those cures.

In the documentarysfirst installment, which focuses on the early days of genetics, viewers meet a family whose daughter is grappling with arare genetic mutation that causes her nerve cells to die. The family searchesfor a cure alongside geneticist Wendy Chung of Columbia University. The secondpart follows efforts to master the human genome and focuses on AudreyWinkelsas, a molecular biologist at the National Institutes of Health studyingspinal muscular atrophy, a disease she herself has, and a family fighting tosave their son from a severe form of the condition.

For science-interested viewers, the documentary does not disappoint. The Gene covers what seems to be every angle of genetics history from the ancient belief that sperm absorbed mystical vapors to pass traits down to offspring to the discovery of DNAs structure to modern gene editing. But the stories of the scientists and patients invested in overcoming diseases like Huntingtons and cancer make the film all the more captivating.

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The PBS documentary The Gene showcases genetics promise and pitfalls - Science News

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SNUH team finds causal gene of inherited retinal disorder – Korea Biomedical Review

Saturday, April 11th, 2020

A group of Korean researchers said they have confirmed a gene responsible for inherited retinal disorders (IRD) among Koreans.

IRD is a combination of several rare diseases that usually develops at a young age and progresses slowly over the lifetime. The patients gradually lose their sight, and most of them eventually lose their vision entirely due to continuous retinal cell degeneration.

The Seoul National University Bundang Hospital (SNUBH) Department of Ophthalmology and Seoul National University Hospital (SNUH) Department of Laboratory Medicine jointly conducted the study.

Currently, antioxidant therapy, artificial retinal transplantation, and stem cell therapy are being used to treat the disorder regardless of mutations, but the only viable treatment is gene therapy. Even when gene therapy is possible, only less than 1 percent of all IRD patients can be treated with it.

In the West, genetic abnormalities of these retinal diseases have been studied and known well. However, researches on Korean cases are still lacking, and the joint research team tackled the subject to find the causative gene for IRDs with 86 domestic patients, the team said in a news release on Wednesday.

The team studied and identified the gene responsible for the disorders by using the latest technique of gene analysis with the most number of patients who have been reported so far.

The study revealed that only 44 percent of the patients, 38 out of 86, possessed the causal gene for IRDs. Even among the patients with retinitis pigmentosa, the most common disorder among the IRDs, only 41 percent had the causative gene.

The causative genes could be quite diverse even in the same disorder. The patients can find a responsible gene only when they receive genetic counseling very actively and can receive gene counseling, too, the research team explained.

Differences were found in the type and frequency of causal gene mutations between Korean and Western cases. However, there were similarities between those of Korean and other Asian nations, including Japan.

The research and diagnosis environment for IRDs has been very poor until now, and our study has significance as a basic data for diagnosis and treatment for Korean patients with IRDs, SNUH Department of Ophthalmology Professor Woo Se-joon said.

Patients need to receive causal gene tests actively to provide the domestic medical communities with sufficient data, and a list of patients who can be treated. By doing so, clinical trials and new drug development in gene therapy will progress smoothly, he added.

Previously, only a few hospitals could diagnose the causative gene for IRDs and afford to test and treat IRD patients due to the high cost of genetic testing. Recently, however, the chance of diagnosis has increased as more hospitals are conducting genetic tests amid the lowered cost thanks to insurance benefits.

Also, the therapeutic opportunity for IRD patients is likely to get broadened, as the retinal pigment epithelium 65 gene (RPE65) therapy won approval from the U.S. Food and Drug Administration for the first time in the world.

Although we do not have a clear way to prevent IRDs at the moment, the prediction of risk and their early detection are developing through the discovery of family history and causative genes, Professor Woo said. Early diagnosis can prevent impaired vision by gene therapy and vision correction, and the patients will be able to choose appropriate jobs with social activities.

Also taking part in the research team were Professors Joo Kwang-sic and Park Kyu-hyung of SNUBH and Professors Seong Moon-woo and Park Sung-sup of SNUH.

The results of this study were published in the Journal of Korean Medical Science.

shim531@docdocdoc.co.kr

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Enochian Biosciences Announces Scientific Presentations at the American Society of Cell and Gene Therapy (ASCGT) Annual Meeting – Yahoo Finance

Saturday, April 11th, 2020

LOS ANGELES, April 09, 2020 (GLOBE NEWSWIRE) -- Enochian Biosciences, a company focused on gene-modified cellular therapy in infectious disease and cancer,announces that all three submissions to the 23rd Annual Meeting of the ASCGT were accepted for presentation. The Conference will be held virtually May 12-15, 2020.

Dr. Serhat Gumruku, inventor and co-founder, will have two presentations: Hijacking HBV Pol to Induce Apoptosis Specifically in Infected HepatocytesIn Vivo: A Novel Approach for Potential Treatment or Cure; and Increased Engraftment of Gene Modified HSPCs Overexpressing ALDH1 UsingIn VivoChemoselection. Dr. Ramesh Halder, Senior Staff Scientist, will present: Gene Modified CD34+Cells With Increased ALDH1 Expression ConfersIn VitroProtection Against Cyclophosphamide.

Forward-Looking StatementsStatements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties, including but not limited to the success or efficacy of our pipeline. All statements other than historical facts are forward-looking statements, which can be identified by the use of forward-looking terminology such as believes, plans, expects, aims, intends potential, or similar expressions. Actual events or results may differ materially from those projected in any of such statements due to various uncertainties, including as set forth in Enochians most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Enochian undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

Contact: ir@enochianbio.com

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Enochian Biosciences Announces Scientific Presentations at the American Society of Cell and Gene Therapy (ASCGT) Annual Meeting - Yahoo Finance

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Gene Therapy Market 2020: Competitive Landscape and Recent Industry Development Analysis by Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure…

Saturday, April 11th, 2020

Global Gene Therapy Market report underlines the specific study of the Biotechnology industry which explains what the market definition, classifications, applications, engagements, and global industry trends are. The market data analyses and evaluated in this Gene Therapy market report makes you achieve the business goals and objectives in preset time frame. It highlights a wide-ranging evaluation of the markets growth prospects and restrictions. This report is very useful to all sizes of business which makes it simpler to take informed decisions regarding different facets of industry. Gene Therapy market report truly acts as a backbone to the business.

A finest market research report acts as an innovative solution for the businesses in todays changing market place and hence this Gene Therapy report is generated. Key players in the market, major collaborations, merger and acquisitions along with trending innovation and business policies are also reviewed in this Gene Therapy report. The report has a list of key competitors with the required specifications and also provides the strategic insights and analysis of the key factors influencing the industry. The data and information of the Gene Therapy report not only helps business make data-driven decisions but also guarantees maximum return on investment (ROI).

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MARKET INTRODUCTION

Gene therapy is introduction of DNA into a patient to treat a genetic disease or a disorder. The newly inserted DNA contains a correcting gene to correct the effects of disease causing mutations. Gene therapy is promising treatment for the genetic diseases and also includes cystic fibrosis and muscular dystrophy. Gene therapy is suitable treatment for the infectious diseases, inherited disease and cancer.

Key Competitors In Market are Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure N.V., AveXis, Vineti, Solid Biosciences., Spark Therapeutics, Inc., CHIMERON BIO, RENOVA THERAPEUTICS, and HORAMA S.A.

MARKET SCOPE

The Global Gene Therapy Market Analysis to 2025 is a specialized and in-depth study of the gene therapy industry with a focus on the global market trend. The report aims to provide an overview of global gene therapy market with detailed market segmentation by cell type, application, and geography. The global gene therapy market is expected to witness high growth during the forecast period. The report provides key statistics on the market status of the leading market players and offers key trends and opportunities in the market.

Market segmentation:

By Cell Type (Somatic Gene Therapy, Germline Gene Therapy);

By Application (Genetic Disorder, Cancer, Neurological Disorder, and Others)

By Geography North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. And 13 countries globally along with current trend and opportunities prevailing in the region.

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Gene Therapy Market 2020: Competitive Landscape and Recent Industry Development Analysis by Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure...

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UGA’s Landon Clark named 2020 Goldwater Scholar – University of Georgia

Saturday, April 11th, 2020

University of Georgia Honors student Landon Clark is among 396 undergraduates across the nation to be recognized as Barry Goldwater Scholars, earning the highest undergraduate award of its type for the fields of the natural sciences, mathematics and engineering.

Clark, from Leesburg, is a third-year CURO Honors Scholar who is triple majoring in biochemistry and molecular biology, genetics and biology.

Since 1995, 61 UGA students have received the Goldwater Scholarship, which recognizes exceptional sophomores and juniors across the United States.

The University of Georgia is proud of Landon and pleased that he is extending our record of success in this prestigious competition, said President Jere W. Morehead. Already as an undergraduate student, he is helping UGA fulfill our commitment to conduct research that helps people lead longer, healthier lives.

This year, recipients were selected from a field of more than 5,000 college sophomores and juniors who were nominated by 461 academic institutions nationwide. Each awardee will receive up to $7,500 toward the cost of tuition, fees, books, and room and board.

Of this years Goldwater Scholars, 50 are mathematics and computer science majors, 287 are majoring in the natural sciences and 59 are majoring in engineering.

I am thrilled for Landon, who is absolutely one of our very best students, said David S. Williams, associate provost and director of the Honors Program, who serves as the UGA campus faculty representative for the Goldwater Scholarship. As a CURO Honors Scholar, Landon was recruited to come to UGA because of his promise as a researcher. This Goldwater award and recognition proves that his hard work is paying off. I look forward to watching his career continue to blossom in the future.

Clarks long-term plans include pursuing an MD/PhD in immunology, performing translational research on immune diseases using gene and immunotherapy techniques, and teaching at a university. As a translational researcher specializing in immunology, he plans to use gene therapy techniques to treat and cure immune disorders in humans.

Since his first weeks as a UGA freshman in August 2017, Clark has conducted research in the lab of Michael Terns, Distinguished Research Professor of Biochemistry and Molecular Biology. He researches the complex processes of CRISPR-Cas immune systems through a model archaeal organism, Pyrococcus furiosus. Now a junior, he has collaborated on three different projects, co-authored a paper, and mentors other undergraduates in this field.

Last summer, he worked in the translational research lab of H. Trent Spencer, a professor of pediatrics and director of the gene and cell therapy program at Emorys Winship Cancer Institute. There, Clark researched immune diseases and potential gene therapy techniques. In 2017, he also conducted research on sociological variables influencing educational inequality with Dawn Robinson, a professor of sociology at UGA.

Clark is a Crane Leadership Scholar, director of academic outreach for the Student Government Association, director of internal communications for UGA Red Cross, an exam writer for UGA Science Olympiad Outreach, co-president of the Biochemistry Undergraduate Society, co-president of the Omicron Delta Kappa National Honor Society, and treasurer of the Honors Program Student Council. He also volunteers in the neuroscience and cardiology units at Piedmont Athens Regional Medical Center.

Clark has presented his research at 10 conferences. He also studied neurology and cancer biology through UGAs Studies Abroad Program in Cortona, located in Tuscany, Italy.

The scholarship honoring Sen. Barry Goldwater was created to encourage outstanding students to pursue careers in the fields of mathematics, natural sciences and engineering. Since its first award in 1989, the Barry Goldwater Scholarship and Excellence in Education Foundation has bestowed 8,628 scholarships worth more than $68 million.

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UGA's Landon Clark named 2020 Goldwater Scholar - University of Georgia

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Research on Hemophilia Gene Therapy Market (impact of COVID-19) 2020-2026 Spark Therapeutics, Freeline Therapeutics, Sangamo Therapeutics, Ultragenyx…

Saturday, April 11th, 2020

Detailed market survey on the Global Hemophilia Gene Therapy Market Research Report 2020-2026. It analyses the vital factors of the Hemophilia Gene Therapy market supported present business Strategy, Hemophilia Gene Therapy market demands, business methods utilised by Hemophilia Gene Therapy market players and therefore the future prospects from numerous angles well. Business associatealysis could be a market assessment tool utilized by business and analysts to grasp the quality of an business. Hemophilia Gene Therapy Market report It helps them get a sense of what is happening in an industry, i.e., demand-supply statistics, Hemophilia Gene Therapy Market degree of competition within the industry, Hemophilia Gene Therapy Market competition of the business with different rising industries, future prospects of the business.

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The Global Hemophilia Gene Therapy Market report is a fully analyzed and intelligent study of the international industry that focuses on a wide range of significant elements such as market size in terms of value and volume, regional growth analysis, competition and segmentation. It is considered as extraordinary findings that accountable to offer insightful details into some essential attributes related to the global Hemophilia Gene Therapy Market 2020. The detailed investigation of this report has been carried out by the list of skillful researchers and investigators with a deep analysis of current industry trends, availability of distinct opportunities, drivers, openings and limitation that influence the Hemophilia Gene Therapy Market on the global scale.

The Global Hemophilia Gene Therapy market worth about xx billion USD in 2020 and it is expected to reach xx billion USD in 2026 with an average growth rate of x%. United States is the largest production of Hemophilia Gene Therapy Market and consumption region in the world, Europe also play important roles in global Hemophilia Gene Therapy market while China is fastest growing region.

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Geographically, Hemophilia Gene Therapy market report is segmented into several key Regions, with production, consumption, revenue. The major regions involved in Hemophilia Gene Therapy Market are (United States, EU, China, and Japan).

Leading companies reviewed in the Hemophilia Gene Therapy report are:

Spark TherapeuticsFreeline TherapeuticsSangamo TherapeuticsUltragenyxuniQureShire PLCBioMarinBioverativ

Hemophilia Gene Therapy Market Product Type Segmentation As Provided Below:The Hemophilia Gene Therapy Market report is segmented into following categories:

The product segment of the report offers product market information such as demand, supply and market value of the product.

The application of product in terms of USD value is represented in numerical and graphical format for all the major regional markets.The Hemophilia Gene Therapy market report is segmented into Type by following categories;Hemophilia AHemophilia B

The Hemophilia Gene Therapy market report is segmented into Application by following categories;Hemophilia A Gene TherapyHemophilia B Gene Therapy

Reportedly, the massive growth graph in the research and development sectors will be liable to generate plenty of excellent opportunities in the upcoming years. The Hemophilia Gene Therapy market is a valuable resource of insightful information for specific business strategists. Apart from this, it also offers an in-depth summary of the Hemophilia Gene Therapy Market along with growth assessment, revenue share, demand & supply data, historical as well as futuristic amount etc. A group of research analysts offers a detailed description of the value chain and its distributors info. Moreover, the Hemophilia Gene Therapy market study report delivers comprehensive information regarding the global industry that enhances the scope, understanding and application of the same.

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Industry analysis, for an entrepreneur or a company, is a method that helps it to understand its position relative to other participants in the Hemophilia Gene Therapy Market. It helps them to identify both the opportunities and threats coming their way and gives them a strong idea of the present and future scenario of the Hemophilia Gene Therapy industry. The key to extant during this changing business setting is to know the variations between yourself and your competitors within the Hemophilia Gene Therapy Market. The deep research study of Hemophilia Gene Therapy market based on development opportunities, growth limiting factors and feasibility of investment will forecast the Hemophilia Gene Therapy market growth.

Finally, The global research document on the Hemophilia Gene Therapy Market discovers a large set of information regarding the competitive business environment and other substantial components. The prime aim of these major competitors is to focus on improved technologies and newer innovations.

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Research on Hemophilia Gene Therapy Market (impact of COVID-19) 2020-2026 Spark Therapeutics, Freeline Therapeutics, Sangamo Therapeutics, Ultragenyx...

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EuroBiotech ReportAZ, GSK in COVID-19, $8B funding call, CRUK cuts and Evotec – FierceBiotech

Saturday, April 11th, 2020

Welcome to the latest edition of our weekly EuroBiotech Report. We start this week with AstraZeneca, which revealed it aims to have a COVID-19-neutralizing antibody in the clinic in the next three to five months. AstraZeneca's compatriot GlaxoSmithKline also shared an update on its COVID-19 activities. GSK added Xiamen Innovax Biotech to the list of organizations it is working with on a COVID-19 vaccine. While those are just two of the many initiatives targeting COVID-19, Wellcome is worried about a funding shortfall. Wellcome wants businesses to commit $8 billion this month to keep programs moving forward quickly. Researchers working outside of COVID-19 are faring worse still, with Cancer Research UK responding to the pandemic with deep cuts. Away from COVID-19, Evotec entered the gene therapy space through a deal with Takeda. And more. Nick Taylor1. AstraZeneca targets summer start for COVID-19 antibody trial

AstraZeneca has outlined its multipronged approach to the development of antibodies against the pandemic SARS-CoV-2 virus. The Big Pharma is applying its own discovery capabilities to the task and helping to evaluate candidates identified by academic partners in China and the U.S.2. GSK allies with Innovax for COVID-19 vaccine R&D project

GlaxoSmithKline has teamed up with Xiamen Innovax Biotech to evaluate a vaccine against the novel coronavirus behind the COVID-19 pandemic. The agreement gives Innovax access to a GSK adjuvant to enhance the immune response triggered by its recombinant protein-based vaccine.3. Wellcome targets $8B raise in weeks to fix COVID-19 funding gap

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

Wellcome is calling on businesses to invest in an $8 billion (7 billion) fund focused on reducing cases of COVID-19 to zero as soon as possible. Almost half the money is earmarked for the development of drugs and vaccines that Wellcome sees as the worlds best exit strategy from the pandemic.4. COVID-19 claims Cancer Research UK funding cuts, forecasting major shortfall

Cancer Research UK (CRUK) said it has been forced to take the difficult decision to make deep funding cuts across its grants and institutes as the fallout from the pandemic continues to affect all areas of biomedical research.

5. Evotec allies with Takeda to move into gene therapy R&D

Evotec has allied with Takeda to expand into gene therapy research. The move sees Evotec establish a 20-person team in Austria, the focal point of Takedas gene therapy operation, and sign up to work on programs for its Japanese partner.And more articles of note>>

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EuroBiotech ReportAZ, GSK in COVID-19, $8B funding call, CRUK cuts and Evotec - FierceBiotech

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Cancer Gene Therapy Market Scope Analysis 2019-2025 – Science In Me

Saturday, April 11th, 2020

Global Cancer Gene Therapy Market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2025, growing at a CAGR of XX% during the period 2019 to 2025.

The report titled Global Cancer Gene Therapy Market is one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global Cancer Gene Therapy market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Cancer Gene Therapy market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Cancer Gene Therapy market is carefully analyzed and researched about by the market analysts.

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The Essential Content Covered in the Global Cancer Gene Therapy Market Report:

In terms of region, this research report covers almost all the major regions across the globe such as North America, Europe, South America, the Middle East, and Africa and the Asia Pacific. Europe and North America regions are anticipated to show an upward growth in the years to come. While Cancer Gene Therapy Market in Asia Pacific regions is likely to show remarkable growth during the forecasted period. Cutting edge technology and innovations are the most important traits of the North America region and thats the reason most of the time the US dominates the global markets. Cancer Gene Therapy Market in South, America region is also expected to grow in near future.

The key players covered in this studyAdaptimmuneBluebird bioCelgeneShanghai Sunway BiotechShenzhen SiBiono GeneTechSynerGene TherapeuticsAltor BioScienceAmgenArgenxBioCancellGlaxoSmithKlineMerckOncoGenex PharmaceuticalsTransgene

Market segment by Type, the product can be split intoOncolytic VirotherapyGene TransferGene-Induced Immunotherapy

Market segment by Application, split intoHospitalsDiagnostics CentersResearch Institutes

Market segment by Regions/Countries, this report coversUnited StatesEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

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Key questions answered in the report

*What will be the market size in terms of value and volume in the next five years?

*Which segment is currently leading the market?

*In which region will the market find its highest growth?

*Which players will take the lead in the market?

*What are the key drivers and restraints of the markets growth?

We provide detailed product mapping and analysis of various market scenarios. Our analysts are experts in providing in-depth analysis and breakdown of the business of key market leaders. We keep a close eye on recent developments and follow latest company news related to different players operating in the global Cancer Gene Therapy market. This helps us to deeply analyze companies as well as the competitive landscape. Our vendor landscape analysis offers a complete study that will help you to stay on top of the competition.

Table of Contents

1 Cancer Gene Therapy Market Overview

1.1 Product Overview and Scope of Cancer Gene Therapy

1.2 Cancer Gene Therapy Segment by Type

1.2.1 Global Cancer Gene Therapy Production Growth Rate Comparison by Type 2020 VS 2026

1.3 Cancer Gene Therapy Segment by Application

1.3.1 Cancer Gene Therapy Consumption Comparison by Application: 2020 VS 2026

1.4 Global Cancer Gene Therapy Market by Region

1.4.1 Global Cancer Gene Therapy Market Size Estimates and Forecasts by Region: 2020 VS 2026

1.4.2 North America Estimates and Forecasts (2015-2026)

1.4.3 Europe Estimates and Forecasts (2015-2026)

1.4.4 China Estimates and Forecasts (2015-2026)

1.4.5 Japan Estimates and Forecasts (2015-2026)

1.5 Global Cancer Gene Therapy Growth Prospects

1.5.1 Global Cancer Gene Therapy Revenue Estimates and Forecasts (2015-2026)

1.5.2 Global Cancer Gene Therapy Production Capacity Estimates and Forecasts (2015-2026)

1.5.3 Global Cancer Gene Therapy Production Estimates and Forecasts (2015-2026)

2 Market Competition by Manufacturers

2.1 Global Cancer Gene Therapy Production Capacity Market Share by Manufacturers (2015-2020)

2.2 Global Cancer Gene Therapy Revenue Share by Manufacturers (2015-2020)

2.3 Market Share by Company Type (Tier 1, Tier 2 and Tier 3)

2.4 Global Cancer Gene Therapy Average Price by Manufacturers (2015-2020)

2.5 Manufacturers Cancer Gene Therapy Production Sites, Area Served, Product Types

2.6 Cancer Gene Therapy Market Competitive Situation and Trends

2.6.1 Cancer Gene Therapy Market Concentration Rate

2.6.2 Global Top 3 and Top 5 Players Market Share by Revenue

2.6.3 Mergers & Acquisitions, Expansion

3 Production Capacity by Region

4 Global Cancer Gene Therapy Consumption by Regions

5 Production, Revenue, Price Trend by Type

5.1 Global Cancer Gene Therapy Production Market Share by Type (2015-2020)

5.2 Global Cancer Gene Therapy Revenue Market Share by Type (2015-2020)

5.3 Global Cancer Gene Therapy Price by Type (2015-2020)

5.4 Global Cancer Gene Therapy Market Share by Price Tier (2015-2020): Low-End, Mid-Range and High-End

6 Global Cancer Gene Therapy Market Analysis by Application

6.1 Global Cancer Gene Therapy Consumption Market Share by Application (2015-2020)

6.2 Global Cancer Gene Therapy Consumption Growth Rate by Application (2015-2020)

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BioIVT Opens New Blood Donor Center to Support Boston-area Research into COVID-19 Therapies, Vaccines and Diagnostics – Bio-IT World

Saturday, April 11th, 2020

Located on the Tufts University Medford, MA campus, this new donor center will enable delivery of fresh blood, leukopaks and buffy coats for COVID-19, cell and gene therapy research within hours of collection

WESTBURY, NY - Apr 6, 2020 - BioIVT, a leading provider of research models and services for drug and diagnostic development, today announced the opening of its new blood donor center on the Tufts University campus in Medford, MA to support academic and pharmaceutical researchers involved in COVID-19, cell and gene therapy research.

BioIVT wants to play a leading role in supporting COVID-19 research efforts and blood donations are a vital resource for the research and development of new therapies, vaccines, and diagnostics. We have many years experience developing blood products, including blood-derived immune cells for cell and gene therapy research, and we want to make that expertise count, said BioIVT CEO Jeff Gatz. Researchers recognize and appreciate BioIVTs rapid response and commitment to high quality, fresh blood products and this new donor center will allow us to offer those attributes and services to additional US clients.

BioIVTs new Boston blood donor center is its seventh. The company has similar facilities located in California, Tennessee and Pennsylvania to serve US clients and in London, UK for EU-based clients.

While the initial focus at our Boston donor center will be on delivering fresh blood, leukopaks and buffy coats within hours of collection, we plan to add more capabilities and donors over time, said Jeff Widdoss, Vice President of Donor Center Operations at BioIVT.

Leukopaks, which contain concentrated white blood cells, are used to help identify promising new drug candidates, assess toxicity levels, and conduct stem cell and gene therapy research. They are particularly useful for researchers who need to obtain large numbers of leukocytes from a single donor.

BioIVT blood products can be supplied with specific clinical data, such as the donor age, ethnicity, gender, BMI and smoking status. Its leukopaks are also human leukocyte antigen (HLA), FC receptor and cytomegalovirus typed. HLA typing is used to match patients and donors for bone marrow or cord blood transplants. FC receptors play an important role in antibody-dependent immune responses.

COVID-19-related Precautions

Blood donor centers are considered essential businesses and will remain open during the COVID-19 quarantine. BioIVT is taking additional safety measures to protect both blood donors and its staff during this difficult time. It has instituted several social distancing measures, including increasing the space between chairs in the waiting room and between donor beds, and limiting the entrance of non-essential personnel. The screening rooms are disinfected between donors and all areas of the center continue to be cleaned at regular intervals.

As soon as each blood donor signs their informed consent form, their temperature is taken. If they have a fever, their appointment is postponed, and they are referred to their physician. Any donor who develops COVID-19 symptoms after donating blood is required to inform the center immediately.

All BioIVT blood collections are conducted under institutional review board (IRB) oversight and according to US Food and Drug Administration (FDA) regulations and American Association of Blood Banks (AABB) guidelines.

Those who would like to donate blood at BioIVTs new Boston-area donor center should call 1-833-GO-4-CURE or visit http://www.biospecialty.com to make an appointment.

Further information about the products available from BioIVTs new donor center can be found at https://info.bioivt.com/ma-donor-ctr-req.

About BioIVT

BioIVT is a leading global provider of research models and value-added research services for drug discovery and development. We specialize in control and disease-state biospecimens including human and animal tissues, cell products, blood and other biofluids. Our unmatched portfolio of clinical specimens directly supports precision medicine research and the effort to improve patient outcomes by coupling comprehensive clinical data with donor samples. Our PHASEZERO Research Services team works collaboratively with clients to provide target and biomarker validation, phenotypic assays to characterize novel therapeutics, clinical assay development and in vitro hepatic modeling solutions. And as the premier supplier of hepatic products, including hepatocytes and subcellular fractions, BioIVT enables scientists to better understand the pharmacokinetics and drug metabolism of newly-discovered compounds and their effects on disease processes. By combining our technical expertise, exceptional customer service, and unparalleled access to biological specimens, BioIVT serves the research community as a trusted partner in elevating science. For more information, please visit http://www.bioivt.com or follow the company on Twitter @BioIVT.

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The Hopes and Challenges of a COVID-19 Vaccine – BioSpace

Saturday, April 11th, 2020

While the world is working to slow the COVID-19 pandemic by social isolation and quarantine, numerous companies globally are working to develop a vaccine against the virus. The most likely timelinesat bestfor a viable vaccine against SARS-CoV-2, the coronavirus causing COVID-19, are 12 to 18 months. And thats if everything goes right.

As a recent article in Bloomberg Businessweek notes, For that to happen in the next year or so, an almost equally implausible set of circumstances has to occur: flawless scientific execution, breakneck trials and a military-style manufacturing mobilization unlike any the pharmaceutical industry has put in place before.

Typically, it takes 10 to 15 years of deliberate and careful work to develop a commercial vaccineand as both the common cold and HIV have demonstrated, sometimes its not possible even then. The infrastructure for the seasonal flu vaccines that come out every year has been in place for decades.

One of the companies that is gaining a lot of attention is Cambridge, Massachusetts-based Moderna. They have a head-start and are using a novel technology to develop their COVID-19 vaccine, mRNA-1273, which is already in clinical trials. mRNA-1273 is a mRNA vaccine that encodes for a prefusion stabilized form of the Spike (S) protein. Whats different about the vaccine is that it is almost like a form of gene therapyit codes for the genetic sequence for the spike protein, and when injected into the body, causes the patients own cells to produce the protein (not the virus), which triggers an immune reaction that will prep itself to battle the virus.

The technology is interesting, but unproven. There are no approved RNA therapies. And Moderna has never brought a product to the market before.

One thing thats of note is how early a start Moderna got on the vaccine. On January 11, 2020, Chinese researchers posted the genetic sequence of the coronavirus, well before most of the world was paying any attention. Moderna scientists had been working on a novel vaccine for a different coronavirus disease and jumped on this sequence and began working on a vaccine against it.

Bloomberg writes, By late February, when President Trump was still downplaying the risk of coronavirus, Modernas scientists had already delivered the first batch of candidate vaccines to researchers at the U.S. National institutes of Health. When the coronavirus was starting to explode in the U.S. in mid-March, the first healthy patient received a dose in a small, government-sponsored safety trial.

Moderna may be the leader, but they are not the only company employing this technology. Pfizer and a few others are as well. Pfizer is working with a German company, BioNTech, hoping to get their experimental RNA vaccine into human trials sometime this month.

Johnson & Johnson is using technology it employed for its experimental Zika and Ebola vaccine. Paris-based Sanofi is modifying technology it uses for its flu shots. There are more than two dozen companies working on coronavirus vaccines that are already in early-stage testing.

There is no precedent for the speed at which we are moving, said Clement Lewin, an associate vice president at Sanofi. I cant think of a parallel, in his thirty years of vaccine work.

The RNA vaccine technology, despite the apparent speed with which Moderna, Pfizer and BioNTech are moving, has some risks. Not as much is known about its efficacy. Early-stage data suggests it should be safe, but there are concerns about unwanted immune responses. And all vaccines run a risk of what is called disease enhancement, where the immune system responds in an unusual way, which can cause the illness to be worse.

It's possible that any vaccines just wont be effective, or the side effects will be such that they wont be considered safe for use.

Scale-up is also a major roadblock. As of April 7, there are 1,362,936 confirmed cases of COVID-19 around the globe. But there are another 8 billion possible people who would possibly benefit from a vaccine.

Under a scenario where Modernas vaccine turns out to be effective and safe, no one has experience in commercial production of RNA vaccines, let alone in the type of scale necessary. The company indicates it has the manufacturing capability to produce millions of doses of the vaccine per month at a factory that was already scaling up for a different vaccine. They are apparently also in discussions with possible partners. But millions is a very long way from producing vaccines for billions of peopleor even a few hundred million people.

If a vaccine were to become available in the next year, an optimistic timeline, the first candidates for the drug would be first-line respondersdoctors, nurses, paramedics, as well as infants, toddlers and pregnant women. Thats a figure that exceeds 26 million people in the U.S. alone.

The next round would be essential personnel and children with preexisting conditions, higher-risk patients and people over the age of 65. Once you add all those, just in the U.S., youre talking more than 100 million who would be considered high-priority for a vaccine.

Mark Feinberg, a former Merck & Co. executive in vaccines who now runs the International AIDS Vaccine Initiative, told Bloomberg that the real question wasnt just efficacy, but how quickly can you ramp up manufacturing to meet global need.

There arent a lot of drugs in the industry that are filled at these scales, period, said Stephen Hoge, president of Moderna. Even large pharma companies dont usually operate on this kind of scale. No one entity or one company will be able to do it by themselves.

With any luck, social distancing and quarantine will slow the spread of the virus. Perhaps the coming summer months will also decrease its spread, although whether there is a seasonality component to the disease is still unknown. This would give biopharma companies a little more room to develop and distribute vaccines or to determine which drugs are best-suited to preventing or treating the disease.

Meanwhile, biopharma companies worldwide are working desperately to develop solutions.

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No sanctions for Novartis as FDA ends review of gene therapy violations – BioPharma Dive

Wednesday, April 1st, 2020

The Food and Drug Administration has quietly closed out an investigation of data violations tied to Novartis' gene therapy Zolgensma, indicating it will not penalize the Swiss drugmaker for submitting manipulated testing results last year.

The violations, documented in an August 2019 inspection of Novartis' AveXis unit, were at the center of a scandal which last year engulfed the pharma and marred a landmark approval for its high-profile spinal muscular atrophy treatment.

In securing the FDA's blessing for the therapy, Novartis had knowingly filed an application containing altered data from preclinical tests in mice. At the time, the FDA warned it would consider civil or criminal penalties against the company, indicating its displeasure that Novartis was aware of the issue but proceeded to ask for approval anyway.

The statement was highly unusual and a signal, former FDA officials said, to other companies in the fast-growing gene therapy field. After seven months of review, however, the FDA has decided against leveling such sanctions, a spokesperson for the agency confirmed to BioPharma Dive.

Instead, the FDA has classified its inspection review as "Voluntary Action Indicated,"or VAI, which means violations were found during the inspection but didn't cross the threshold for regulatory action.

"FDA has completed its review of the information and records of the inspection, the evidence collected, and the firm's responses as well as the corrective actions to the inspectional observations, and the agency has classified the inspection as Voluntary Action Indicated," the FDA spokesperson said.

"Based on its review of the information available, FDA continues to find Zolgensmato be safe and effective for its intended use," added the spokesperson. Throughout its investigation, the FDA maintained that it found no evidence human data were changed.

FDA inspections, such as the one conducted of AveXis last August,result in what are known as Form 483s, technical documents that detail potential violations of the Food Drug and Cosmetic Act. Companies are allowed to respond, after which the FDA decides whether to proceed with a formal warning.

Novartis submitted its responseto the FDA's inspection in late August last year,pledging to retrain employees and improve quality control oversight. The company placed much of the blame for the manipulation on two senior AveXisexecutives, Brian and Allan Kaspar, who were fired in mid August. Through his lawyer, Brian Kasparhas publicly denied any wrongdoing.

Novartis also committed to informing the FDA within five business days of any credible allegations related to data integrity questions with any pending drug application in the future.

Novartis disclosed the FDA's Form 483 classification in a presentation Monday of new clinical data for Zolgensma, briefly noting the FDA's decision.

"We are pleased with this positive outcome and reiterate our firm commitment to data integrity and transparency in our engagements with regulators," a Novartis spokesperson said in a statement.

Zolgensma was only the second gene therapy for an inherited disease to win U.S. approval, showing in clinical studies it could keep alive infants who otherwise were likely to die from spinal muscular atrophy, or SMA. The neuromuscular disease robs newborns of a protein needed for muscle development, usually leading in its most severe form to death or permanent ventilation by age two.

Some 200 infants received Zolgensma in 2019, earning Novartis $361 million last year. The one-time therapy costs $2.1 million per patient, making it the most expensive drug in the world on a per-dose basis.

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2020 Innovations in Bioreactors, Gene Therapy, Vermifiltration & Membranes for Desalination Processes – ResearchAndMarkets.com – Business Wire

Wednesday, April 1st, 2020

DUBLIN--(BUSINESS WIRE)--The "Innovations in Bioreactors, Gene Therapy, Vermifiltration, and Membranes for Desalination Processes" report has been added to ResearchAndMarkets.com's offering.

This edition of the Industrial Bioprocessing TOE features trends and innovations in fixed bed and disposable bioreactors used for processing viral vaccines and for the production of recombinant proteins used in gene therapy. The TOE also provides intelligence on the transformation of cellulose waste and other plant waste in to bio-char, levulinic acid, and formic acid that can be used for production of biofuels. The TOE features innovations based on the novel gasification systems that can be vital in converting biomass in to renewable energy. Additionally, the TOE provides intelligence on the use of innovative membrane technologies that can enhance water permeability and selectivity to generate more freshwater from desalination processes. The TOE also focuses on innovations that are based on the use of modular vermifiltration processes for wastewater treatment, and the generation of bioplastics and biofertilizers from plant and food waste.

The Industrial Bioprocessing TOE provides intelligence on technologies, processes and strategic insights of industries involving bioprocessing, including innovations in the development and production of chemicals, pharmaceuticals, nutraceuticals, alternative fuels, chemical feedstocks, food and beverages, and consumer products.

Key Topics Covered:

Advancements in Bioreactors, Gene Therapy, Vermifiltration, and Membranes for Desalination Processes

For more information about this report visit https://www.researchandmarkets.com/r/xrab0l

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Novartis closes on EU approval for SMA gene therapy Zolgensma – – pharmaphorum

Wednesday, April 1st, 2020

Novartis is on the final straight to approval of its spinal muscular atrophy (SMA) gene therapy Zolgensma in the EU, after getting a recommendation for approval from the CHMP.

The positive opinion gets Novartis marketing application for Zolgensma (onasemnogene abeparvovec) back on track after the company revealed last year that regulators in Europe and Japan had asked for more information on manufacturing of the gene therapy which delayed approval by some months.

At the time, Novartis AveXis unit which developed Zolgensma said the questions raised by the regulators werent related to a data manipulation scandal that emerged with the therapy last year, shortly after it was approved in the US. It was approved in Japan on 19 March.

The delay to its approval in Europe prompted Novartis to set up a managed access programme for some patients, which operated like a lottery and is expected to provide free access to the therapy for 100 patients, although it has come in for criticism as being an unfair way to determine access.

SMA is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. Approximately 550 to 600 infants are born with SMA in Europe each year.

Now, the CHMP has backed conditional approval of Zolgensma for babies and young children with SMA, which could lead to full EMA approval within the next couple of months.

Novartis says it will offer a day one access programme to EU governments and reimbursement agencies to enable immediate access if the EMA gives it the nod.

The recommendation covers children with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1; or patients with 5q SMA with a bi-allelic mutation in SMN1 and up to three copies of the SMN2 gene.

Zolgensma is the worlds most expensive drug, with a price tag of more than $2.1 million per one-shot intravenous infusion, which Novartis argues is justified given the lifelong benefits the therapy can bring.

The company says SMA costs EU healthcare systems between 2.5 million and 4 million within the first 10 years of life alone.

The data scandal doesnt appear to have affected its uptake in the US sales in 2019 reached $361 million, more than half of that total from the last quarter of the year alone.

The quick uptake has come despite the established presence on the market of Biogens Spinraza (nusinersen), an antisense drug given by injection into the spine, which became the first treatment option for SMA when it was approved in 2016 and reached $2.1 billion in sales last year.

GlobalData recently predicted however that Zolgensma will generate global sales of $2.5 billion by 2025, overtaking Spinraza and leading the SMA market.

The CHMP recommendation is another shot in the arm for Novartis gene therapy, coming shortly after it reported additional safety and efficacy data with intrathecal delivery of the gene therapy i.e. delivery into the spinal column rather than intravenously.

Intrathecal administration is considered key to extending the use in therapy for children with milder forms of the disease who tend to exhibit symptoms later in childhood. Novartis also reported new data demonstrating long-term efficacy with Zolgensma, with patients showing benefits for up to five years after they were dosed with the therapy

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VBL and its ‘gene therapy’ cancer treatment are back with a peek at PhIII potential – Endpoints News

Wednesday, April 1st, 2020

Three years after a brain cancer failure sent the company reeling, VBL Therapeutics is touting its first hint of positive Phase III results.

The Israeli biotech announced its lead drug, VB111, met an interim efficacy benchmark in a trial testing it against standard-of-care alone in recurrent, chemotherapy-resistant ovarian cancer. The patients in the treatment arm had a CA-125 response rate a measure of cancer antigen often used as a proxy in ovarian cancer studies and in detecting ovarian cancer at least 10% higher than those in the control, the independent review determined.

The response rate in the first 60 enrolled and evaluable patients was 53%. That indicated a treatment response rate of at least 58% an encouraging number, the company said, because of the CA-125 data from their earlier Phase II trial.

We are very pleased by the outcome of this interim analysis, which demonstrates the potential benefit of VB-111 over standard-of-care in a randomized-controlled study, VBL CEO Dror Harats said. The OVAL Phase 3 interim data are at least as good as the CA-125 response results observed in our VB-111 Phase 2 study.

In that earlier study, which indicated a dose-dependent response, patients who showed a CA-125 response ultimately had an overall survival rate of808 days, versus 351 days for those who did not. The primary endpoint for this study, which is set to be completed in 2022, is overall survival.

The data come two years after VBL announced the combo of VB-111 and Avastin had failed to beat out Avastin alone in a Phase III study of glioblastoma, a notoriously hard-to-treat indication. It was their first Phase III trial after a smattering of Phase II trials in different solid tumors had turned up mixed results.

VB-111 is what the company deems a gene therapy agent. It uses an adenovirus the viral vector traditionally used for gene therapy that carries a gene to cause explosive cell death in tumors, rupturing blood cells. They have anti-inflammatory and other cancer programs in development.

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Affinia Therapeutics Raises $60M in Series A Financing to Advance Rational Design AAV Platform and Transformative Gene Therapies – GlobeNewswire

Wednesday, April 1st, 2020

WALTHAM, Mass., March 31, 2020 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies and a mission to develop transformative medicines for devastating diseases, today announced it has closed an oversubscribed $60 million Series A financing. Seed venture investors F-Prime Capital and New Enterprise Associates (NEA) co-led the round alongside new investor Atlas Venture, with participation from seed investors Alexandria Venture Investments, Lonza and Partners Innovation Fund.

The proceeds will be used to advance the companys platform and develop transformative gene therapies for people affected by muscle and central nervous system (CNS) diseases with significant unmet need.

The company is led by recently appointed Chief Executive Officer Rick Modi, who has a proven track record of building value at companies including AveXis, InterMune, MedImmune and Centocor. Joining the board of directors as part of the financing are Dave Grayzel, M.D., Partner, Atlas Venture; Ed Mathers, General Partner, NEA; and Robert Weisskoff, Ph.D., Partner, F-Prime Capital. Industry veteran and gene therapy leader Sean Nolan will chair the board.

At Affinia Therapeutics, were setting a new standard in gene therapies by leveraging our proprietary platform to methodically engineer novel AAV vectors and gene therapies that have remarkable targeting properties, said Modi, CEO of Affinia. We are pleased to partner with such a distinguished syndicate to advance our platform and investigational product candidates toward the clinic for patients in need.

The companys technology was licensed from Lonza and Massachusetts Eye and Ear. It was developed at the Grousbeck Gene Therapy Center and further advanced under a sponsored research agreement with Lonza led by Luk Vandenberghe, Ph.D., Associate Professor at Mass. Eye and Ear and Harvard Medical School and a co-inventor of AAV9.

In addition to Vandenberghe, joining Affinia Therapeutics as scientific co-founders are Botond Roska, M.D., Ph.D., Director, Institute of Molecular and Clinical Ophthalmology Basel; Aaron Tward, M.D., Ph.D., Assistant Professor, University of California, San Francisco; and Eric Zinn, Ph.D. student, Mass. Eye and Ear and Harvard University. Together, these scientists have authored more than 200 papers and filed more than 20 patents in the field of gene therapy.

Affinia Therapeutics is bringing together complementary expertise allowing us to realize a rational design future for AAV vectors, promoters and other components of gene therapies. By leveraging synthetic and systems biology combined with high-throughput screening and tissue and single-cell resolution, we are aspiring to achieve much-needed improved pharmacological control of this novel modality in medicine, said Vandenberghe, Director, Grousbeck Gene Therapy Center at Mass. Eye and Ear.

The Series A financing comes after Vandenberghe and his team successfully developed AAVSmartLibraries comprising thousands of functional novel AAV vectors. Each vector is uniquely identified, and the libraries can be screened across species for parameters of high interest, including tissue tropism, manufacturing yield and pre-existing immunity. Observations arising from each library screen provide insights into the vectors structure-function, enabling the rational design of novel vectors and gene therapies with remarkably improved properties. Affinia Therapeutics has potentially the worlds largest library of patented functional AAV vectors.

Affinia Therapeutics methodical process for designing and evaluating vectors is a differentiated approach to gene therapy, and the highly experienced leadership team will help carry these discoveries to the development, manufacturing and commercialization of transformative medicines, said Mathers, General Partner at NEA. We are pleased to accelerate the impact of this exciting field.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need.www.affiniatx.com.

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Novartis wins key European recommendation for gene therapy Zolgensma – Reuters

Wednesday, April 1st, 2020

FILE PHOTO: The company's logo is seen at a building of Swiss drugmaker Novartis in Rotkreuz, Switzerland, January 29, 2020. REUTERS/Arnd Wiegmann

ZURICH (Reuters) - Swiss drugmaker Novartis on Friday won a key European recommendation for its gene therapy Zolgensma against spinal muscular atrophy (SMA), clearing a hurdle for $2.1 million per patient treatment for approval in Europe within months.

The European Medicines Agencys (EMA) Committee for Human Medicines (CHMP) recommended conditional approval for Zolgensma for certain patients: those with Type 1 SMA, the severest form of the disease, or for SMA patients with up to three copies of the so-called SMN2 gene, an indicator of the diseases severity.

The EMAs conditional approval is meant to speed up access to medicines for unmet needs, based on less-complete data than normally expected.

Typically the European Commission approves medicines for use shortly after a CHMP recommendation, and Novartis is expecting a decision by June. The medicine, the worlds costliest one-time treatment at its U.S. list price, has already been approved in the United States and Japan.

Novartis is counting on the gene therapy becoming a billion-dollar-per-year seller. Zolgensma is the second treatment for SMA to be approved after Biogens Spinraza three years ago. Roche is expecting its oral drug risdiplam to win U.S. regulators blessing in May.

Reporting by John Miller; Editing by Michael Shields

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Gene Therapy for Frontotemporal Dementia With a GRN Mutation Gets Fast Track Status – Neurology Advisor

Wednesday, April 1st, 2020

The Food and Drug Administration (FDA) has granted Fast Track designation to PR006 (Prevail Therapeutics), an experimental gene therapy, to slow the progression of frontotemporal dementia with a GRN mutation (FTD-GRN).

FTD-GRN is a progressive neurodegenerative disease caused by mutations in the GRN gene. Patients with a GRN mutation have reduced levels of progranulin, a protein critical for lysosomal function, neuronal survival, and normal microglial activities. PR006 delivers a healthy GRN gene using an AAV9 vector to increase progranulin levels in these patients.

The Food and Drug Administration (FDA) recently accepted the Investigational New Drug application for PR006, allowing the Company to proceed with a phase 1/2 trial.

FTD-GRN progresses rapidly and there are currently no therapeutic options available, said Asa Abeliovich, MD, PhD, Founder and CEO of Prevail. We believe PR006 has the potential to fill this unmet medical need and make a significant impact for patients.

The FDA previously granted Orphan Drug designation to PR006 for the treatment of patients with frontotemporal dementia.

For more information visit prevailtherapeutics.com.

This article originally appeared on MPR

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David Hallal bags another whopper round, looking to recruit more cell and gene therapy upstarts to ElevateBio BaseCamp – Endpoints News

Wednesday, April 1st, 2020

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDAs orphan status for remdesivir reserved to encourage the development of rare disease therapies Gilead CEO Daniel ODay had some explaining to do about the companys approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, ODay also reassured some analysts who had been fretting that Chinas quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

ODay emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall at least to the most heavily threatened groups.

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Investigation Begins into Gene Therapy for Osteoarthritis – Everything Horse UK

Wednesday, April 1st, 2020

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A programme investigating the potential of gene therapy for the treatment of osteoarthritis in horses, dogs and cats, has begun at the Royal Veterinary College (RVC), led by Skeletal Regeneration expert, Dr Scott Roberts.

Dr Scott Roberts, who joined the RVC as Senior Lecturer in Translational Skeletal Research in the Department of Comparative Biomedical Sciences, has recently initiated the programme of investigative research.

The research is inspired by Dr Roberts interest in skeletal stem cell signalling and the effect of signal modulation on skeletal tissue regeneration. He initiated this work during his time at KU Leuven (Belgium) but has subsequently researched this topic as Principal Investigator at University College London and as Senior Principal Scientist at UCB Pharma. During his time at UCB, Dr Roberts identified musculoskeletal drug targets and led the subsequent drug discovery projects. His research has resulted in several patent applications on the use of cells and therapeutic antibodies to promote the regeneration of bone and cartilage.

The new gene therapy research programme in osteoarthritis will be undertaken in partnership with the LEP funded Vaccinology and Cell Therapy Hub at the RVC. This will not only provide an environment for further research on regenerative medicine but also the facilities to create reagents for clinical translation.

The Hub with its close connections to both scientists and veterinary clinicians is also well-placed to take the science from bench-to-bedside and facilitate clinical trials in veterinary patients. This will include horses at the Equine Referral Hospital, and dogs and cats at the Queen Mother Hospital for Animals at the RVC.

It is proposed that this treatment would transition into human clinical studies, exemplifying the RVCs commitment to the One Health approach which recognises and facilitates the synergy between animal and human health.

Dr Scott Roberts said:

This research has the potential to change the way that we approach degenerative joint disease and I am delighted to have access to the Vaccinology and Cell Therapy Hub while we undertake this work. We hope that this science will lead to a ground-breaking treatment for osteoarthritis in animals, and eventually humans. We are optimistic about the future of this research, particularly given its contribution to the RVCs One Health ethos. I look forward to taking advantage of the RVCs world-leading facilities, as well as the scientists and clinicians who will help us take this research forward.

Professor Jonathan Elliott, Vice Principal of Research and Innovation at the RVC, said:

We are delighted that Scott Roberts has joined the RVC and will pursue his translational research into novel therapies that stimulate repair of cartilage for osteoarthritis in horses, dogs and people. This appointment builds on the RVCs expertise in Skeletal Biology. Scotts links to the Pharma Industry are very important for his translational science to have impact. His work fits perfectly with the goals of our soon to open Vaccinology and Cell Therapy Hub, made possible by the funding from the Hertfordshire Local Enterprise Partnership.

The research Dr Roberts is undertaking is critical as cartilage has a very poor capacity to repair itself, with cartilage injuries often progressing to osteoarthritis. There is also currently no approved evidence-driven therapy for the treatment of this disease.

In addition to osteoarthritis, Dr Roberts research aims to create regenerative therapeutics for non-healing bone fractures. This is based on a comprehensive understanding of tissue development, as tissue repair is now regarded as a re-emergence of embryonic signalling cascades. Dr Roberts has used knowledge in this area to identify developmentally inspired methodologies to create laboratory grown tissue implants that have the capacity to drive bone fracture repair.

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AVROBIO Outlines Response to COVID-19 and Current Assessment of Business Impact – BioSpace

Wednesday, April 1st, 2020

Our first priority during this unprecedented time is the health of our employees, our patients and their communities, and the employees of our clinical sites, partners and vendors. In accordance with advice from health authorities, AVROBIO has moved quickly to virtual operations, except for essential laboratory work, which continues with additional COVID-19-related safety measures in place, said Geoff MacKay, AVROBIOs president and CEO. We continue to support patient identification efforts across our clinical trials in Canada, Australia and the United States. As the global healthcare community responds to the increase in COVID-19 cases, many hospitals, including our clinical sites, have temporarily paused elective medical procedures, which includes dosing of new patients in clinical trials of our investigative gene therapies. While were fully committed to moving our clinical programs forward, AVROBIO supports this temporary reallocation of resources to ensure hospitals can focus on meeting the needs of patients with COVID-19. We are closely monitoring this rapidly evolving situation and the potential impact on our clinical trial programs and business generally.

Program Updates AVR-RD-01 clinical trials in Fabry disease AVROBIO is conducting two clinical trials for its investigational gene therapy for Fabry disease (AVR-RD-01). Four patients have been dosed in the global Phase 2 trial (FAB-201), which is evaluating treatment-nave patients, and five patients in the fully enrolled Phase 1 (FACTs) investigator-led clinical trial of AVR-RD-01.

AVR-RD-04 Phase 1/2 clinical trial in cystinosis AVROBIOs investigational gene therapy for cystinosis (AVR-RD-04) is being studied in a Phase 1/2 investigational trial in collaboration with the University of California, San Diego (UCSD). The first patient was dosed in October 2019. The single-arm trial is expected to enroll up to six patients.

AVR-RD-02 Phase 1/2 clinical trial in Gaucher disease AVROBIO's investigational gene therapy for Gaucher disease (AVR-RD-02) is being studied in a Phase 1/2 clinical trial to evaluate the safety and efficacy in individuals with Gaucher disease type 1. The global trial is designed to enroll eight to 16 individuals with Gaucher disease type 1 including both those who are treatment-nave and those who are stable on enzyme replacement therapy.

Business Operations On March 10, AVROBIO created an internal, cross-functional COVID-19 Response Team to closely monitor the evolving situation and advise on the companys response. We have implemented a work-from-home policy for all employees excluding those providing essential services, such as our laboratory staff. For those employees, AVROBIO has implemented safety measures designed to comply with applicable federal, state and local guidelines in response to the COVID-19 pandemic. We may be required to take additional actions that impact our operations as required by applicable laws or regulations, or which we determine to be in the best interests of our employees. AVROBIO continues to evaluate the impact of the COVID-19 pandemic on its operations and will provide a further update in conjunction with its first quarter financial results announcement and Quarterly Report on Form 10-Q in May 2020. At this time, all preclinical programs and research activities remain on track, and we do not anticipate any material impact on our regulatory activities.

Financial Position In February 2020, the company raised gross proceeds of $100 million through a follow-on common stock offering. Based on the companys current operating plan, AVROBIO expects its cash and cash equivalents of $187.0 million as of Dec. 31, 2019, together with the net proceeds from the February 2020 follow-on common stock offering, will enable the company to fund its operating expenses and capital expenditure requirements into Q2 2022.

About AVROBIO Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, the expected safety profile of our investigational gene therapies, the potential impact of the COVID-19 outbreak on our clinical trial programs and business generally, as well as our plans and expectations with respect to the timing and resumption of any development activities that may be temporarily paused as a result of the COVID-19 outbreak, and statements regarding our financial and cash position and expected cash runway. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Cautionary Note on Future Updates The statements contained in this press release reflect our current views with respect to future events, which may change significantly as the global consequences of the COVID-19 pandemic rapidly develop. Accordingly, we do not undertake and specifically disclaim any obligation to update any forward-looking statements.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200330005703/en/

Link:
AVROBIO Outlines Response to COVID-19 and Current Assessment of Business Impact - BioSpace

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