Archive for the ‘Gene Therapy Research’ Category

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market - Forecasts from 2019 to 2024" report has been added to ResearchAndMarkets.com's offering.

The global gene therapy market is expected to grow at a CAGR of 16.92% reach $8.748 billion by 2024 from $3.425 billion in 2018.

Gene therapy is an experimental technique that involves the introduction of DNA usually containing a functional gene into a patient for the treatment of genetic disorders. Gene therapy works by replacing, repairing or repressing the mutated gene. Therefore, with the right target and approach, it can address the root cause of a severe disease where known genetic mutations lead to deficient or non-functional protein production or other related problems. The gene therapy may be a promising option for genetic diseases such as muscular dystrophy and cystic fibrosis.

The growth in the market may be attributed to the increase in awareness among the people regarding the benefits of gene therapy coupled with the increased funding activities for research and development of gene therapy by various governments. However, the high cost of gene therapy might restrain the growth of the market during the given forecast period. Problems with suppressing the immune responses due to the introduction of new genes may also hamper the market growth during the given time frame.

The Global Gene Therapy Market - Forecasts from 2019 to 2024 is an exhaustive study that aims to present the key market trends through various chapters focusing on different aspects of the market. The study provides a detailed market overview through the market dynamics sections which detail key market, drivers, restraints, and opportunities in the current market. The report analyzes key opportunity regional markets, and the current technology penetration through lifecycle analysis. The report also analyzes the market through comprehensive market segmentation by Technique, Vector Type, Indication and geography.

Major players in the gene therapy market have been covered along with their relative competitive position and strategies. The report also mentions recent deals and investments of different market players over the last year. The company profiles section details the business overview, financial performance for the past three years, key products and services being offered along with the recent developments of these important players in the gene therapy market.

Key Topics Covered:

1. INTRODUCTION

1.1. Market Overview

1.2. Market Definition

1.3. Scope of the study

1.4. Currency

1.5. Assumptions

1.6. Base, and Forecast Years Timeline

2. Research methodology

2.1. Research Design

2.2. Secondary Sources

3. Executive Summary

4. Market Dynamics

4.1. Market Segmentation

4.2. Market Drivers

4.3. Market Restraints

4.4. Market Opportunities

4.5. Porter's Five Forces Analysis

4.5.1. Bargaining Power of Suppliers

4.5.2. Bargaining Power of Buyers

4.5.3. Threat of New Entrants

4.5.4. Threat of Substitutes

4.5.5. Competitive Rivalry in the Industry

4.6. Life Cycle Analysis - Regional Snapshot

4.7. Market Attractiveness

5. Gene Therapy Market by Technique

5.1. Gene Augmentation

5.2. Gene Inhibition

5.3. Killing of specific cells

6. Gene Therapy Market by Vector Type

6.1. Viral Vectors

6.2. Non-Viral Vectors

7. Gene Therapy Market by Indication

7.1. Genetic Disorders

7.2. Cardiovascular

7.3. Oncology

7.4. Others

8. Gene Therapy Market by Geography

8.1. North America

8.1.1. USA

8.1.2. Canada

8.1.3. Mexico

8.2. South America

8.2.1. Brazil

8.2.2. Argentina

8.2.3. Others

8.3. Europe

8.3.1. UK

8.3.2. Germany

8.3.3. Italy

8.3.4. Spain

8.3.5. Others

8.4. Middle East and Africa

8.4.1. Israel

8.4.2. Saudi Arabia

8.4.3. Others

8.5. Asia Pacific

8.5.1. China

8.5.2. Japan

8.5.3. India

8.5.4. Australia

8.5.5. Others

9. Competitive intelligence

9.1. Competitive Benchmarking and Analysis

9.2. Strategies of Key Players

9.3. Recent Investments and Deals

10. Company Profile

10.1. Novartis

10.2. Amgen

10.3. Bluebird Bio

10.4. Glaxosmithkline

10.5. Shenzhen Sibiono Genetech

10.6. Spark Therapeutics

10.7. AveXis, Inc.

10.8. Chiesi Farmaceutici

10.9. Alnylam

For more information about this report visit https://www.researchandmarkets.com/r/vcck48

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Gene Therapy Market Expected to Grow with a CAGR of 16.92% During the Forecast Period, 2019-2024 - ResearchAndMarkets.com - Business Wire

Vertex Pharmaceuticals is preparing to grow even biggeranother 256,000 square feet bigger, to be exact.

The drugmaker is in advanced talks to lease a building in Innovation Square, a research campus in Bostons Raymond Flynn Marine Industrial Park, The Boston Globe reported.

The target is the entire second phase of the new R&D hub that developer Related Beal is building on the South Boston waterfront. Its close to Vertexs existing 1.1 million-square-feet Fan Pier headquarters and would serve as a research and manufacturing facility for gene and cell therapies, according to the newspaper.

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Vertex scoured the greater Boston region for a new foothold, including sites in Cambridge, Waltham and Watertown, but picked the Innovation Square because its one of the most advanced projects in the neighborhood, on track to open in 2021, Vertex CEO Jeffrey Leiden reportedly said.

Expansion at the Innovation Square comes as the biotech giant diversifies beyond its fundamental cystic fibrosis business and into the burgeoning gene and cell therapy arena.

The question became how are we going to grow those programs if were running out of space at Fan Pier? said Leiden, as quoted by the Globe. The answer is a new building.

Leiden is transitioning to executive chairman, handing the baton to Chief Medical Officer Reshma Kewalramani. But before he moves up, a blueprint for Vertexs future growth has been laid out.

RELATED:The top 10 best-paying places to work in biopharma | 7. Vertex Pharmaceuticals

In June, Vertex put down $245 million upfront to acquire Exonics and its gene editing technology, which uses CRISPR to repair dystrophin, the protein missing in patients with Duchenne muscular dystrophy (DMD). At the same time, it shelled out $175 million upfront to deepen its ties with CRISPR Therapeutics, also for using CRISPR-Cas9 to develop DMD and myotonic dystrophy Type 1 therapies.

The first project coming out of the CRISPR-Vertex partnership has just shown promise. CTX001, a CRISPR-based therapy for severe blood disorders marked by abnormal hemoglobin, helped a beta thalassemia patient live without transfusions for nine months, and a sickle cell patient was free of the painful vaso-occlusive crises after four months, the pair unveiled last week.

Vertex also agreed to pay $950 million to snatch up Semma Therapeutics and its stem cell treatment for Type 1 diabetes.

RELATED:Vertex, CRISPR's gene-editing treatment for blood disorders shows promise in early data

The new building Vertex plans to lease will house 300 to 400 people, including employees from Exonics and Semma, as well as new hires, Leiden said, according to the Globe. Besides the lab and office space at its Fan Pier HQ, Vertex also has a lease for about 100,000 square feet of space in the Marine Industrial Park for certain logistical and laboratory operations and manufacturing equipment, the companys annual securities filing shows.

On the companys third-quarter earnings call in October, Leiden said the company will continue to do deals on early-stage assets, especially bolt-on deals to furtherits gene editing strategy.

Meanwhile, the cystic fibrosis franchise will continue to provideVertexs revenue backbone for some time. Last month, the company wonFDA approval for Trikafta, a triple combo designed to treat cystic fibrosis patients with a mutated delF508 gene, which is found in 90% of the U.S. cystic fibrosis population.

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Vertex plans major Boston expansion to support gene, cell therapy ambitions - FiercePharma

NEW YORK, Nov. 26, 2019 /PRNewswire/ -- Hoth Therapeutics, Inc. (Nasdaq: HOTH) ("HOTH" or the "Company"), a biopharmaceutical company focused on developing new generation therapies for dermatological disorders such as atopic dermatitis, chronic wounds, psoriasis and acne, today announced it has entered into a licensing agreement with North Carolina State University (NC State) to study NC State's Exon Skipping Approach for Treating Allergic Diseases.

This Exon Skipping Approach was developed by Dr. Glenn Cruse, Principal Investigator and Assistant Professor in the Department of Molecular Biomedical Sciences at the NC State College of Veterinary Medicine. During Dr. Cruse's research, a new approach for the technique of antisense oligonucleotide-mediated exon skipping to specifically target and down-regulate IgE receptor expression in mast cells was identified. These findings set a breakthrough for allergic diseases as they are driven by the activation of mast cells and the release of mediators in response to IgE-directed antigens.

Mr. Robb Knie, Chief Executive Officer of Hoth, commented, "This new collaboration will allow us to leverage this invention from the renowned expertise of Dr. Glenn Cruse and his scientific team at North Carolina State University. We look forward to seeing how their work advances and what this might mean for patients suffering from undesirable steroid side effects who need an alternate treatment for asthma and other allergic diseases."

The high-affinity IgEreceptor (FcRI) plays a central role in the initiation ofallergic responses. The research project looks to target novel genes, which are critical for surface IgE receptor expression. The project will utilize splice-switching oligonucleotides (SSOs) to force expression of a truncated isoform of the target genes to reduce expression ofFcRIin mouse asthma models.

Through this collaborative project, NCSU looks to establish the most effective approach for targeting genes that regulate surface expression of FcRI in mast cells that mediate allergic airway inflammation. The study will be administering SSOs for the target genes, to optimize delivery and examine the best therapeutic approach.

About Hoth Therapeutics, Inc.Hoth Therapeutics, Inc. isa clinical-stage biopharmaceutical company focused on developing new generation therapies for dermatological disorders. HOTH's pipeline has the potential to improve the quality of life for patients suffering from indications including atopic dermatitis, chronic wounds, psoriasis, and acne. HOTH has the exclusive worldwide rights to BioLexa, the company's proprietary lead drug candidate topical platform that uniquely combines two FDA approved compounds to fight bacterial infections across multiple indications. HOTH is preparing to launch its clinical trial for the treatment of adolescent subjects, 2-17 years of age, with mild to moderate atopic dermatitis during 2020. To learn more, please visitwww.hoththerapeutics.com.

Forward Looking StatementsThis press release includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include, but are not limited to, statements that relate to the advancement and development of the BioLexa Platform, the commencement of clinical trials, the availability of data from clinical trials and other information that is not historical information. When used herein, words such as "anticipate", "being", "will", "plan", "may", "continue", and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon Hoth's current expectations and various assumptions. Hoth believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. Hoth may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described under the caption "Risk Factors" in Hoth's Form 10K for the period endingDecember 31, 2018, and Hoth's other filings made with the Securities and Exchange Commission. Consequently, forward-looking statements should be regarded solely as Hoth's current plans, estimates and beliefs. Investors should not place undue reliance on forward-looking statements. Hoth cannot guarantee future results, events, levels of activity, performance or achievements. Hoth does not undertake and specifically declines any obligation to update, republish, or revise any forward-looking statements to reflect new information, future events or circumstances or to reflect the occurrences of unanticipated events, except as may be required by law.

ContactsInvestor Relations Contact:Phone: (646) 756-2997Email:investorrelations@hoththerapeutics.comwww.hoththerapeutics.com

KCSA Strategic CommunicationsValter Pinto / Daniela Guerrero(212) 896-1254 / (212) 682-6300Hoth@kcsa.com

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SOURCE Hoth Therapeutics, Inc.

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Hoth Therapeutics and North Carolina State University Enter License Agreement for Gene Therapy - P&T Community

(Reuters) - Blackstone Group Inc (BX.N) said on Monday it will invest $400 million in a joint venture with Swiss drug company Ferring that is working on an experimental gene therapy for bladder cancer, the private equity giants largest ever bet on drug development.

FILE PHOTO: The ticker and trading information for Blackstone Group is displayed at the post where it is traded on the floor of the New York Stock Exchange (NYSE) April 4, 2016. REUTERS/Brendan McDermid

Investing in yet-to-be-approved medicines is a lucrative but also risky proposition for buyout firms, and only few have had the stomach to place such bets. Blackstone made its foray in the sector last year, acquiring Clarus, an investment firm specializing in life sciences.

For its part, Ferring will invest $170 million in the joint venture with Blackstone, dubbed FerGene, bringing its total funding to $570 million, the companies said in a statement.

FerGene is developing a gene therapy for bladder cancer patients with an aggressive form of the disease whose current options include having their bladder removed. The treatment works by entering the walls of the bladder where it releases a gene to trigger the patients own body to make a protein to fight off cancer.

We believe, and Ferring also believes, that this can change the standard of care in bladder cancer, a terrible disease, Nicholas Galakatos, senior managing director of Blackstone Life Sciences, said in an interview.

Oncology is a new area for Ferring, but it is one that we as Blackstone Life Sciences have a lot of experience in

The team assembled by Blackstone has worked at several of the worlds largest cancer drugmakers, including Roche unit Genentech, Merck & Co Inc (MRK.N), and Millennium Pharmaceuticals, now a part of Takeda Pharmaceutical Co Ltd (4502.T).

To minimize its risk, Blackstone invests in the late stages of drug development, when a medicine has already gone through important milestones. Late-stage drug development can also be expensive because of the clinical trials involved, something that Blackstone is seeking to capitalize on by partnering with pharmaceutical firms looking to share the cost burden.

FerGenes therapy, named nadofaragene firadenovec, is currently in the final stage of clinical research, results from which will be presented on Dec. 5 at the Society of Urologic Oncologys annual meeting.

Since it launched its life sciences unit, Blackstone has also formed a new company with Novartis AG (NOVN.S) to study a type of heart drug. Blackstone invested $250 million in that venture.

Reporting by Rebecca Spalding in New York; Editing by Alistair Bell

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Blackstone to invest $400 million in gene therapy venture with Ferring - Reuters

SOUTH SAN FRANCISCO, Calif., Nov. 21, 2019 /PRNewswire/ --CODA Biotherapeutics, Inc., a preclinical-stage biopharmaceutical company developing a chemogenetic gene therapy platform to treat neurological diseases, today announced the appointment of Annahita Keravala, Ph.D., as Senior Vice President, Gene Therapy. In this role, Annahita will lead the gene therapy aspects of CODA's chemogenetic platform. The company also announced the promotions of Orion Keifer, M.D., Ph.D., to Vice President, Discovery and Translational Research, and Steve Dodson, Ph.D., to Vice President, Pharmacology and Early Development, respectively.

Michael Narachi, President and Chief Executive Officer, said, "We are excited to have Annahita, Orion, and Steve in these vital roles as we advance our lead candidates toward the clinic, as well as build our pipeline based on CODA's chemogenetic gene therapy platform. Their combined gene therapy, neuroscience and small molecule expertise, and experience in early stage research and development, will prove invaluable. With this leadership team, we will fulfill our mission of discovering and developing transformative therapies for patients with intractable neurological diseases for whom limited or no treatment options exist."

Annahita brings more than two decades of experience in gene therapy using viral and non-viral vectors. In particular, she has extensive expertise in discovering novel vector technologies and gene therapy drug development for ophthalmic, systemic and inflammatory diseases. Annahita joins CODA from Rocket Pharmaceuticals, where she was Associate Vice President, AAV Platform. At Rocket, she provided strategic, scientific and operational leadership, oversaw all aspects of discovery research, preclinical and assay development, and provided technical insight to the Chemistry, Manufacturing and Controls (CMC) team. This culminated in a successful Investigational New Drug (IND) application filing.

"I am thrilled to be joining CODA at such an exciting time in the Company's development and growth. What attracted me was the opportunity to partner with Mike, Orion, Steve and our talented team to help bring cutting-edge therapeutic options to patients living with some of the most challenging and debilitating neurological conditions and disorders like chronic neuropathic pain and focal epilepsy for which there remains great unmet medical need," said Dr. Keravala.

Prior to her tenure at Rocket Pharma, Annahita held several positions of increasing responsibility at Adverum Biotechnologies (formerly Avalanche Biotechnologies). As Director of Adverum's Novel Vector Technology group, she designed the overall research strategy, led her team to discover and optimize next-generation adeno-associated virus (AAV) vectors, and oversaw process development and preclinical testing to support the company's pipeline. Earlier, Annahita was a Research Scientist at Stanford University School of Medicine. An author of multiple patents, she is also widely published in prestigious scientific journals. Annahita earned a Ph.D. in molecular genetics and biochemistry from the University of Pittsburgh, a M.Sc. in life sciences and biotechnology from the University of Bombay, Bombay, India, and a B.Sc., with Honors in life sciences and biochemistry from St. Xavier's College, Bombay, India. She completed a post-doctoral fellowship in the Department of Genetics at Stanford University School of Medicine.

CODA's Vice President, Discovery and Translational Research, Orion Keifer, M.D., Ph.D., is a neuroscientist with neurosurgical training and hands-on expertise in small and large animal models, focused small molecule, and cell and gene therapies for neurological diseases. Before joining CODA, he worked as consultant translational scientist and surgeon for Above and Beyond focusing on precision medicines for neurodegenerative disorders. Orion earned an M.D. and a Ph.D. in neuroscience from Emory University, and a M.S. in brain and cognitive neurosciences and B.S. degrees in biomedical engineering, applied psychology and applied biology with Highest Honors from Georgia Tech. He completed his post-doctoral training in the Department of Neurosurgery at Emory University.

Steve Dodson, Ph.D., is CODA's Vice President, Pharmacology and Early Development.Prior to joining CODA, Steve served as Senior Director, Drug Discovery and Development at Second Genome, Inc. Previously he held positions of increasing responsibility at NeuroTherapeutics Pharma, Inc., and Renovis, Inc., where his work focused on the discovery and development of small molecule therapeutics to treat pain, central nervous system disorders and inflammation. Steve received his Ph.D. in biological sciences from Stanford University and a B.S. in genetics from University of California, Berkeley.

About CODA BiotherapeuticsCODA Biotherapeutics, Inc., is a preclinical-stage biopharmaceutical company developing an innovative gene therapy platform to treat neurological disorders and diseases. The company is creating the ability to control neurons with its revolutionary chemogenetics-based technology. CODA is located in South San Francisco, CA. For more information, please visit http://www.codabiotherapeutics.com.

SOURCE CODA Biotherapeutics, Inc.

http://www.codabiotherapeutics.com

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CODA Biotherapeutics Deepens Gene Therapy Expertise with Industry Veteran, Annahita Keravala, Ph.D., and Key Promotions - PRNewswire

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Newswise Boston, Mass.Beth Israel Deaconess Medical Center (BIDMC) is among a group of leading hospitals, universities, large pharmaceutical companies, small biotech firms and industry partners working together to create a new center for advanced biological innovation and manufacturing. The new centerwill explore and cultivate innovations in cell and gene therapy, advance biologic innovation and manufacturing, and accelerate developments in immunotherapy, cell therapies, gene editing, and other technologies that carry the promise of lasting impact on human health globally. By fostering collaboration and innovation, this center seeks to speed innovation and broaden the universe of patients that can be served by these emerging therapies.

Our mission at Beth Israel Deaconess Medical Center is to provide extraordinary care supported by world-class research and education, said Peter J. Healy, President of BIDMC. We are happy to be a founding member of this innovative consortium, which will allow us to work collaboratively across the diverse health care ecosystem. Together, we will propel the fields of cell therapy, gene therapy and gene editing forward with the shared goal of transforming how we care for patients right here in Boston and around the world.

Harvard University, Massachusetts Institute of Technology (MIT), Fujifilm Diosynth Biotechnologies (FDB), GE Healthcare Life Sciences, Alexandria Real Estate Equities, Inc., will comprise the Board of Directors, while BIDMC joins other contributing members, including Boston Childrens Hospital, Brigham and Womens Hospital, Dana-Farber Cancer Institute, Massachusetts General Hospital, MilliporeSigma and the Commonwealth of Massachusetts.

The overarching mission of the newly established consortium is to catalyze the development of transformative therapies by shortening the path between research and clinical application. The consortium will harness world-leading expertise to propel forward fast-emerging and promising science, the cost and risks of which are daunting for any single institution to tackle alone. By housing institutions with strengths in each link in the chain of innovation within one facility, the partners believe new innovations in both science and manufacturing will speed the introduction of new therapies to patients.

This new innovative consortium and facility will provide unique resources for our investigators to facilitate translation of research findings to clinical applications in cell-based and other novel therapies, said Gyongyi Szabo, MD, PhD, FAASLD, AGAF, FACP, Chief Academic Officer of BIDMC and Beth Israel Lahey Health. Today, more than 30 funded investigators at BIDMC are already engaged in early stage innovation focused on preclinical engineering of viral vectors, mammalian cells and cell manufacturing. BIDMCs membership in this facility will provide an accelerated path for our investigators to bring these preclinical studies to Phase I and Phase II clinical trials.

BIDMC houses a number of state of the art core facilities and laboratories on the leading edge of biotechnology. TheNon-Coding RNA Precision Diagnostics and Therapeutics Core Facilityis apre-clinical facility at BIDMC and HMS dedicated to non-coding RNA, offering informatics, sequencing, imaging and delivery of therapeutics in vitro. BIDMC is also home to theRandi and Brian Schwartz Family Cancer Immunotherapy and Cell Manipulation Facility,a laboratory that serves as a hub forBIDMCs state of the art program in personalized cell therapy for cancer. This facility is a critical resource in whichBIDMC's physician-scientists produce innovative immune-based treatments, including national leadership of apromising experimental therapeutic vaccine against several forms of blood cancer.

The $50 million center will be an independent non-profit organization located in the greater Boston area and will be named in the new year.

About Beth Israel Deaconess Medical Center Beth Israel Deaconess Medical Center is a patient care, teaching and research affiliate of Harvard Medical School and consistently ranks as a national leader among independent hospitals in National Institutes of Health funding.

For more information, visit http://www.bidmc.org.

BIDMC is part of Beth Israel Lahey Health, a new health care system that brings together academic medical centers and teaching hospitals, community and specialty hospitals, more than 4,000 physicians and 35,000 employees in a shared mission to expand access to great care and advance the science and practice of medicine through groundbreaking research and education.

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BIDMC joins local universities, teaching hospitals and industry partners in creating new central facility for regenerative therapies - Newswise

In the United States, the last Thursday of the month of November is designated as Thanksgiving, a feast day focused on giving thanks for all we have. In light of the Thanksgiving season, here are six things about biopharma that we are thankful for.

Innovation

The biopharma industry is built on innovation. Its often shocking to consider just how far the biopharma industry has come in our lifetimes, although sometimes it seems we take it for granted. There are now cures for infectious diseases like Hepatitis C, effective treatments for HIV that have turned it from a nearly-always fatal disease to a chronic condition, and new gene and cell therapies are beginning to hit the market that can cure previously untreatable diseases. For example, Spark Therapeutics Luxturna was approved by the FDA in 2017 to treat a rare, genetic form of blindness and Novartis/AveXis Zolgensma was approved for spinal muscular atrophy (SMA) in May 2019.

Every day marks new discoveries in the industry and in academia that lead to new approaches to new drugs. CRISPR gene editing promises to lead to new ways to treating hundreds of genetic diseases, and immuno-therapy is leading to new treatments and sometimes cures for tough-to-treat cancers and inflammatory diseases.

Global Oversight

A year ago, in November 2018, a Chinese researcher, He Jianku, announced he had utilized CRISPR-Cas9 gene editing to alter the DNA of embryos for seven couples. He used CRISPR to disable a gene called CCR5. This gene creates a protein that allows HIV to enter a cell. All the men of the seven couples had HIV and the women did not. The gene editings goal wasnt to prevent transmission of HIV, according to He, because the seven mens HIV infections were strongly suppressed by standard HIV drugs.

Because the technique was performed on the genomes of the babiesand at this time at least one set of twins has been bornshould those children have children someday, they will pass the change onto their children. This resulted in a frenzy of condemnation, not just by government regulators, but by the scientific community. The arguments are largely that it is way too early and way too dangerous to be conducting this type of research until ethical and legal guidelines have been developed and the risks understood.

This led to leading scientists and several different international bodies calling for a moratorium on heritable genome editing. The scientists included two of the inventors of CRISPR, Feng Zhang and Emmanuelle Charpentier. The moratorium doesnt call for a permanent ban, but stated, Rather, we call for the establishment of an international framework in which nations, while retaining the right to make their own decisions, voluntarily commit to not approve any use of clinical germline editing unless certain conditions are met.

As grateful as we are for innovation, we are also grateful that the scientific community is aware that innovation can be misused and is taking steps to regulate it.

Vaccines

One of our oldest medical advances, vaccines, is one of the most significant. Here are 14 diseases that vaccines either control or have widely eliminated: polio, tetanus, influenza, hepatitis A and B, rubella, Hib, measles, whooping cough, pneumococcal disease, rotavirus, mumps, chickenpox and diphtheria. It has completely eradicated smallpox.

Although the tried-and-true approach to vaccines is to use a dead or attenuated virus, new approaches are expanding the types of diseases vaccines are being developed for, such as cancer and Alzheimers disease. According to the Pharmaceutical Research and Manufacturers of America (PhRMA), there are 264 vaccines in development to prevent and treat diseases. These include infectious diseases (137), cancer (101), allergies (10), autoimmune disease (8) and Alzheimers disease (4).

There are regular breakthroughs in vaccine development. In May, the FDA approved Sanofis Dengvaxia, a vaccine for all four serotypes of dengue. The disease, a hemorrhagic fever, is endemic in the U.S. territories of Puerto Rico and the U.S. Virgin Islands. The vaccine has a controversial history. It was pulled from the Philippine market in 2017 over safety concerns. However, it has been approved in 10 countries in Latin America and Asia where the disease is endemic. It was approved in Europe in December 2018. And in November 2019, the European Commission approved the worlds first Ebola vaccine. The vaccine is manufactured by Merck & Co. and has a trade name of Ervebo.

We are grateful that these deadly diseases are, one by one, being eliminated thanks for vaccines.

Investors

Funding is the life blood of research and development. Where would the pharmaceutical industry be without those who provide infusions of cash to support that research, as well as other business practices. Funding can come from a number of sources, such as venture capital, the stock market and, of course, the time-honored practice of mergers and acquisitions. M&A has played a big role in industry growth and its expected to continue to do so, particularly in the areas of immuno-oncology and gene therapy. This year alone there have been several mega-acquisitions, including Takedas $60 billion acquisition of Shire; Bristol-Myers Squibbs $74 billion deal for Celgene; and AbbVies $63 billion plan to acquire Allergan.

There have been a number of pharma companies that have braved the stock market with initial public offerings this year, including Beam Therapeutics $100 million IPO, a $115 million IPO from SpringWorks Therapeutics, Bridge Biopharmas $240 million IPO and more. For companies that have not gone public, there have been some big financing rounds reported lately. Recently, Cambridge, Mass.-based eGenesis topped the list with $100 million in a Series B financing round, followed closely by a spate of other companies.

Without these financial infusions of cash, many of the medications we take for granted would not be possible.

Talent

While funding may be the life blood of the industry, its pretty clear that the researchers and other employees are the minds behind the innovations. Weekly, BioSpace notes the comings and goings of some of the top executive talent in a Movers & Shakers column. These executive leaders have shaped the pipelines and research arms of companies and, in some cases, helped shaped the future of the industry. Names like Hal Barron, Jos Baselga, William A. Lee, John C. Reed, Michael Severino, George Yancopoulos, Steffan Land and more have been responsible for guiding and shaping the research conducted in the industry. In addition to those executives, there are thousands of lab-level scientists working day in and day out to understand the mechanisms of action of medications and learning how diseases respond to those therapies.

While the vast majority of the researchers in the industry do not see their names in official company statements, a select few do end up receiving special recognition, such as the elite who win Nobel Prizes in medicine. This last year, a trio of scientists won the Nobel for their work in understanding how oxygen levels affect cellular metabolism and physiological function. The work has helped in the development of new medications to fight anemia, cancer and many other diseases. In 2018, the Nobel was awarded to researchers who cracked the code that led the way to the development of checkpoint inhibitors.

For the millions upon millions of people who rely on medications to live their best lives, we are certainly thankful for the people behind the development of these life-saving and life-enhancing treatments.

Justice Served

New treatments and the stories of those patients who benefit from life-saving and altering medications should receive the bulk of the headlines for the industry. Unfortunately, scandal and crime tends to be among the most memorable and oft-reported stories. Names like Martin Shkreli and Elizabeth Holmes are far more well-known than those previously-mentioned Nobel laureates. And their names are largely known due to the levels of infamy they both rose to in the industry. Shkreli, who is currently serving seven years in federal prison for securities fraud, is mostly known for his unflinching defense of a 5,000% increase on the toxoplasmosis drug, Daraprim, his company owned, as well as the odd behavior he displayed on social media platforms. For Shkreli, his crimes are secondary to his level of infamy. Holmes will soon go to trial for her alleged role in defrauding investors in her now-shuttered company, Theranos. She went from lauded entrepreneur to the subject of podcasts, primetime investigations, books, articles and more. While both Shkreli and Holmes gave the industry something of a black eye, we can all be thankful that these bad players are few and far between and their illegal and unethical behavior is are in the minority of industry leaders. We can be thankful that these bad players ultimately must face justice.

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6 Things About Biopharma to Be Thankful For - BioSpace

BioMarin Pharmaceuticals is seeking marketing approval in Europe for its investigational gene therapy, valoctocogene roxaparvovec, for the treatment of adults with severe hemophilia A.

The company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the experimental gene therapy, formerly known as BMN 270. Administered as a single infusion, the therapy uses adeno-associated virus (AAV) vectors to deliver a functional copy of clotting factor VIII, the protein that is missing in people with hemophilia A.

An ongoing Phase 3 trial, GENEr8-1 (NCT03370913), is investigating the treatments safety and efficacy, and is still recruiting adult patients. Go here for more information on trials locations and here for eligibility criteria.

The EMA had previously given valoctocogene roxaparvovec the designation of priority medicines, or PRIME, in 2017. Now, the potential therapy has been granted accelerated assessment, which may potentially shorten its MAA review process from 210 to 150 days.

Accelerated assessment is given by the EMAs Committee for Medicinal Products for Human Use and Committee for Advanced Therapies to innovative medications that are of major interest to public health. This endorsement is meant to speed up the review process of eligible medications, but does not impact the committees decision to recommend their approval.

BioMarins MAA submission was based on updated three-year data from a Phase 1/2 study (NCT02576795)and on an interim analysis of the ongoing Phase 3 GENEr8-1 trial (NCT03370913), which is still recruiting an anticipated 130 patients from 73 sites around the world to test the dose of 6e13 vg/kg (vector genomes per kilogram). Another Phase 3 trial, the GENEr8-2 (NCT03392974) study, is also ongoing and testing a lower dose (4e13 vg/kg).

Three-year data from the Phase 1/2 trial showed that a single administration of valoctocogene roxaparvovec at the higher dose markedly reduced bleeding episodes and the need for factor VIII infusions in a small group of adults with severe hemophilia A. Specifically, there was a 96% reduction in both the mean ABR (annualized bleed rate) and the mean factor VIII usage over the three years.

The levels of clotting factor VIII remained stable over the course of three years following treatment.

Valoctocogene roxaparvovec was generally well-tolerated by patients. None of the participants developed inhibitors to factor VIII, and none withdrew from the study due to adverse events.

We are grateful to the study participants, who have made this progress possible in the span of approximately four years since the first participant was enrolled in the clinical program, Hank Fuchs, MD, president of BioMarins global research and development, said in a press release.

We are very pleased with the level of engagement we have had with global health authorities, as it aligns with our belief that gene therapy represents the next wave of innovation and potentially could be a meaningful advancement for treating people with severe hemophilia A, Fuchs said.

Valoctocogene roxaparvovec will be the first gene therapy for hemophilia whose MAA will be reviewed by health authorities for potential approval in the E.U. BioMarin is expecting the EMA to start reviewing its application in January 2020 and said it will provide an update at that time.

In the meantime, the company is planning to submit a biologics license application for valoctocogene roxaparvovec to the U.S. Food and Drug Administration (FDA) by the end of the year. The investigational treatment has been given a breakthrough therapy designation by the FDA, as well asorphan drug status from both the FDA and EMA.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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For Hemophilia A, BioMarin Seeks Approval of Its Gene Therapy in Europe - Hemophilia News Today

Can a reformulation of an old drug on the market for asthma, allergy and mastocytosis protect against liver disease, Alzheimers, and amyotrophic lateral sclerosis?

A team from Massachusetts General Hospital is making a case for the latter two. Having recently found an injection of cromolyn sodium effective in inhibiting amyloid beta (A) aggregation in vitro and in mouse models, researchers set out to investigate whether the same compound can achieve the same in ALS.

Their conclusion, published on Natures open access journal Scientific Reports:

Our results indicate that cromolyn sodium treatment significantly delayed the onset of neurological symptoms, and improved deficits in PaGE performance in both male and female mice, however, there was only an effect on survival in female mice.

While the precise etiology of ALS remains poorly understood, one theory proposes that neuroinflammatory processes are implicated in its initiation and progression.

As cromolyn inhibits mast cell degranulation, the scientists at Mass General hypothesized that it could convert immune cells in the brain, including microglia and astrocytes, from a pro-inflammatory to an anti-inflammatory state as well as reducing the levels of cytokines and chemokines.

After comparing the effects of once-daily injections of cromolyn versus a placebo in wild type mice and mice carrying a genetic mutation for ALS, respectively, the researchers came up empty on the microglia and astrocytes theory. But they did find lower levels of pro-inflammatory cytokines/chemokines in the spinal cord and plasma in addition to observing that the transgenic mice treated with cromolyn sodium had the highest motor neuron counts among the four groups.

Our study supports the notion that inflammation has a significant role in the progression of ALS and therefore exploring anti-inflammatory treatments may be of great value for developing an effective treatment, said Ghazaleh Sadri-Vakili, lead author of the study and director of the NeuroEpigenetics Laboratory at Mass General, in a statement.

Sadri-Vakili has previously noted that the neurology department at Mass General has a plan in place to get the drug provided by AZTherapies into the clinic for ALS.

Unlike the formulations of cromolyn currently available through prescription and over the counter, which are absorbed through lung and nasal inhalation or ingested drops, the version injected into mice in the study can be fully available in the bloodstream and cerebrospinal fluid, according to the biotech.

AZTherapies is conducting a Phase III trial of another formulation of cromolyn, combined with oral ibuprofen, to treat early Alzheimers. It is unclear whether any clinical programs are in place to test cromolyn sodium in primary sclerosing cholangitis after Baylor Scott and White Health researchers reported it decreased biliary proliferation and fibrosis in mice.

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Mass General team presents mouse data to back the case for using reformulated asthma drug to treat ALS - Endpoints News

One of the big themes in R&D over the past few years has been the onslaught of spending on developing new oncology drugs. The FDA has encouraged early approvals, opening the door to smaller trials as an onslaught of investment cash made it possible for small players to go the distance.

Big Pharma, meanwhile, has enjoyed the comfort of better scientific insights and the arrival of some huge new PD-1s on the scene. Next up: A tsunami of combo therapies coming at you from the US, Europe and China the new factor in drug hunting.

For the industry, that means a major new source of revenue from coming therapies. For US patients, that means a much better shot at longer survival and possibly even a cure. As well as bankruptcy.

Wait. What?

Researchers just published a new study in The American Journal of Medicine highlighting that 42% of cancer patients exhaust their savings within 2 years of diagnosis. And 62% of the 9.5 million cancer patients they reviewed were in debt after therapy, with 40% to 85% quitting work due to cancer. After 4 years of therapy, 38.2% were insolvent.

You hear a lot every time a new drug is approved about what drug companies are doing to make their therapies accessible, but the simple fact is that in the US large percentages of patients are being crushed by the price of branded drugs. And while the new drugs being introduced may be more important than ever, the unvarnished truth is that basic pricing strategies are more about maximizing revenue than accommodating patients.

The resulting financial toxicity is enormous.

Lets remember that one of the reasons were seeing all the investment cash pouring in is that Wall Street has embraced a big wave of biotech IPOs. And thats where execs are focused when they price new drugs.

Thats not a wild guess, either.

This is the bottom line researchers totted up after discussing pricing strategies on new drugs for multiple sclerosis with 4 biotech execs, published in Neurology this week.

Participants consistently stated that initial price decisions were dictated by the price of existing competitors in the market. Revenue maximization and corporate growth were drivers of price escalations in the absence of continued market penetration. Lower revenue predictions outside the United States also informed pricing strategies. The growing complexity and clout of drug distribution and supply channels were also cited as contributing factors. Although decisions to raise prices were motivated by the need to attract investment for future innovation, recouping drug-specific research and development costs as a justification was not strongly endorsed as having a significant influence on pricing decisions.

So while the industry likes to talk a lot about the pricing levels needed to back innovation, its just not an accurate reflection of reality.

In just a few weeks, were going to wake up to a new year that will be dominated by drug pricing discussions. Were going to be doing some of this ourselves at JP Morgan.

The industry still has a shot at coming up with some kind of workable reform on drug prices. Barring a market solution, though, you can expect plenty of unworkable and destructive suggestions on drug importation and compulsory licensing and more. And someday, lawmakers will do something about it.

Continued here:
There's one endpoint that the booming biopharma industry has failed at miserably: financial toxicity - Endpoints News

Japans Asahi Kasei Pharma has stepped up with a deal to buy out Copenhagen-based Veloxis Pharmaceuticals for $1.3 billion, bagging an organ rejection drug in the process.

The deal comes 17 years after Veloxis was founded, and the two big shareholders Novo Holdings and Lundbeckfond Invest have both signed off on the buyout. The biotech makes an immunosuppressive drug called Envarsus XR for kidney transplants.

Veloxis has been a low profile biotech on the continent, but recently saw its share price zoom up. Asahi Kasei is paying DKK 6 cash per share, just a 6% premium over the average trading price tracked from October 14 to November 22. But its a 75% premium over the one-year weighted average. The deal was announced on Monday.

Japanese pharmas have been snapping up pharma products around the world for a number of years now, looking to expand beyond Japan as they look for new and growing markets. And Asahi Kasei President Hideki Kobori says they plan to follow up on the acquisition with some new strategic deals.

The acquisition of Veloxis by Asahi Kasei is a great culmination of more than a decade of research, development, and commercialization of life science innovation, which is now benefitting thousands of patients around the world, said Novo Holdings senior partner Christoffer Sderberg in a prepared statement. We are proud of what the employees of Veloxis have accomplished and are pleased that they will all continue with Asahi Kasei.

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Veloxis wins $1.3B buyout, and the new owner plans to follow up with new deals - Endpoints News

After quietly plowing the arid cancer vaccine field for a decade, a low profile Swiss biotech is throwing itself in the ring as a player to watch in the frenetic race to extend the benefits of immunotherapies to solid tumors.

Co-founded by biotech vet Bernard Mach who was involved in the creation of Biogen and Novimmune and his son Nicolas, MaxiVAX is laser-focused on developing a two-part cancer vaccine that activates the immune system to attack cancer. And it has been awarded a 2.785 million ($3.07 million) grant from the European Commission on top of CHF 5 million ($5.01 million) from a Series B2 round to push through a Phase II study in head and neck cancer in Switzerland and kick off a US study for an undisclosed rare tumor.

Much has been made of cancer vaccines potential to complement immune checkpoint inhibitors, which can be incredibly effective in subsets of patients, sometimes as few as 20%. But the first generation of cancer vaccines, largely made of peptides derived from cancer cells, has failed to induce the immune effects needed to make a difference on their own.

A key issue, MaxiVAX CEO Dimitrios Goundis said, is that peptides carry mutations that vary from patient to patient. Their solution: Isolate the whole tumor cells, containing the complete antigenic repertoire for the immune cells to profile and target, inactivate them, then implant them subcutaneously next to an adjuvant meant to sustain the immune response.

So our therapy basically is a vaccine, which is patient specific, and an immune boosting agent (GM-CSF) which is delivered by encapsulated genetically modified cells, which allow continued delivery of GM-CSF over several days at the site of vaccination, he told Endpoints News. And with that we address we think all the weaknesses that weve identified over the course of the last 20, 30 years when people start to look at vaccines against cancers in a more systematic way.

GM-CSF, or granulocyte-macrophage colony-stimulating factor, is a naturally occurring immune modulator that has a relatively short half life, normally disappearing within a couple hours in the bloodstream. To keep it flowing, MaxiVAX is inserting a gene into hundreds of thousands of cells to produce a steady supply of GM-CSF such as immune cells that can stay energized as they get familiarized with irradiated tumor cells.

The improvement should be clear cut, he said. While checkpoints have extended median survival in advanced and refractory head and neck cancer from three to six months, that still means half of the patients dont live that long. For the ongoing Phase II, for which they are recruiting around 40 patients across six sites, MaxiVAX will be monitoring the number of patients who pass the 6-month survival threshold as the main efficacy measure.

Its a hard endpoint, he said. So were not only looking at whether or not the tumor is reduced in size or disappears, but also what is the effect of that in prolonging the life of the patients.

He expects the final results from the open-label trial to be in around 2021. Before that, MaxiVAXs small team of 10 will also initiate a Phase II for which they have already obtained an IND in the US.

The new funds from private investors MaxiVAX has managed to stay away from venture capitalists in raising over $15 million so far will also go toward building out manufacturing.

Right now the biotech relies on the Geneva Hospital, where Nicolas Mach is head of oncology, to isolate the tumor cells and to generate and fill the capsules with GM-CSF producing cells. Its offices are located in the neighborhood, which also houses AMAL Therapeutics, another next-gen cancer vaccine player recently bought out by Boehringer Ingelheim.

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Another cancer vaccine play out of Geneva shoots for 'hard endpoint' in PhII head and neck cancer test - Endpoints News

Cancer could be defined as uncontrolled cell growth in the body leading to organ malfunction. If untreated, it can lead to death. Uncontrolled growth of cell is managed by the body in several ways, one of them is by deploying white blood cells to detect and eradicate these cancerous cells. It has been discovered that the immune system could be manipulated to influence cancerous cells to destroy itself.

Access Report Details at: https://www.themarketreports.com/report/global-cancer-gene-therapy-market-by-manufacturers-countries-type-and-application-forecast

Market share of global Cancer Gene Therapy industry is dominate by companies like Adaptimmune, Bluebird bio, Celgene, Shanghai Sunway Biotech, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, Altor BioScience, Amgen, Argenx, BioCancell, GlaxoSmithKline, Merck, OncoGenex Pharmaceuticals, Transgene and others which are profiled in this report as well in terms of Sales, Price, Revenue, Gross Margin and Market Share (2017-2018).

With the help of 15 chapters spread over 100 pages this report describe Cancer Gene Therapy Introduction, product scope, market overview, market opportunities, market risk, and market driving force. Later it provide top manufacturers sales, revenue, and price of Cancer Gene Therapy, in 2017 and 2018 followed by regional and country wise analysis of sales, revenue and market share. Added to above, the important forecasting information by regions, type and application, with sales and revenue from 2019 to 2024 is provided in this research report. At last information about Cancer Gene Therapy sales channel, distributors, traders, dealers, and research findings completes the global Cancer Gene Therapy market research report.

Market Segment by Regions, regional analysis covers:

Market Segment by Type, covers:

Market Segment by Applications, can be divided into

Purchase this premium research report at: https://www.themarketreports.com/report/buy-now/1498456

Table of Contents

1 Market Overview

2 Manufacturers Profiles

3 Global Cancer Gene Therapy Market Competitions, by Manufacturer

4 Global Cancer Gene Therapy Market Analysis by Regions

5 North America Cancer Gene Therapy by Countries

6 Europe Cancer Gene Therapy by Countries

7 Asia-Pacific Cancer Gene Therapy by Countries

8 South America Cancer Gene Therapy by Countries

9 Middle East and Africa Cancer Gene Therapy by Countries

10 Global Cancer Gene Therapy Market Segment by Type

11 Global Cancer Gene Therapy Market Segment by Application

12 Cancer Gene Therapy Market Forecast (2019-2024)

13 Sales Channel, Distributors, Traders and Dealers

14 Research Findings and Conclusion

15 Appendix

Ask your report related queries at: https://www.themarketreports.com/report/ask-your-query/1498456

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Global Cancer Gene Therapy Market Report with Study of North America, Europe and Asia-Pacific, South America, Middle East and Africa Regions -...

At an old Amgen facility tucked just beyond the Rockies. In a warehouse behind a Walmart supercenter in Durham, North Carolina. On a long-time Bristol Myers Squibb site outside Princeton. The tech has emerged, and now the arms race to physically build a generation of gene therapies has begun.

Novartis will spend $500 million scaling its gene therapy manufacturing efforts, Reuters reported today. Thatll put it nearly on par with Pfizer, who committed $600 million for its facilities even before any of its gene therapies have been approved. Together, 11 companies Reuters surveyed will spend $2 billion on gene therapy production.

Additionally, the Boston Globereported today that Vertex had completed its search for a gene therapy research and manufacturing campus in Boston, settling on a 256,000 square-foot center at the Raymond Flynn Marine Industrial Park.

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Make that 2 new sickle cell disease blockbuster hopefuls OK'd by the FDA in just a few days - Endpoints News

The study of Gene Therapy Market is useful in providing answers to several critical questions that are important for the industry stakeholders such as collaboration solution and service vendors, distributors and partners, end users, etc., besides allowing them in strategizing investments and capitalizing on market opportunities. The report also constructed elementary and necessary Gene Therapy inspection movement, stating through basic analysis by driving wide gatherings with experts holding Gene Therapy key positions inside the business, for example, Presidents, VIPs, executives, and directors. The analysts authoring the report provide a meticulous evaluation of all of the segments included in the report.

Get a Sample Copy of Report: https://prophecymarketinsights.com/market_insight/Insight/request-sample/39

The report involves perceptive data on the main sectors of the global Gene Therapy market, paired with the sub-segments. The aforementioned factors are boosting its adoption and propelling the growth of the global Gene Therapy market. This report studies the global Gene Therapy Market status and forecast, categorizes the global Gene Therapy Market size (value & volume), revenue (Million USD), product price by manufacturers, type, application, and region.

Segmentation Overview:

By Gene Therapy Type (Germline Gene Therapy and Somatic Gene Therapy)

By Type of Vector (Viral Vector and Non-viral Vector)

By Disease Indication (Cardio Vascular Diseases, Cancer, Genetic Disorders, Neuro Disorders, Infectious Diseases, and Others)

By Region (North America, Europe, Asia Pacific, Latin America, Middle East, and Africa)

The Gene Therapy market report comprises an in general successful system, confinements and in and out disclosures of the past information alongside the inspected present and future needs that may concern the development. The most recent latest report on the Gene Therapy market is a vital collection of insights pertaining to this vertical, with regards to certain parameters. In this report, absolute skills have been reprocessed to the anticipated size of the pattern in Gene Therapy market from the earnings of leading participants. In the global Gene Therapy Market research report, various important aspects such as regional market insights, region-wise trends, country-level analysis, competitive landscape, company market share analysis, and key company profiles are covered :

GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

Download PDF Brochure of this study: https://prophecymarketinsights.com/market_insight/Insight/request-pdf/39

The Global Gene Therapy market report will be useful guidance material for below aspirants:

Leading Gene Therapy players Producers of major Gene Therapy components, distributors, suppliers, target consumers and others

Major Gene Therapy service providers

Potential Gene Therapy users and target industries

Annual product launch events, up-to-date market trends, and upcoming technologies will be useful to all the Gene Therapy market aspirants

Institutes and organizations actively involved in gaining information on Gene Therapy industry

All other institutes like Government bodies, private firms, ventures will get benefit from Gene Therapy market report A key trend that is expected to predominantly influence the Gene Therapy market in a coming year is an evolution of new applications in future. The Gene Therapy research and development teams in various companies are working towards the further technical development of Gene Therapy in order to cater the next-generation industries.

The Gene Therapy market have widespread application in multiple industries. However, professionals across the globe are intensively working towards the development of Gene Therapy technologies. This will be even more flexible and reliable, along with being cost-effective Gene Therapy industry. This trend is anticipated to drive the demand for Gene Therapy in various other novel industries. Furthermore, Gene Therapy readers will get a clear perspective on the most affecting driving and restraining forces in the Gene Therapy market and its impact on the global market. The report predicts the future outlook for Gene Therapy market that will help the readers in making appropriate decisions on which Gene Therapy market segments to focus in the upcoming years accordingly. In a word, the Gene Therapy report offers a whole consequential study of the parent Gene Therapy market, key tactics followed by leading Gene Therapy industry Players and upcoming segments. Likewise, the former and current Gene Therapy industry forecast analysis in terms of volume and value along with research conclusions is a decisive part of Gene Therapy study. So that Gene Therapy report helps the new aspirants to inspect the forthcoming opportunities in the Gene Therapy market.

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How will Gene Therapy continue to evolve in the next 10 years? - Montana Ledger

With the help of 15 chapters spread over 100 pages this report describe Gene Therapy for Age-related Macular Degeneration Introduction, product scope, market overview, market opportunities, market risk, and market driving force. Later it provide top manufacturers sales, revenue, and price of Gene Therapy for Age-related Macular Degeneration, in 2017 and 2018 followed by regional and country wise analysis of sales, revenue and market share. Added to above, the important forecasting information by regions, type and application, with sales and revenue from 2018 to 2024 is provided in this research report. At last information about Gene Therapy for Age-related Macular Degeneration sales channel, distributors, traders, dealers, and research findings completes the global Gene Therapy for Age-related Macular Degeneration market research report.

Access Report Details at: https://www.themarketreports.com/report/global-gene-therapy-for-age-related-macular-degeneration-market-by-manufacturers-countries-type-and-application-forecast

Market share of global Gene Therapy for Age-related Macular Degeneration industry is dominate by companies like Retrosense Therapeutics, Regenxbio, Agtc and others which are profiled in this report as well in terms of Sales, Price, Revenue, Gross Margin and Market Share (2018-2019).

Market Segment by Regions, regional analysis covers:

Market Segment by Type, covers:

Market Segment by Applications, can be divided into

Purchase this premium research report at: https://www.themarketreports.com/report/buy-now/1497384

Table of Contents

1 Market Overview

2 Manufacturers Profiles

3 Global Gene Therapy for Age-related Macular Degeneration Market Competitions, by Manufacturer

4 Global Gene Therapy for Age-related Macular Degeneration Market Analysis by Regions

5 North America Gene Therapy for Age-related Macular Degeneration by Countries

6 Europe Gene Therapy for Age-related Macular Degeneration by Countries

7 Asia-Pacific Gene Therapy for Age-related Macular Degeneration by Countries

8 South America Gene Therapy for Age-related Macular Degeneration by Countries

9 Middle East and Africa Gene Therapy for Age-related Macular Degeneration by Countries

10 Global Gene Therapy for Age-related Macular Degeneration Market Segment by Type

11 Global Gene Therapy for Age-related Macular Degeneration Market Segment by Application

12 Gene Therapy for Age-related Macular Degeneration Market Forecast (2019-2024)

13 Sales Channel, Distributors, Traders and Dealers

14 Research Findings and Conclusion

15 Appendix

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Global Gene Therapy for Age-related Macular Degeneration Market Forecast (2019-2024) Report: By Regions, Type and Application with Sales and Revenue...

Wall Street analysts expect Axovant Gene Therapies Ltd (NASDAQ:AXGT) to report earnings of ($0.63) per share for the current quarter, Zacks reports. Zero analysts have issued estimates for Axovant Gene Therapies earnings, with the highest EPS estimate coming in at ($0.57) and the lowest estimate coming in at ($0.66). Axovant Gene Therapies posted earnings of ($2.16) per share in the same quarter last year, which suggests a positive year-over-year growth rate of 70.8%. The firm is scheduled to report its next quarterly earnings report on Thursday, February 6th.

According to Zacks, analysts expect that Axovant Gene Therapies will report full-year earnings of ($3.56) per share for the current fiscal year, with EPS estimates ranging from ($5.76) to ($1.84). For the next fiscal year, analysts anticipate that the business will post earnings of ($2.29) per share, with EPS estimates ranging from ($2.84) to ($1.20). Zacks Investment Researchs earnings per share calculations are an average based on a survey of sell-side research analysts that follow Axovant Gene Therapies.

A number of equities research analysts have recently issued reports on AXGT shares. ValuEngine upgraded shares of Axovant Gene Therapies from a sell rating to a hold rating in a research report on Thursday, August 1st. Robert W. Baird upgraded Axovant Gene Therapies from a neutral rating to an outperform rating and cut their price objective for the stock from $16.00 to $13.00 in a research report on Monday, August 12th. Zacks Investment Research upgraded Axovant Gene Therapies from a hold rating to a strong-buy rating and set a $6.00 price objective for the company in a research report on Wednesday, November 13th. Finally, Chardan Capital lifted their price objective on Axovant Gene Therapies from $10.00 to $15.00 and gave the stock a buy rating in a research report on Monday, October 28th. Two research analysts have rated the stock with a hold rating, eight have given a buy rating and one has assigned a strong buy rating to the company. Axovant Gene Therapies presently has a consensus rating of Buy and an average target price of $24.72.

Several hedge funds and other institutional investors have recently made changes to their positions in AXGT. BlackRock Inc. acquired a new position in shares of Axovant Gene Therapies during the 2nd quarter valued at $1,482,000. Jane Street Group LLC lifted its holdings in shares of Axovant Gene Therapies by 28.8% during the 2nd quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock valued at $289,000 after acquiring an additional 10,375 shares in the last quarter. Finally, Tower Research Capital LLC TRC lifted its holdings in shares of Axovant Gene Therapies by 955.3% during the 2nd quarter. Tower Research Capital LLC TRC now owns 4,221 shares of the companys stock valued at $27,000 after acquiring an additional 3,821 shares in the last quarter. 13.84% of the stock is owned by institutional investors.

Axovant Gene Therapies Company Profile

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

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Axovant Gene Therapies Ltd (NASDAQ:AXGT) Expected to Post Earnings of -$0.63 Per Share - Mitchell Messenger

Local and state officials gathered Friday in Alachua to commemorate a $6 million expansion at Progress Park.

Representatives of Thermo Fisher Scientific Inc., a global science company involved with research and biotechnology product development, presented the companys expanded gene therapy and viral vector services site. The day included tours of the 95,000-square-foot facility.

Chris Murphy, vice president and general manager of Thermo Fisher Viral Vector Services, said gene therapy provides genetic information to patients who lack it.

Ive been in this industry for over 20 years in gene therapy, and over the last three years, its completely transformed, he said. This site and this group and these folks here couldnt be better positioned to be part of that revolution.

Larry Pitcher, general manager and head of the Alachua site, said the company at its three-building location currently employs about 250 people who work with viral vectors to treat and potentially cure rare, complex diseases.

Key players in the more than decade-long development, who also spoke at the unveiling, included Greater Gainesville Chamber of Commerce Board Chair Bryan Harrington, the University of Floridas Assistance Vice President of Technology Commercialization Jim OConnell, state Sen. Keith Perry, and Alachua Mayor Gib Coerper.

OConnell noted that the research started 16 years ago at the University of Florida when Richard Snyder founded Florida Biologix, which changed to Brammer Bio through a merger with Brammer Biopharmaceuticals in 2016. Brammer Bio then merged with Thermo Fisher Scientific in May.

OConnell said a grant from state representatives originally kickstarted the initiative.

I think its critical to recognize these things dont happen overnight, he said.

Murphy said he first came to Progress Park 18 years ago. He highlighted the influence of the company merger and the scale of the industry.

(Its) been just a remarkable synergy and nothing but positiveness, and, frankly, the investment continues, he said. $270 million will be invested by pharma-services groups across the world.

Pitcher mentioned the continued growth of the Alachua site over the last two to three years and the increase of about 150 jobs.

Its really about the patient communities that we serve, Pitcher said, and getting these therapies to them as soon as possible.

The presentation concluded with Greater Gainesville President and CEO Eric Gobet gifting Thermo Fisher representatives with a key to the region from the Greater Gainesville Chamber.

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$6 Million Expansion For Gene Therapy Facility Completed At Alachua's Progress Park - WUFT

DUBLIN, Nov. 22, 2019 /PRNewswire/ -- The "Cell and Gene Therapy Market - Global Outlook and Forecast 2019-2024" report has been added to ResearchAndMarkets.com's offering.

The global cell and gene therapy market is growing at a CAGR of over 24% during the forecast period 2018-2024.

Key Market Insights

The major drivers contributing to the growth of the global cell and gene therapy market are the growing incidence of several chronic and terminal diseases, including cancer, the launch of new products, the increasing availability in clinical evidences of these products in terms of safety and efficacy, the rapid adoption of CAR T-cell therapy, favorable regulatory support in the development of these treatment, and improved manufacturing expertise in these products.

Market DynamicsMarket Growth Enablers

Market Growth Restraints

Market Opportunities and Trends

Cell and Gene Therapy Market: Segmentation

This research report includes detailed market segmentation by product, application, end-user, and geography.

The global cell therapy market is growing at a steady rate, and this trend is expected to continue during the forecast period due to the increased patient base with a wide range of diseases/ailments. The segment is likely to witness upward growth on account of expanded expertise in the manufacturing of stem cell-based products.

The gene therapy segment is expected to witness faster growth as the penetration of these products is increasing at a significant rate, especially in developed economies. The market is expected to grow during the forecast period due to the increased patient base for the existing gene remedy products, expected the launch of other gene therapy-based products for several indications, and expanded indication approvals for existing commercially available products.

The oncology segment accounts for the highest share of the global market. The growth of the oncology segment is increasing at a fast rate on account of the growing prevalence of several types of cancers. Currently, the available products not only modify the disease but also improve the quality of the patient's life, thereby decreasing the mortality rate. The market in the dermatology segment is increasing at a steady rate. This segment owns its growth to the increasing incidence and prevalence rate of several types of wounds, which are difficult to treat under normal conditions and the launch of innovative products. The dermatology segment is likely to showcase growth due to the high product availability of wound care products in the market.

Hospitals are the leading end-user segment. The segment is growing mainly due to the increasing incidence/prevalence of chronic diseases such as cancer, cardiovascular diseases, diabetes, and chronic wound on account of diabetes feet, pressure ulcers, and other injuries.

Market Segmentation by Products

Market Segmentation by Distribution Channel Type

Market Segmentation by End-users

Geographical Insights

The US market dominates the cell and gene therapy market in North America due to the high prevalence of chronic diseases and other conditions, which require these treatment methods. There is also comparably high utilization and wide accessibility of these therapies. The oncology segment is likely to witness significant growth in North America.

The market in Europe is expected to witness upward growth in the near future on account of the growing prevalence of chronic diseases and rising elderly population. In Europe, cell and gene therapy products are considered to be part of the Advanced Therapy Medicinal Products (ATMPs), which are commonly known as regenerative medicine globally.

Key Vendor Analysis

The global market is characterized by the presence of a few global, large-scale companies and several small to medium-scale companies offering one or two cell and gene therapy products. Global players are majorly offering innovative products with the potential of disease-modifying characteristics that are generating significant revenues, especially in Europe and US regions. Most innovative and breakthrough products are approved in the European countries and the US.

Vendors are targeting mostly developed economies such as the US, Germany, France, the UK, Spain, and Japan as the uptake of these products is higher in these countries than low and middle-income countries. However, the market in these regions is at the nascent stage.

Key Vendors

Other Prominent Vendors

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Worldwide Cell & Gene Therapy Market Insights Study, 2019-2024 - Market Set to Surpass a CAGR of 24%, Driven by Favorable Regulatory Support & Special...

The central government has decided to support research on gene therapy in a big way to tackle Indias escalating burden of non-communicable diseases (NCDs) and inherited critical disorders such as spinal muscular atrophy (SMA), thalassemia and haemophilia. Scientific institutions and universities conducting research on genomics and its therapeutic applications will soon get a financial fillip from public coffers, under an initiative facilitated and funded by the Indian Council of Medical Research (ICMR).

Gene therapy is a technique that uses genetic modifications to treat or prevent diseases. The experimental technique allows clinicians to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery or replacing a mutated gene that causes diseases with a healthy copy of it.

According to the proposal note, a copy which CNBCTV18.com has reviewed, the funds will be disbursed for research projects across various therapeutic areas including gene editing, gene transfer techniques and immunotherapy based on genetic modifications. The apex body for biomedical research promotion has already started accepting proposals from scientists and clinicians.

Why gene therapy is crucial

In the current era of genomics and genetic testing, it is evident that a large swath of the Indian population harbours causative mutations leading to various disorders. Conversely, for many inherited and complex disorders, the underlying genetic causes are known.

And most of these illnesses are currently not treatable by molecule drugs or traditional therapies. According to patient advocacy groups, though there are major developments in gene therapy for cancer and other chronic diseases in the developed world, most of them are not accessible or affordable to a majority of the Indian population.

For instance, the US Food and Drug Administration (FDA) recently approved Zolgensma, the first gene therapy to treat children less than two years of age with SMA, a leading genetic cause of infant mortality. Though the drugs clinical trial in older SMA-affected children remains partially halted because of an inflammatory response observed in an animal study, there are very few options left for these patients.

Majority of these rare diseases, including SMA, are progressive and require life-long treatment and continuous support, says Archana Panda, co-founder of Cure SMA India, a non-profit group that works for making SMA treatment accessible to Indians.

In the case of SMA, a disease that leads to loss of motor neurons and early death, Nusinersen is the only drug approved by the US FDA. Developed by US-biotech major Biogen and marketed as Spinraza, the drug is administered directly to the central nervous system intravenously and the therapy could cost around $750,000 in the first year.

But in recent years, significant strides have been made in the field of gene therapy. This has culminated in the recent FDA approvals for Luxturna (gene therapy for retinitis pigmentosa) and Yescarta (CAR-T cell therapy for lymphoma). Many such approaches are currently under investigation or in early clinical trials. Now, the ICMR-backed initiative aims to fill the gaps in research thrust in India by providing emphasis on the focus areas which require more attention to address the needs for the large existing patient base, the note reads.

Hope on the horizon for patients

Apart from multi-factorial diseases such as cancer, diabetes and lung ailments, the research will also focus on treatment for rare diseases such as neuro-muscular diseases (including Duchenne muscular dystrophy, Becker muscular dystrophy, SMA and myopathies), retinal or corneal disorders (including Retinitis Pigmentosa, Stargardt disease and Fuchs dystrophy), inherited heart diseases and blood disorders including Thalassemia, Sickle Cell Disease and haemophilia.

The initiative assumes significance as it is estimated that one in 20 Indians is affected by one of the 7,000 diseases listed by the World Health Organisation as rare diseases. For many of these illnesses, gene therapy is the final hope. About 16 lakh Indians are diagnosed with cancer every year, and 8 lakh people lose their life to the disease.

While the fund support for research which can lead to human trials is only a baby step, experts such as Dr Mamta Muranjan, who heads the Genetic Clinic at KEM Hospital in Mumbai, emphasise the need for such focused long-term policies. Most patients with rare diseases remain undiagnosed for a long time. Even if the diagnosis is on time, the family of the patient is usually unable to afford the treatment cost. Insurance policies generally do not cover these life-long treatment expenses, she says.

Possibility of translation into human trials

According to official sources, the three-year state-funded research projects would be selected on the basis of their potential for development of functional treatment options. The strategies proposed should have the possibility of translation into future human trials.

Read more here:
Govt to fund gene therapy research on NCDs, rare diseases - CNBCTV18

Timrepigene emparvovec is a potential first-in-class AAV2 gene therapy for the treatment of choroideremia, a rare, degenerative, X-linked retinal disorder that leads to blindness

CAMBRIDGE, Mass., Nov. 21, 2019 (GLOBE NEWSWIRE) -- Biogen Inc.(BIIB) announced today the enrollment of the last patient in the global Phase 3 STAR clinical study, which is evaluating the investigational gene therapy timrepigene emparvovec (BIIB111/AAV2-REP1) for the treatment of choroideremia (CHM). CHM is a rare, degenerative, X-linked inherited retinal disorder that leads to blindness.

We are excited to advance innovative investigational treatments for inherited retinal disorders that have significant unmet medical need due to the lack of treatment options, said Alfred Sandrock, Jr., M.D., Ph.D., Executive Vice President, Research and Development, and Chief Medical Officer at Biogen. Timrepigene emparvovec could be a transformative gene therapy for individuals living with choroideremia who would otherwise face inevitable blindness. Completing enrollment of our Phase 3 study represents a significant milestone in bringing this new potential therapy to patients.

STAR is a randomized, masked, prospective, parallel-controlled group Phase 3 study that enrolled 170 adult males with CHM. The study is evaluating the safety and efficacy of a single subretinal injection of timrepigene emparvovec. The primary endpoint is the proportion of patients with an improvement of at least 15 letters from baseline in best corrected visual acuity (BCVA) at 12 months post treatment as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity protocol. The STAR study was initiated based on proof-of-concept data from Phase 1/2 studies, which indicated that at month 24, over 90 percent of patients treated with timrepigene emparvovec via targeted subretinal injection maintained visual acuity instead of experiencing the natural decline in BCVA expected in this degenerative disease. In a subset of treated patients with moderate to severe visual acuity loss, 21 percent experienced a gain in visual acuity of at least 15 ETDRS letters from baseline as compared to one percent of untreated patients in a natural history study.

CHM primarily affects males and is caused by a loss of function in the CHM gene which encodes the Rab escort protein-1 (REP-1). The REP-1 protein plays a role in intracellular protein trafficking, and the loss of function in the CHM gene leads to abnormal intracellular protein trafficking and impaired elimination of waste products from the retinal pigment epithelium and photoreceptors. Initially, patients with CHM experience poor night vision and over time, progressive visual loss ultimately leads to blindness.

Biogen added timrepigene emparvovec to its portfolio in June 2019 as part of its acquisition of Nightstar Therapeutics.

For more information about the Phase 3 STAR study, visit http://www.clinicaltrials.gov (NCT03496012).

About timrepigene emparvovec (BIIB111/AAV2-REP1)Timrepigene emparvovec is an AAV2 vector administered by subretinal injection, which aims to provide a functioning CHM gene and expression of the REP-1 protein to restore membrane trafficking and thereby slow, stop or potentially reverse decline in vision. Data from the Phase 1/2 studies demonstrated a slower rate of decline in visual acuity in patients treated with timrepigene emparvovec compared to untreated patients in the natural history study. In addition, some patients treated with timrepigene emparvovec showed improvements in visual acuity. The studies also demonstrated that timrepigene emparvovec was generally well tolerated with an acceptable safety profile.

Timrepigene emparvovec has received regenerative medicine advanced therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA), which includes all of the benefits of the fast track and breakthrough therapy designation programs and orphan drug designations in the U.S., Europe and Japan. The safety and efficacy of a single subretinal injection of timrepigene emparvovec is currently being evaluated in the ongoing Phase 3 STAR study.

About Biogen At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the worlds first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics, and is focused on advancing research programs in multiple sclerosis and neuroimmunology, neuromuscular disorders, movement disorders, Alzheimers disease and dementia, ophthalmology, immunology, neurocognitive disorders, acute neurology, and pain.

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We routinely post information that may be important to investors on our website atwww.biogen.com. To learn more, please visitwww.biogen.comand follow us on social media Twitter,LinkedIn,Facebook,YouTube.

Biogen Safe Harbor StatementThis news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, relating to the potential, benefits, safety and efficacy of timrepigene emparvovec; the potential clinical effects of timrepigene emparvovec; results from the Phase 1/2 studies of timrepigene emparvovec; the clinical development program for timrepigene emparvovec; the treatment of CHM; the potential of our commercial business and pipeline programs, including timrepigene emparvovec; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by words such as aim, anticipate, believe, could, estimate, expect, forecast, intend, may, plan, potential, possible, will, would and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk and only a small number of research and development programs result in commercialization of a product. Results in early stage clinical trials may not be indicative of full results or 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.

These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation, uncertainty of success in the development and potential commercialization of timrepigene emparvovec; unexpected concerns may arise from additional data, analysis or results obtained during the STAR study; regulatory authorities may require additional information or further studies, or may fail or refuse to approve or may delay approval of our drug candidates, including timrepigene emparvovec; the occurrence of adverse safety events; the risks of other unexpected hurdles, costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; and product liability claims. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.

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Biogen Announces Enrollment Completion of Global Phase 3 Gene Therapy Study for an Inherited Retinal Disorder - Yahoo Finance

MANHATTAN, Kan., Nov. 21, 2019 /PRNewswire/ -- Libella Gene Therapeutics, LLC("Libella") announces an institutional review board (IRB)-approved pay-to-play clinical trial in Colombia (South America) using gene therapy that aims to treat and ultimately cure aging. This could lead to Libella offering the world's only treatment to cure and reverse aging by 20 years.

Under Libella's pay-to-play model, trial participants will be enrolled in their country of origin after paying$1 million. Participants will travel to Colombia to sign their informed consent and to receive the Libella gene therapy under a strictly controlled hospital environment.

Traditionally, aging has been viewed as a natural process. This view has shifted, and now scientists believe that aging should be seen as a disease. The research in this field has led to the belief that the kingpin of aging in humans is the shortening of our telomeres.

Telomeres are the body's biological clock. Every time a cell divides, telomeres shorten, and our cells become less efficient at dividing again. This is why we age. A significant number of scientific peer-reviewed studies have confirmed this. Some of these studies have shown actual age reversal in every way imaginable simply by lengthening telomeres.

Bill Andrews, Ph.D., Libella's Chief Scientific Officer, has developed a gene therapy that aims to lengthen telomeres. Dr. Andrew's gene therapy delivery system has been demonstrated as safe with minimal adverse reactions in about 200 clinical trials. Dr. Andrews led the research at Geron Corporation over 20 years ago that initially discovered human telomerase and was part of the team that led the initial experiments related to telomerase induction and cancer.

Telomerase gene therapy in mice delays aging and increases longevity. Libella's clinical trial involves a new gene-therapy using a proprietary AAV Reverse (hTERT) Transcriptase enzyme and aims to lengthen telomeres. Libella believes that lengthening telomeres is the key to treating and possibly curing aging.

Libella's clinical trial has been posted at the United States National Library of Medicine (NLM)'s clinicaltrials.gov database. Libella is the world's first and only gene therapy company with a clinical trial posted at clinicaltrials.gov that aims to reverse the condition of aging.

On why they decided to conduct its project outside the United States, Libella's President, Dr. Jeff Mathis, said, "Traditional clinical trials in the U.S. can take years and millions, or even billions,of dollars. The research and techniques that have been proven to work are ready now. We believe we have the scientist, the technology, the physicians, and the lab partners that are necessary to get this trial done faster and at a lower cost in Colombia."

Media Contact:Osvaldo R. Martinez-ClarkPhone: +1 (786) 471-7814Email: ozclark@libellagt.com

Related Files

curing_aging_booklet.pdf

Related Images

william-bill-andrews-ph-d.jpg William (Bill) Andrews, Ph.D. Dr. Bill Andrews is a scientist who has spent his entire life trying to defeat the processes that cause us to age. He has been featured in Popular Science, The Today Show, and numerous documentaries on the topic of life extension including The Immortalists documentary.

Related Links

Dr. Bill Andrews speech at RAADfest 2018 (Sept 21, San Diego, CA)

bioaccess: Libella's CRO partner in Colombia.

SOURCE Libella Gene Therapeutics, LLC

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Breakthrough Gene Therapy Clinical Trial is the World's First That Aims to Reverse 20 Years of Aging in Humans - PRNewswire

Dive Brief:

Fujifilm is looking to take advantage of the gene therapy market which, for CDMOs, is expected to reach $1.7 billion by 2025, according to Fujifilm Diosynth Biotechnologies' market research.

"We are very much aware of the incredible growth in such an important therapeutic space," Martin Meeson, president and chief operating officer of FDB U.S., said in a Nov. 14 statement. "We know that we need to invest now, in technology, assets and people in order to achieve a market leadership position."

The new Gene Therapy Innovation Center on the existing FDB campus in Texas will be about 60,000 square feet and operational starting in fall 2021. FDB expects the center to triple its gene therapy development capabilities and add about 100 jobs.

Meanwhile, the first stage of the expansion for the existing manufacturing facility should be complete by the spring of 2021, Fujifilm said.

The Japan-based company's fresh investments in gene therapy come amid a wave of expansions, mergers and partnerships in the growing field.

This month, Swiss manufacturer Lonza announced a new partnership with cold chain specialist Cryoport as part of its goal to provide a seamless "vein-to-vein" network in cell and gene therapy. Lonza also opened a 300,000-square-foot plant last year in Texas dedicated to manufacturing the therapies.

Catalent earlier this year bought Paragon Bioservices for $1.2 billion to strengthen its position as a CDMO of gene therapies. And Thermo Fisher acquired viral vector manufacturer Brammer Bio for $1.7 billion.

Fujifilm forecasts sales of 100 billion yen for its CDMO business in the fiscal year ending March 2022.

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Fujifilm to invest $120M in gene therapy, build center in Texas - BioPharma Dive

Hertfordshire Local Enterprise Partnership is to invest 1.2m into Stevenage Bioscience Catalyst (SBC) to help grow a world-class cell and gene therapy cluster.

The 1.2m Local Growth Fund investment will provide much-needed temporary accommodation for growing cell and gene therapy companies, strengthening the UKs sectorial advantage and Hertfordshires position within the Oxford-London-Cambridge golden research triangle.

SBC is a world-leading science park of global significance and home to the largest cluster of cell and gene therapy companies in Europe. Since its opening in 2012, it has attracted over 40 high-profile occupier businesses and world-class research organisations to Stevenage.

This includes the Cell and Gene Therapy Catapult Manufacturing Centre which already supports five companies developing their manufacturing at scale and is currently undergoing expansion to help support up to 12 cell and gene therapy companies.

With both SBC and the Cell and Gene Therapy Catapults first large scale manufacturing centre on the campus, companies can benefit from close proximity to manufacturing, development and research. This unique co-location has been a key driver of growth in this exciting and emerging treatment area.

Occupiers of the SBC campus have raised almost 1bn of investment, of which around 60% has been invested into cell and gene therapy companies. Now several of these companies are wanting to expand their premises at SBC.

To help meet this demand, Hertfordshire LEP has granted 1.2m capital contribution to the development of the new Spark Building. This will provide interim lab and office accommodation whilst more permanent accommodation is developed nearby. The Spark Building, due for completion by the end of 2019, will be co-located with the SBC Incubator, Accelerator and the Cell and Gene Therapy Catapult Manufacturing Centre.

Dr Sally Ann Forsyth, Chief Executive Officer at Stevenage Bioscience Catalyst, commented: We are delighted to receive the investment and continued support from Hertfordshire LEP. This ensures that we support the growth of our occupiers and continue to build on our position as Europes hub for cell and gene therapy.

Paul Witcombe, Head of Enterprise and Innovation, Hertfordshire LEP, commented: Stevenage Bioscience Catalyst has made a huge contribution to the rapid clustering of world-class corporate and academic research excellence on the GSK campus at Stevenage. To date, we have already approved up to 12m from the Local Growth Fund to develop essential infrastructure to support scientific research and we anticipate further opportunities to support the cluster.

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1.2m investment in Stevenage Bioscience Catalyst for world-class cell and gene therapy - Pharmafield

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The worlds first breakthrough gene therapy clinical trial aims to reverse 20 years of aging in humans.

USA: Using genetherapy to express active telomerase (hTERT) in humans has the potential to treat many of the age-related diseases, including aging itself. Now, Libella Gene Therapeutics, has set up a pay-to-play clinical trial inColombia(South America) that aims to use the gene therapy to reverse aging by 20 years. If successful, the therapy will become the worlds only treatment to treat and cure aging.

The trial, approved by Libellas institutional review board (IRB), will be a pay-to-play model wherein volunteers have to pay $1m for enrollment in their country. Participants will then travel to Colombia to sign their consent. Gene therapy will be administered to participants under a controlled hospital setting.

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Aging has been viewed as a natural aging process. But now, scientists believe aging to be a disease. Further research in this field has led to the belief that aging in humans is primarily caused by the shortening of telomeres caps at the end of each DNA strand that protects the end of the chromosome. Every time a cell divides telomeres shorten and the cells become less efficient at dividing again resulting in aging. This has been confirmed by a significant number of scientific peer-reviewed studies. Some of these studies have shown actual age reversal in every way imaginable simply by lengthening telomeres.

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Bill Andrews, Libellas Chief Scientific Officer, has developed a gene therapy that aims to lengthen telomeres. Dr. Andrews gene therapy delivery system has been demonstrated as safe with minimal adverse reactions in about 200 clinical trials. Dr. Andrews led the research at Geron Corporation over 20 years ago that initially discovered human telomerase and was part of the team that led the initial experiments related to telomerase induction and cancer.

Telomerase gene therapy in mice delays aging and increases longevity. Libellas clinical trial involves a new gene-therapy using a proprietary AAV Reverse (hTERT) Transcriptase enzyme and aims to lengthen telomeres. Libella believes that lengthening telomeres is the key to treating and possibly curing aging.

In the Phase I trial, the gene therapy will be evaluated for its safety and tolerability in approximately five participants. It is expected to be completed in 2021.

The primary endpoint is the incidence of adverse events, while secondary outcomes are hTERT expression and telomerase activity.

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For more information visit: Libella Gene Therapeutics

Medha Baranwal joined Medical Dialogues as an Editor in 2018 for Speciality Medical Dialogues. She covers several medical specialties including Cardiac Sciences, Dentistry, Diabetes and Endo, Diagnostics, ENT, Gastroenterology, Neurosciences, and Radiology. She has completed her Bachelors in Biomedical Sciences from DU and then pursued Masters in Biotechnology from Amity University. She has a working experience of 5 years in the field of medical research writing, scientific writing, content writing, and content management. She can be contacted atmedha@medicaldialogues.in. Contact no. 011-43720751

To know about our editorial teamclick here

Source: Libella Gene Therapeutics

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Gene therapy to reverse age by 20 years: World's first trial - Specialty Medical Dialogues