Archive for the ‘Gene Therapy Research’ Category

In this report, the Global Gene Therapy for Inherited Genetic Disorders market is valued at USD XX million in 2017 and is expected to reach USD XX million by the end of 2025, growing at a CAGR of XX% between 2017 and 2025. Global Gene Therapy for Inherited Genetic Disorders market has been broken down by major regions, with complete market estimates on the basis of products/applications on a regional basis.

Browse full research report at https://www.crystalmarketreport.com/global-gene-therapy-for-inherited-genetic-disorders-market-report-history-and-forecast-2014-2025-breakdown-data-by-companies-key-regions-types-and-application

Summary

In the medicine field gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patients cells as a drug to treat disease.A genetic disorder is a genetic problem caused by one or more abnormalities formed in the genome.

Gene therapy has the potential to cure inherited diseases that cannot be cured using conventional drugs. It is being increasingly adopted in clinical trials because of its high efficacy and safety towards the treatment of inherited genetic disorders.

In 2018, the global Gene Therapy for Inherited Genetic Disorders market size was xx million US$ and it is expected to reach xx million US$ by the end of 2025, with a CAGR of xx% between 2019 and 2025.

This report studies the Gene Therapy for Inherited Genetic Disorders market size by players, regions, product types and end industries, history data 2014-2018 and forecast data 2019-2025; This report also studies the global market competition landscape, market drivers and trends, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

This report focuses on the global top players, covered

BioMarin Pharmaceutical Inc.

bluebird bio Inc.

Novartis AG

Orchard Therapeutics Plc

Spark Therapeutics Inc.

Market segment by Regions/Countries, this report covers

North America

Europe

China

Rest of Asia Pacific

Central & South America

Middle East & Africa

Market segment by Type, the product can be split into

Eye Disorders

Hematological Disorders

Central Nervous System Disorders

Muscular Disorders

Others

Market segment by Application, the market can be split into

Hospital

Clinic

Research Institute

Others

The study objectives of this report are:

To study and forecast the market size of Gene Therapy for Inherited Genetic Disorders in global market.

To analyze the global key players, SWOT analysis, value and global market share for top players.

To define, describe and forecast the market by type, end use and region.

To analyze and compare the market status and forecast among global major regions.

To analyze the global key regions market potential and advantage, opportunity and challenge, restraints and risks.

To identify significant trends and factors driving or inhibiting the market growth.

To analyze the opportunities in the market for stakeholders by identifying the high growth segments.

To strategically analyze each submarket with respect to individual growth trend and their contribution to the market

To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.

To strategically profile the key players and comprehensively analyze their growth strategies.

In this study, the years considered to estimate the market size of Gene Therapy for Inherited Genetic Disorders are as follows:

History Year: 2014-2018

Base Year: 2018

Estimated Year: 2019

Forecast Year 2019 to 2025

For the data information by region, company, type and application, 2018 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

Key Stakeholders

Raw material suppliers

Distributors/traders/wholesalers/suppliers

Regulatory bodies, including government agencies and NGO

Commercial research & development (R&D) institutions

Importers and exporters

Government organizations, research organizations, and consulting firms

Trade associations and industry bodies

End-use industries

Available Customizations

With the given market data, QYResearch offers customizations according to the companys specific needs. The following customization options are available for the report:

Further breakdown of Gene Therapy for Inherited Genetic Disorders market on basis of the key contributing countries.

Detailed analysis and profiling of additional market players.

Browse full research report at https://www.crystalmarketreport.com/global-gene-therapy-for-inherited-genetic-disorders-market-report-history-and-forecast-2014-2025-breakdown-data-by-companies-key-regions-types-and-application

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Global Gene Therapy for Inherited Genetic Disorders Market Report, History and Forecast 2014-2025, Breakdown Data by Companies, Key Regions, Types and...

Sareptas lousy summer has come to a close with some encouraging fresh functional data from its limb-girdle muscular dystrophy (LGMD) gene-therapy program.

In February, the drugmaker divulged data from three patients in the first cohort of an open-label Phase I/II study testing the use of an experimental gene therapy MYO-101 (now called SRP-9003) in LGMD patients aged four to 15 years.

Data showed the therapy rejuvenated the production, by an average of 51%, of beta-sarcoglycan the protein required for muscle function that is missing in this patient population. On Friday, Sarepta broke out data that showed that the induced uptick in beta-sarcoglycan did, in fact, translate to functional improvements, nine months post-infusion.

Based on the natural history of the disease, these changes were definitely not expected at these time points, Sarepta executives underscored on a conference call with analysts.

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We'll see this week if the public markets' love affair with biotech unicorns is still running hot or not - Endpoints News

Researchers at the NIH have rolled out a new vehicle for sickle cell gene therapy with higher speeds and better horsepower, potentially allowing vastly more efficient gene transfer and a much larger carrying capacity. The best part? Unlike current sickle cell gene therapy models, the NIH one doesnt have to drive in reverse.

In mice and monkeys, the new vehicle was up to 10 times more efficient and had a carrying capacity the amount of DNA it can haul of up to 6 times that of the conventional vectors currently deployed in gene therapy trials across the country. Most notably, the new vector can read the therapeutic gene sequence forward rather than reading them backward a counter-intuitive trick researchers had used to overcome long-running barriers to gene therapy but which sacrificed efficiency. The results were published open access inNature Communications.

Our new vector is an important breakthrough in the field of gene therapy for sickle cell disease, said study senior author John Tisdale, chief of the Cellular and Molecular Therapeutic Branch at the National Heart, Lung, and Blood Institute (NHLBI). Its the new kid on the block and represents a substantial improvement in our ability to produce high capacity, high-efficiency vectors for treating this devastating disorder.

Gene therapy trials for SCD have launched the past few years, bringing a handful of well-covered cases of patients responding strongly to the treatment, even as more data shows current techniques are no cure-all. One of the bigger longstanding questions, though, is how to best deliver the genetic fix.

The simple genetic underpinnings of the disease have been well-understood since the 1950s one A-T substitution in the -globin gene and researchers have accordingly targeted it since the first gene therapy research in the 1980s. But the particular problems of building a proper vector for the hemoglobin gene, in addition to the myriad other obstacles to gene therapy broadly, have impeded progress.

Click on the image to see the full-sized version

The lentiviral vector bluebird bio has used to bring its sickle cell gene therapy to trial is a workaround to an early problem unique to sickle cell therapy. RNA splicing a natural process critical to preparing the vector will remove introns that are key to expressing the genes to produce hemoglobin. Developers have been able to get around this by using a vector that reads the DNA backwards, last gene to first. Most gene therapy techniques read as you would a sentence, first word to last.

The researchers also noted their vectors were cheaper to produce.

Excerpt from:
New NIH viral vector flips the script on sickle cell disease gene therapy - Endpoints News

News Release

Thursday, October 3, 2019

New grants aimed at better understanding diseases, moving potential treatments closer to the clinic.

Of an estimated 6,500 to 7,000 known rare diseases, only a fraction maybe 5% have U.S. Food and Drug Administration-approved treatments. To increase that percentage, the National Institutes of Health has awarded approximately $31 million in grants in fiscal year 2019 to 20 teams including five new groups -- of scientists, clinicians, patients, families and patient advocates to study a wide range of rare diseases. An additional $7 million has been awarded to a separate data coordinating center to support these research efforts.

The grants, which support consortia that together form the Rare Diseases Clinical Research Network (RDCRN), are aimed at fostering collaborative research among scientists to better understand how rare diseases progress and to develop improved approaches for diagnosis and treatment. This is the fourth five-year funding cycle for the RDCRN, which is supported by multiple NIH Institutes and Centers and led by NIHs National Center for Advancing Translational Sciences (NCATS) and the NCATS Office of Rare Diseases Research.

Individually, most rare diseases affect only a few hundred to several thousand people; collectively, rare diseases affect more than 25 million Americans. Many rare diseases are life-threatening and about half of those affected are children.

Because rare diseases affect a small number of people, they can be extremely challenging to study. Scientists often lack basic information about a rare diseases symptoms and biology, and the ways a disease can affect people over time. Research funding can be scarce.

Over the years, RDCRN scientists have partnered with patients and advocates to develop new insights into the causes and progression of and potential therapies for rare diseases that were simply not receiving the attention they deserved, said NCATS Director Christopher Austin, M.D. Their pioneering work in discerning underlying clinical differences and commonalities in hundreds of rare conditions has already changed the rare disease landscape in immeasurable ways.

Established by Congress under the Rare Diseases Act in 2002, the RDCRN has included more than 350 sites in the United States and more than 50 in 22 other countries. To date, they have encompassed 237 research protocols and included more than 56,000 participants in studies ranging from immune system disorders and rare cancers to heart and lung disorders, brain development diseases and more.

Each RDCRN member is a consortium of clinical and scientific experts and patient groups who study a group of rare diseases. Each consortium must study three or more diseases, partner with rare disease patient advocacy groups, provide rare disease research training to investigators and perform natural history studies that chart the course and progression of diseases. The primary focus of the RDCRN is clinical research, and the network does not generally support clinical care outside of research activities.

A key component of the RDCRN is the Data Management and Coordinating Center (DMCC), which was awarded to the Cincinnati Childrens Hospital Medical Center. The DMCC manages shared resources and data from the RDCRN research studies. The DMCC emphasizes the standardization of data, increased data sharing and broad dissemination of research findings.

The RDCRN consortia have a rich history of accomplishment. For example, Lysosomal Disease Network scientists led crucial natural history studies and gene editing research that provided a foundation for first-in-human genome editing clinical studies for a rare metabolic disease. Primary Immune Deficiency Treatment Consortium members showed the advantage of early stem cell transplants for patients with a rare immune system disorder, severe combined immunodeficiency, and the groups work contributed to advances in gene therapy-based treatments for the disease.

New groups, new emphasis

The five new consortia are:

According to ORDR director Anne Pariser, M.D., an important focus of the latest group of awards is on clinical trial readiness.

Some of the RDCRN research groups have been working together for 10 or 15 years and have gathered important data and developed a good understanding of the diseases they study, in addition to new potential therapies. Were emphasizing the need to be prepared to conduct clinical trials, Pariser said.

Were trying to get the drug candidates closer to be ready for clinical testing and de-risk the processes that lead to a successful clinical trial, said RDCRN program officer Tiina Urv, Ph.D. To get funding to conduct trials, they need to have strong natural history studies that show how the disease progresses, ways to measure outcomes of treatments and biomarker studies that provide indicators of how a drug is working in patients.

Collaboration is key. Consortia can involve numerous partner research teams from different sites, along with rare disease patients and advocacy groups. Scientists from different institutions come together to pool patients, data, experience and resources.

Scientists cant work alone. They wouldnt have enough patients, and they wouldnt have adequate resources and information about the diseases, Urv said. Patients and families help scientists decide what is important to study, test and treat.

To read more about the five new consortia, 15 continuing consortia and the DMCC, see: https://ncats.nih.gov/rdcrn/consortia

In addition to NCATS, other NIH funding support comes from the National Institute of Allergy and Infectious Diseases, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institute of Neurological Disorders and Stroke, the National Heart, Lung, and Blood Institute, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of Dental and Craniofacial Research, the National Institute of Mental Health and the Office of Dietary Supplements.

About the National Center for Advancing Translational Sciences (NCATS):NCATS conducts and supports research on the science and operation of translation the process by which interventions to improve health are developed and implemented to allow more treatments to get to more patients more quickly. For more information about how NCATS is improving health through smarter science, visithttps://ncats.nih.gov.

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

NIHTurning Discovery Into Health

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NIH funding bolsters rare diseases research collaborations | National Institutes of Health - National Institutes of Health

October 3, 2019

The cell and gene therapy field has exploded over the past few years. Fueled by innovations in immunotherapy, cell engineering and biomanufacturing, as well as landmark FDA approvals, a new era of medicine is upon us that is delivering new approaches to the treatment of cancer and other serious and life-threatening diseases.

This nascent industry is expanding all across the globe and Maryland, in particular, has seen an exponential boom within its cell and gene therapy cluster. Its this boom that Maryland Tech Council (MTC) CEO Marty Rosendale hopes to capitalize on to help Maryland rise above the likes of leading clusters such as Boston and the San Francisco Bay Area to be the number one destination for life science companies.

Rosendale is a competitive guy, and hes seen this same fire and competitive drive from many of the trade organizations 435 tech and life science members. In a recent Baltimore Business Journal article titled, Md. Tech Council CEO wants state biotech industry to aim higher than top 3, Rosendale shared, I think its time to look again, focus in on areas were already doing very well in, and aim for No. 1.

An important distinction is that Being the #1 place for companies to grow their business is different from being #1 in the rankings of GEN and JLL, he added.

Rosendale believes that Maryland, the anchor of the Biohealth Capital Region (BHCR), is already #1 for many companies due to the regions strong quality of life, close proximity to the National Institutes of Health and other research institutions and its deep talent pool.

He points to recent examples like Kite Pharma choosing to build its manufacturing site in Frederick County and the recent acquisition of Paragon Bioservices by Catalent as confirmation of the BHCRs ascending reputation as a global biohealth cluster. Add to this the regions growing cell and gene therapy industry, its blossoming immunotherapy ecosystem and a rapidly developing advanced biomanufacturing capability, and its easy to see the source of Rosendales confidence.

Success will bring more success. The more we can drive success it will build on itself and the message will become clearer and clearer. Its about getting that message out and incorporating it into the public discussion, he added.

Getting the message out and building on the regions success is what motivated Maryland Life Sciences (a division of MTC) to launch the upcoming Bio Innovation Conference, which is expected to draw a crowd of more than 400 life science professionals on Monday.

The Bio Innovation Conference is focused on cell and gene therapy this year and whats happening in this space and why its happening in Maryland, stated Rosendale.It is designed to explore what can we do, together, to help build this industry. We have panels on advanced biomanufacturing, capital markets and the science behind the industry and great keynotes in Dr. John Tisdale of NIH and Michelle McMurray-Heath of JLABS @ Washington DC, he added.

Were experiencing an exciting time in the region, with wave upon wave of innovations, collaborations, acquisitions, tech transfer and growth, said Rosendale. Companies from all over the country are moving here to experience all that Maryland and the region have to offer. This conference provides the perfect backdrop to showcase it all and discuss how to further propel us to the No. 1 spot among life sciences hubs.

Notable speakers in the cell and gene therapy/biomanufacturing space include:

In our recent podcast, Maryland Secretary of Commerce Kelly Shulz summed up what we need in one word: Stories. We need to tell stories. We need to tell people our success stories and why theyve been successful and why being in Maryland helped them succeed. The more we can tell these stories, the more we can get everyone talking about our region and thereby increase our recognition, Rosendale stated.

If you are attending the Bio Innovation Conference on Monday, youll hear dozens of success stories and will walk away with no doubt as to why biotech, particularly cell and gene therapy and biomanufacturing, is thriving in Maryland.

Over the past 8 years, Chris has grown BioBuzz into a respected brand that is recognized for its community building, networking events and news stories about the local biotech industry. In addition, he runs a Recruiting and Marketing Agency that helps companies attract top talent through a blended model that combines employer branding and marketing services together with a high powered recruiting solution.

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Bio Innovation Conference to Highlight Cell and Gene Therapy Industry & Why Maryland is Becoming the #1 Destination for Biotech Companies -...

Tenaya CEO Faraz Ali(Tenaya Therapeutics)

Although heart disease remains the leading cause of death worldwide, its an area that hasnt seen as much interest or investment as other areas, and its treatments still focus on dealing with symptoms. With a multipronged approach and a fresh infusion of $92 million, Tenaya Therapeutics is trying to change that.

The heart is a complicated organ, and it can go wrong in different ways. Part of what weve learned the hard way is that prior approaches are not working, Tenaya CEO Faraz Ali told FierceBiotech.

Founded in 2016 by scientists from the Gladstone Institute in California and the University of Texas Southwestern Medical Center, Tenaya is going after the underlying causes of heart disease to head off heart failure. It raised $50 million in its series A round from The Column Group, which also pitched into its $92 million B round alongside the likes of Casdin Capital and GV.

CIOs Perspectives: Driving Clinical Trial Innovation with a Unified Platform

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RELATED: GV leads $58.5M round for Verve, a startup looking to pit gene editing against heart attacks

In an age where companies are forming themselves around one platform thats disease-agnostic and can be applied to many different areas, South San Francisco-based Tenaya is doing the opposite. It's working on three different platforms for heart disease:regenerative treatments, gene therapies and precision medicines.

The company was founded with a big, bold mission to follow the science and use the right tool for the job, Ali said. If the problem is loss of cardiomyocytes (the muscle cells that make the heart beat), such as after a heart attack, we can look for a way to generate new myocytes, create new tissue and improve the ability of the heart to contract that way.

Thats where regenerative treatments come in. Tenaya's approach delivers transcription factors that can nudge heart fibroblasts, cells that play a role in scar formation after a heart attack, to become heart muscle.

If the problem stems from geneticsif a faulty gene doesnt cause heart muscle to die, but leads to an arrhythmia or scarring, stopping it from working properlythe solution is not to create new muscle, but to get the muscle thats there to work, Ali said. And thats where gene therapy, adding a healthy copy of a defective gene, comes in.

Finally, Tenayas taking a leaf out of the book of cancer drug developers: Its become quite the norm to look for therapies that work in a particular genetic background, he said. The companys precision medicine platform uses stem cell-derived heart muscle cells as disease models to identify new targets for heart failure and screen new drugs. Its first focus is on small molecules for the treatment of dilated cardiomyopathiesa group of conditions in which an enlarged heart chamber makes it less efficient at pumpingin genetically defined patient groups.

What the funding allows us to do is advance multiple promising projects from each of the platforms out of the pure research stage and into the clinic, Ali said. Tenaya hopes to move to human studies over the next few years.

RELATED: Renovacor bags $11M to push precision medicine for rare heart disease

Though the company isnt divulging just yet which targets its going after, Ali did say some of its programs are chasing more prevalent conditions such as heart attacks while others are looking at smaller, more defined populations.

Once we get a signal of efficacy and safety and advance into later-stage clinical development, we could potentially expand into larger, more prevalent indications, he said.

Tenaya has about 45 staffers who are mostly focused on research, early development and manufacturing. If all goes to plan, the company plans to double its size by the end of 2021, adding more employees in development and manufacturing.

Two-thirds of our platforms that were working on [gene therapy and regenerative treatments] are heavily dependent on viral vectors, adeno-associated viruses Everyone learned in the last decade or so that manufacturing is the Achilles heel of the gene therapy spaceit's difficult to do and highly technical, its nothing like small molecule manufacturing, Ali said. We made the decision to invest early, and well ahead of being in the clinic, to invest in manufacturing.

Tenaya isnt just doing it because other gene therapy players have proved it necessary. If it wants to go after larger heart disease populations rather than the smaller groups affected by rare disease, its going to need a lot of viral vector to deliver its treatments.

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Tenaya Therapeutics bags $92M to develop triple threat for heart disease - FierceBiotech

Novartis(SWX: NOVN), a multinational pharmaceutical company based in Basel, Switzerland, is launching an AI innovation lab to enable its associates to use AI across the business.

The company selected Microsoft Corp. as its strategic AI and data-science partner. The new lab aims to bolster Novartis AI capabilities from research through commercialization and accelerate the discovery and development of transformative medicines for patients worldwide.

As part of the strategic collaboration, Novartis and Microsoft have committed to a multi-year research and development effort. The lab will bring AI to Novartis associates. By bringing together vast amounts of Novartis datasets with Microsofts advanced AI solutions, it will create new AI models and applications that can augment associates capabilities to take on the next wave of challenges in medicine.The lab will use AI to tackle hard computational challenges within the life sciences, starting with generative chemistry, image segmentation & analysis for smart and personalized delivery of therapies, and optimization of cell and gene therapies at scale.

Microsoft and Novartis will also collaborate to develop and apply next-generation AI platforms and processes that support future programs across these two focus areas. The overall investment will include project funding, subject-matter experts, technology, and tools.

Joint research activities will include co-working environments on Novartis Campus (Switzerland), at Novartis Global Service Center in Dublin, and at Microsoft Research Lab (UK) starting with tackling personalized therapies for macular degeneration; cell & gene therapy; and drug designBasel, and Redmond.

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Originally posted here:
Novartis and Microsoft Team Up to Advance Medicine with AI - FinSMEs

By Rob Wright, Chief Editor, Life Science LeaderFollow Me On Twitter @RfwrightLSL

Matt Patterson

THAT WAS A TERRIFYING MOMENT,reflects Matt Patterson, cofounder, chairman, and CEO of Audentes Therapeutics, a genetic medicines company focused on the adeno-associated virus (AAV). It was the fall of 2012, and Patterson was preparing a final pitch to OrbiMed Advisors to lead a $25 million to $30 million Series A funding round for Audentes, which was then just an idea for a company. But pitching OrbiMed wasnt what was terrifying. After all, at the time Patterson was working as an entrepreneur in residence (EIR) for OrbiMed, so he knew the people in the room. However, this was his last pitch after his first few hadnt garnered much interest. You have to remember, in 2012 there werent a lot of gene therapy companies other than bluebird bio, he explains.

In his role as an EIR, Patterson had connected with a researcher at Wake Forest University working on X-linked myotubular myopathy (XLMTM), a severe neuromuscular disease with no available treatment. It took me about 72 hours to decide I wanted to start a company to develop something for these patients and their families, and I quickly came up with an initial vision for how to do a clinical trial, what endpoints to study, and what might be compelling to regulatory authorities, he notes.

But when he made that first pitch to OrbiMed, Audentes was considered extremely high risk, since in addition to being gene therapy, it was based on early-stage preclinical work and a single asset idea. To decrease that risk and make the company more compelling for his final pitch, Patterson added programs for Pompe disease and Duchenne muscular dystrophy through additional academic connections.

As an EIR, you are obligated to provide your employer [i.e., OrbiMed] the first opportunity to invest in any idea you have, though legally youre a consultant and can leave to do your own thing, he clarifies. And although he really wanted OrbiMeds support, he already had determined and communicated that if they werent interested by this point, hed seek funding elsewhere. This was the nerve-wracking part; not having OrbiMed would make raising funds much more difficult. How do you explain to other VCs why the firm you worked for didnt want to be part of the build? he asks.

Ultimately, OrbiMed provided Audentes with $1 million in seed funding and a soft commitment to support the Series A. It wasnt the robust financial infusion Patterson had hoped for, but it was enough to get started.

FROM THE CLEANROOM TO THE BOARD ROOM

Though Patterson studied science in college, he says he didnt know much about biotech until 1993. A college friend, also a biochemistry major, got a lab job at Biogen Idec, he shares. The friend encouraged Patterson to look into the same work, and eventually he landed a manufacturing tech job at Genzyme. I was a protein purification technician in a GMP facility, he recounts proudly. In other words, his days involved gowning in and then spending 6 to 8 hours working away in a lab coat, hairnet, and safety glasses. It has played an important role for me as a CEO, because I can relate well to the day-to-day challenges faced by people working in manufacturing and laboratory areas, he says.

After nearly five years at Genzyme, during which time he transitioned to working in regulatory affairs, he moved to California and joined BioMarin as employee number 15. Now in a more senior role, he had the opportunity to work with and learn from folks like Emil Kakkis, M.D., Ph.D., current president and CEO of Ultragenyx Pharmaceuticals. At BioMarin I realized rare diseases was where I wanted to work for the long term, and after leading the regulatory group though approval of our first product, they offered me the opportunity to expand into other more business-oriented roles, he says. This eventually led to Patterson joining Amicus Therapeutics in 2004 as chief business officer and employee number eight. Less than two years later, he was picked to be COO, and less than five years after that he found himself in an even bigger role. John Crowley, Amicus chairman and CEO, was considering alternative careers at the time and had decided to step down, so I became acting CEO. Being a CEO hadnt been a driving force in my early days, but rather something that became more real over time as I found myself in ever-expanding roles. However, Crowley ended up returning as Amicus CEO, and Patterson decided it was time to consider a new opportunity.

In late 2011 he joined OrbiMed as an EIR, where he was working closely with General Partner Jonathan Silverstein, who had brought Patterson to the firm. One of the benefits of being at a VC is there is an amazing number of technologies and company profiles that flow through these firms, he elaborates. It also seemed an interesting way to see a wide variety of prospects, to learn, and eventually land a CEO opportunity. During this time, Patterson became reacquainted with gene therapy. Amazed by the amount of progress being made, especially in the monogenic rare disease targets where he was so personally passionate, he began reaching out to various academic contacts he had made during his time at BioMarin and Amicus. This was how he connected with the researcher at Wake Forest working on XLMTM. The early research was extremely compelling and had all the hallmarks of past successful programs I had worked on, meaning there was significant medical need, a clear understanding of the scientific basis of the disease, compelling early data from two animal models (i.e., mouse and dog), and a motivated patient and medical community wanting to see research advance. All of these things were exciting to Patterson, and he became convinced that he could help.

In preparing for the previously mentioned pitches to OrbiMed, Patterson worked up a vision for how much he needed to raise to accomplish milestones meaningful to VC investors. This is an important part of building early-stage businesses, because when you think about financing, you have to think about how much money is needed to comfortably get to milestones that will produce additional value, as VCs look at it from the perspective of what they are going to get for their money and in what time frame, he explains. This is where working at OrbiMed helped refine Pattersons knowledge, making it easier for him to think about how to pitch an opportunity. Nonetheless, even with this wisdom and pitching to people he knew it remained a challenging conversation.

By the fall of 2012, Patterson had become even more passionate about the company, which by then even had a name Audentes Therapeutics. For those Latin nerds, Audentes means those who have courage, those who have boldness or daring, he notes. Patterson says he consulted his mother (a former Latin teacher) on the name to make sure his grammatical use of the word was accurate. This also explains how the company eventually came up with its NASDAQ ticker symbol, BOLD. The courage theme always resonated with me when I think about the patients and families within the rare disease communities, so I wanted a word that captured that.

THE BUILD ITS NEVER AS EASY AS YOU THINK

After officially founding the company in November 2012, Patterson worked out of his apartment in Manhattan for about six months before moving to San Francisco. I always knew that if I wanted to build an innovative biotech, I needed to put it where I could hire really talented people, and for me, at the time, that was either Boston or San Francisco. Having worked in both regions, Patterson also figured personal connections might prove important to getting those first few staffers on board. People say they like the idea of going to a cool, small startup, but the reality is its not for everyone, he states. Once prospects begin to kick the tires, its personal relationships and a history of working together that tend to make the difference.

But there was a lot to be done before he could begin recruiting. For example, he needed to have weekly calls with the scientists on both U.S. coasts and in France to make sure they were making progress that would keep Audentes on track from a development standpoint. He had to put sponsored-research agreements in place to help fund some of the work that needed to happen to continue to prove the company and its compounds had potential. There were other licenses that were needed to enable the technology to go forward and make the investment story more compelling, he elaborates. So, Patterson started a conversation with another company that had IP related to the vector Audentes wanted to use and convinced them not to force him to pay up front for a license. I pitched the company and my background, and thankfully they thought it credible, he shares. The two agreed on terms that deferred payment until Patterson landed the Series A funding. This, along with securing licenses for any IP developed by the academic scientists he was working with, was pivotal, because now he could comfortably say to VCs that he had key IP agreements in place. The clock was ticking, and all these things needed to happen in parallel, because the reality is a million bucks wasnt going to last very long.

Next, Patterson set about trying to improve his PowerPoint slide deck for the 2013 J.P. Morgan (JPM) healthcare conference in San Francisco. As an executive of Amicus for many years, the JPM environment wasnt new. But instead of getting a room at the Westin St. Francis [the host hotel for the annual gathering] and getting one-on-ones all day with investors, it was me lugging around my laptop and meeting with VC contacts in the lobby of the nearby Hilton with dozens of people around us screaming their own investment stories, he relates. It was an experience that helped him better understand what did and did not resonate with investors which in this case was primarily gene therapy. I heard a lot of different excuses for why people didnt want to invest in gene therapy, but essentially most of them were just saying it was too risky.

Over the next four to five months, Patterson spoke with about 25 VCs with little progress. In retrospect, given the profile of what I was trying to do, the best use of my time would have been with firms that had a history of investing in innovative science at an early stage, because at that time there was really only a handful of VCs interested in early stage, with most of them clearly being more comfortable with programs already having proof-of-concept. But where he did make progress was when he finally connected with Kush Parmar, M.D., Ph.D., at 5AM Ventures. He was a relatively new member of the 5AM team at the time, but lucky for me, he was personally interested, as he had been doing some work on gene therapy and was aware of the advancements made in academic research as well as its potential in rare diseases, Patterson grins. With Parmar as a champion, 5AM was immediately interested, which helped bring OrbiMed further along, as the two had a history of working together. And while OrbiMed, led by Silverstein, stepped up to lead the Audentes Series A, at a $30 million round, they wanted Patterson to find yet one more firm. He continued his dialogue with multiple firms, but with the credibility of OrbiMed and 5AM backing, it suddenly got easier. Once theres momentum in a deal, VCs will often follow each other, the CEO notes. In the end, we were glad to add Versant Ventures as our third, and that became the Series A, closing in July 2013, with OrbiMed investing $15 million, and 5AM and Versant splitting the other $15 million, and all three joining the Audentes board.

HOW A TOTAL FAILURE BECAME A SOURCE OF SUCCESS

Another round of financing was completed in the fall of 2014. At the time, gene therapy was gaining more acceptance as a powerful treatment option for a range of rare diseases, so Patterson started planning to expand the companys pipeline to help mitigate risk and increase the impact of their therapies. And then, the unthinkable happened. We had a total failure of the manufacturing of the lead product in late 2014, which was being done at an academic center, the executive explains.

When he started the company, Patterson felt confident he understood the manufacturing component. After all, biologics manufacturing at Genzyme and BioMarin was where he got his start. Turns out I didnt know as much as I thought, because on the surface gene therapy looks a lot like a traditional biologics manufacturing processes, but the manufacture of AAV gene therapy products was far more complex scientifically than I had initially appreciated, and that was a tough lesson to learn, he states. Further, there was no one in the world that knew how to do it at the scale necessary to run a clinical development program and eventually be commercial. Obviously, the company needed to find a new way. The initial thought was to come up with a small-scale process and work with a CMO. But heres the problem. First, there were no CMOs that knew how to make AAV gene therapy products at the time. Second, if the company did decide to go the CMO route, it would be a long-term collaboration with Audentes essentially teaching the CMO gene therapy process development and scale up. I realized this was a really important turning point in building the company, he relates. Because how to manufacture this type of product, do it well, and at large scale was something that was going to become a big issue, especially considering we were seeing more gene therapy companies being started.

Instead of lamenting over this setback, Patterson viewed it as an opportunity, akin to the biotechs of the 1980s and 90s working on innovative technologies and having to build their own internal manufacturing capabilities. Similarly, we realized this was our moment to be a leader, and we recognized the importance of owning that manufacturing capability, he shares. But there was just one problem he had to convince a board composed primarily of VCs that the money they had just invested should go toward building an internal, large-scale GMP manufacturing facility. That wasnt the plan during the fundraising round, and it wasnt a plan normally supported by investors. Still, Patterson was able to convince them of the incredible importance of having such capabilities to the eventual success of the business. It was the most important strategic decision weve ever made as a business and probably my greatest success as a CEO, he smiles.

The company found an old decommissioned GMP plant in South San Francisco that was really just a section of a warehouse that had been used for biologics manufacturing. We invested $15 million into the facility and leased the adjacent space to be able to expand capacity down the road, Patterson says. Taking such a staged approach made the move more financially feasible for the business. It also made it easier to pitch to the board of directors, who thankfully had the courage to support it, he adds. Its a tired phrase in biologics that the process is the product, but it is very much the case in gene therapy.

That same year, the company began to consider another big move. It was clear we were building a capital-hungry enterprise, which meant going public so we would have the funds needed to continue to build the business the way we knew it needed to be built, he states. After a mezzanine financing round, Patterson and his team began the laborious task of choosing banks for the IPO. It was important to have the panache of at least one larger bank, but I also wanted to make sure it was a bank that would fight for us if times got tough, he says. He also wanted a couple of smaller banks that he felt would work hard to get the deal done, as there were signs the markets would be a little more uncertain in 2016. The syndicate ended up consisting of Bank of America (BofA), Merrill Lynch, Cowen and Company, and Piper Jaffray as joint book-running managers for the offering, and Wedbush PacGrow as acting comanager.

The first quarter of 2016 turned out to be the worst quarter in biotech in many years. But the additional private financing gave Audentes the runway to be patient, and it was finally able to complete the IPO in the summer of 2016.

The banks, of course, wanted Patterson to immediately embark on a road show to announce the deal, but they were surprised to see three weeks blocked off in June on his calendar. He explained he was getting married and didnt believe cancelling his honeymoon in the interest of the IPO would be good for his well-being. Thankfully, all the banks agreed, he shares. So, it turned out to be a pretty fun and exciting six-week window, both personally and professionally.

Today, Audentes Therapeutics is a publicly traded company valued at nearly $1.8 billion.

However, there are other measures of success. For example, in September 2017, Audentes initiated a clinical trial of its lead program, AT132 for XLMTM, and positive results to date led them to announce plans to file for approval of that product in mid-2020. In the meantime, the company has expanded its pipeline of neuromuscular targeted programs, going after Pompe disease, Duchenne muscular dystrophy, and myotonic dystrophy and has grown to more than 250 employees. Throughout the last seven years, weve gone from humble beginnings to where we are today, and while Im sure we have a long road ahead of us, I am very proud of what it has become, Patterson concludes.

When Matt Patterson was at OrbiMed Advisors working as an entrepreneur in residence (EIR), he became enamored with the idea of founding a gene therapy company. He admits, though, that there were plenty of other opportunities that he could have pursued considering the amazing number of technologies and company profiles that flowed through OrbiMed. I had looked at a couple of later-stage companies, as OrbiMed wanted me to be pitching a company that was already in the clinic with some data, he says. But he had made up his mind that he was only interested in finding an opportunity where he could work as CEO. He says that if a company was in the clinic with data that looked good, and they were looking for a CEO, then something else must have been wrong. A fixer-upper wasnt interesting to me, and I didnt want to be a hatchet man whose job it was to fire most of the team and refocus the company, he explains.

He did find some interesting rare-disease assets at some larger companies that he thought were, frankly, not well-suited to advance, and so he set out to try to convince those companies that those assets would be better off in his hands. For example, when Bristol-Myers Squibb (BMS) bought Amylin Pharmaceuticals, Amylin had an orphan drug product called metreleptin for a genetic disorder. I tried to convince BMS that they should out-license that program to me. The conversation lasted about three days, meaning two interactions of, Thats interesting. Well get back to you. and then, No. In the end, he stayed committed to his original plan and ended up starting a new company, Audentes, around the X-linked myotubular myopathy (XLMTM) program he discovered at Wake Forest University.

Throughout the course of growing Audentes Therapeutics, Matt Patterson admits to taking the concept of culture very seriously, but he wasnt overly formal about defining it especially in those early years. Ive always been fascinated by the fact that, in biotechnology, one could have a brilliant scientific hypothesis and vision, but the idea goes nowhere because you failed to hire and retain a talented team, he mentions. Its critical that people enjoy their work and are inspired to put in the effort needed to be successful. I was always paying attention to our culture. We had a high rate of employee retention, and everything seemed to be going well, he elaborates. But when the company reached the 100-employee mark, he felt culture was something to be discussed a little bit more formally. We spoke about it at a leadership team meeting and attempted to engage the employee base more broadly in the issues they found important, so theyd have a voice, and we could learn.

The company also did various surveys and small group meetings that were really productive, but in the end, Patterson says neither fundamentally changed the companys overarching vision or mission as a business. All of those exercises led to the creation of three core values, although Patterson admits he was skeptical when they began creating them. Ive been at companies where they went through that exercise, and it became something like a 3x5 card that sat on the desk of every employee but was never truly embraced. He recalls starting the first meeting by saying, I dont think this is going to be a good use of time, and the last thing I want is to come up with a few catchy phrases that management thinks sound good but really dont resonate, as that seems silly. Id rather publish nothing than have that outcome. But as the program evolved and the group began getting into the brass tacks of core values and how to make them real, he became more enthusiastic. We came up with three that we felt were meaningful, could be acted upon, and made an example of on a daily basis.

The first is to be bold, find a way. This means striving to really figure out how to solve a tough challenge, whether scientific, clinical, or other. The second is to care deeply about patients, the work, and the team. The third, which is about being focused and working hard every day to achieve the companys goals, is #GSD, which stands for get stuff done, though Patterson shares that they tend to use a four-letter word in place of stuff internally. We felt those three captured who we were and who we wanted to be, he says. When someone does something outstanding to solve a challenge or to help a teammate, Audentes might recognize them with a Boldy, a symbolic award that includes a small financial component.

They implemented the three core values about 18 months ago, and ever since then they seem to come up all the time. When employees use them casually and regularly without management telling them to, thats the most important validation, he contends. And though this CEO went into meetings about culture and core beliefs as a bit of a skeptic, he says he came out a believer.

Link:
Building A 1 Billion Gene Therapy Company In 6 Years - Life Science Leader Magazine

GAITHERSBURG, Md.--(BUSINESS WIRE)--The Maryland Tech Council (MTC) is preparing for one of its most highly-attended conferences of the year, the Bio Innovation Conference, presented by Maryland Life Sciences (a division of MTC). The Bio Innovation Conference provides a forum for professionals from industry, academia and government to discuss trends and insights into Marylands growing life sciences industry, while showcasing the states and regions innovations and successes in the life sciences industry. The all-day event attracts more than 400+ top life science professionals and takes place on October 7, 2019 at the Bethesda North Marriott Hotel & Conference Center.

Were experiencing an exciting time in the region, with wave upon wave of innovations, collaborations, acquisitions, tech transfer and growth, said MTC CEO Martin Rosendale. Companies from all over the country are moving here to experience all that Maryland and the region have to offer. This conference provides the perfect backdrop to showcase it all and discuss how to further propel us to the No. 1 spot among life sciences hubs.

This years keynote speakers include, John Tisdale, MD, Chief, Cellular and Molecular Therapeutics Branch National Heart, Lung, and Blood Institute, National Institutes of Health, and Michelle McMurry-Heath, MD, PhD, Vice President, External Innovation, Global Leader for Regulatory Science and Executive Director of Scientific Partnerships for JLABS @ Washington, DC. Maryland Secretary of Commerce Kelly M. Schulz will give opening remarks, and Montgomery County Executive Marc Elrich will help close out the conference.

The conference will also highlight the cell and gene therapy and biomanufacturing revolution taking place in Maryland and throughout the region. Notable speakers in the cell and gene therapy/biomanufacturing space include:

Were pleased that the Maryland Tech Council selected Montgomery County as the location for this important conference, said County Executive Elrich. The life sciences sector is thriving here, so its fitting that the conference is taking place in our county. My administration is focused on creating more opportunities for new and emerging businesses to grow and locate here.

This year for the first time, attendees will be able to simplify the process of searching for, identifying, and meeting with potential partners and business development executives with the BIO One-on-One Partnering system.

Maryland is continuing to make major strides in cell and gene therapy, as well as biomanufacturing, said Secretary Schulz. This conference offers a great opportunity to bring together our best and brightest industry leaders to take a closer look at how the industry is evolving and how Maryland is leading the way.

For more information or to register, visit: marylandlifesciences.com/conference/about/.

About Maryland Life Sciences

Maryland Life Sciences (MDLS), a division of the Maryland Tech Council, is a regional association for the life sciences community. We support our member companies who are driving innovation through advocacy, education, workforce development, cost savings programs and connecting entrepreneurial minds. MDLS represents biotechnology, clinical and research data, therapeutic, genetic, medical device, pharmaceutical and service companies that support Marylands thriving industry. The valuable resources we provide to our members help them reach their full potential making Maryland a global leader in the life science industry. For more information: mdtechcouncil.com/communities/life-sciences/.

About Maryland Tech Council

The Maryland Tech Council (MTC) is a collaborative community that is actively engaged in building strong technology and life science industries by supporting the efforts of our individual members. We are the largest technology and life sciences trade association in the state of Maryland, and we provide value by giving members a forum to learn, share, and connect. MTC brings the regions community together into a single, united organization that empowers our members to achieve their business goals through advocacy, networking and education. The vision for the Maryland Tech Council is to propel Maryland to become the number one innovation economy for life sciences and technology in the country. For more information: mdtechcouncil.com.

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Maryland Life Sciences Bio Innovation Conference Highlights Region's Growth with Emphasis on Cell and Gene Therapy, Biomanufacturing - Business Wire

In a significant setback, Vivek Ramaswamy-backed Arbutus Biopharma is ceasing the development of its experimental hepatitis B therapy, after two healthy volunteers contracted serious liver infection in an early-stage trial.

The therapy, AB-506, is an oral hepatitis B virus (HBV) capsid inhibitor designed to thwart viral reproduction, versus existing standard therapies primarily nucleoside analogues that are designed to diminish viral replication.

The two subjects are experiencing resolution of their acute hepatitis. We will continue to follow them and the other study participants said Arbutus chief development officer Gaston Picchio said in a statement on Thursday afternoon.

The companys shares $ABUS sank about 30% to $1 in Friday premarket trading.

The Phase Ia/Ib trial was testing AB-506 in healthy subjects as well as patients with chronic HBV. and HBV-DNA positive subjects with chronic HBV infection. The tranche of healthy volunteers were given AB-506 doses ranging from 30-1000 mg while chronic HBV patients were given different doses of AB-506, with or without a nucleoside analogue.

In July, Arbutus provided a positive update on the trial, reporting that no serious adverse events or clinically significant safety findings were observed in the 33 healthy subjects, adding that ALT levels and other liver function tests were normal throughout the ten days of dosing. Although there were no serious side-effects observed in the group of 24 chronic hep B patients, there were four cases of ALT flares.

On Thursday, the Canadian company provided an update after 28 days of dosing and said the full dataset would be unveiled at a scientific conference later this year.

Terminating the AB-506 program also means Arbutus $ABUS will not initiate a combination study of AB-506 and its experimental RNAi therapy, AB-729, in the second half of 2020, it added.

AB-506 is Arbutus latest casualty. Last October in an announcement unveiling the appointment of Picchio the company said its experimental HBV RNA de-stabilizer, AB-452, will be delayed entering Phase I studies, after some long-term toxicity issues in animal studies emerged.

(T)his latest setback, raises questions about the companys small molecule design capabilities, Chardan analysts wrote in a note on Friday.

Arbutus, formerly known as Tekmira, pivoted from its Ebola effort to hep B after the acquisition of OnCore from a group of Pharmasset vets in 2015. In 2017, crushed by the failure of his Alzheimers bet, Roivants Vivek Ramaswamy who was already an investor invested a further $116.4 million at a premium rate to acquire preferred shares in Arbutus. Last year, Ramaswamy and Arbutus joined forces to spawn Genevant Sciences to develop a slate of RNA-based therapeutics backed by Arbutus proprietary lipid nanoparticle and ligand conjugate delivery technologies.

Arbutus Lipid Nanoparticle technology, which enables RNAi drugs to be encapsulated in tiny particles made of lipids that travel through the bloodstream to target tissues, was used by Alnylam $ALNY to develop its pioneering RNAi therapy, Onpattro, securing a stream of royalty revenue for Arbutus.

See more here:
Two acute cases of liver disease in healthy subjects kill Arbutus' hep B program - Endpoints News

From Matthew Knowles revealing a male breast cancer and BRCA2 diagnosis to a sarcoma survivor donating thousands of toys in lieu of birthday presents, heres what is making headlines in the cancer space this week.

In an interview with Good Morning America, the music executive described how he first started to see blood on his T-shirt, then his wife noticed it on the bedsheet. Thats when he went to the doctor and a mammogram, ultrasound and biopsy confirmed it was stage 1a breast cancer.

He underwent surgery in July and also had genetic testing done to see if he carried the BRCA gene mutation, which he tested positive for, raising his risk of not only breast cancer, but also melanoma, prostate and pancreatic cancer. I am going to get the second breast removed in January, because I want to do anything I can to reduce the risk, Knowles said during the interview. We use the words cancer-free, but medically theres no such thing as cancer-free. Theres always a risk. My risk of a recurrence of breast cancer is less than 5%, and the removal of the other breast reduces it down to about 2%.

The first call he made was to his kids and former wife. Each child has a 50/50 chance of inheriting a BRCA mutation from their parent. Knowles said he is speaking out to inspire other men to be open about male breast cancer.

A tradition at Boston Childrens Hospital took center stage this week as the hospital turned 150. The hospital looked back on a former patient who was treated for neuroblastoma at 2 years old. When Avery McAvoy was discharged, the nurses and caregivers held a bubble parade something they do for all pediatric patients who are released.

Avery is now 6 years old and started first grade this year. She is still cancer-free.

People around the world are encouraged to Lip Sync for Lymphoma. The aunt of Kevin Siddall, who died after a six-month battle with non-Hodgkin lymphoma at 14 years old, came up with the idea to raise money for research.

Those who wish to participate can record themselves lip synching their favorite song, then upload it to a website. They can then ask friends and family to vote for them, which costs one dollar. Participants can also challenge others to join in the fun.

The top 10 performers will battle it out on stage on Oct. 17.

A 5-year-old cancer survivor forgoes birthday presents to donate toys to other kids at the childrens hospital where he was treated.

Weston Newswanger received a rhabdomyosarcoma diagnosis in November 2016 and his mom said that toys became a big part of keeping him happy during treatment.

When she asked him what he wanted for his birthday he said, I dont want anything. I dont need anything, reported CNN.

Family and friends made his wish of giving dinosaurs and Play-Doh a reality by collecting more than 3,000 items. Newswanger and his mom delivered the toys on Tuesday to PennState Childrens Hospital.

Original post:
Friday Frontline: Cancer Updates, Research and Education on October 4, 2019 - Curetoday.com

The global biologics market 2017 was estimated to be USD xx Million. It is anticipated to increase to a value of USD xx Million with a CAGR of xx% over the forecast period. Continuous activities in research and development, investment in biologics and occurrence of chronic diseases are the factors that are playing a major role in the growth of biologics market. As per WHO, chronic diseases will account for approximately 80% of death by 2020 across the world. Constant development in gene & cellular therapy is catalyzing the market because of its therapeutic outcome and high efficiency. CAR-T-Cell are therapies recently accepted by FDA for the indications of oncology.

Request for sample copy of Biologics Market reportat:https://www.adroitmarketresearch.com/contacts/request-sample/165

Drugs are very complicated and need maintenance and controlled conditions for production and development processes. Huge finances are required to regulate the procedures of quality control initially. Biologics are prone to heat, light and require positive atmosphere which is not available worldwide. Strict rules and investment with restricted access to patient for biologics, particularly in emerging countries will affect the growth of global biologics market.

Biopharmaceuticals is getting popular over chemically synthesized molecules which are projected to increase the generation of revenue considerably. Moreover, by using the biologics many metabolic disorders can be treated to increase the demand of biologics market. Developed bioengineering technologies as well for the production of biopharmaceutical is estimated to fuel the pharmaceutical industry. With the advancements in automation, process of assortment can be done with the help of high throughput screening (HTS) system for the selection of duplicates.

Read more details of the report at:https://www.adroitmarketresearch.com/industry-reports/biologics-market

Biologics market trends are initiative by government to support biologic drugs that allow funding and investment in research and development eventually increasing the quality of biologic drugs. This will positively upsurge the demand for biologics market growth. Success rate of biologic drugs is increasing thus increasing the demand for manufacturing biologics amongst the big pharmaceutical companies. For example, Bristol-Myers Squibb capitalized about $800 Million in Irish large-scale biologics facility and Novartis AG capitalized about $750 Million in biologics facility in Singapore estimating to complete the production by the end of 2019. Accepting innovative therapies and increase in chronic diseases are the factors that are fueling the growth of biologics market.

Global biologics market is segmented on the basis of application, source, type and region. Based on application, biologics market is divided into cancer, anemia, diabetes and many more. By source, market is divided into animal, human and microorganism. Based on type, biologics market is splited into blood products, gene therapy, vaccines, proteins, monoclonal antibodies and much more.

Topographically, regions involved in accelerating the biologics market share are Asia Pacific, Europe, U.S, South America, North America and Middle East & Africa. U.S biologics market is dominating the market in North America with huge investment and research and development. Growth in the concerns and awareness regarding oncology and its treatment will help to surge the global biologics market in North America. Asia Pacific is propelled to expand rapidly due to less strict rules for the clinical trials of drugs, developing awareness of biologics for the treatment of chronic diseases and manufacturing of biologics in the developing countries.

Key Segments in the Global Biologics Market are-

By Application market is segmented into:

By Source market is segmented into:

By Type market is segmented into:

By Regions market is segmented into:

What to expect from the Global Biologics Market report?

Predictions of future made for this market during the forecast period.

Information on the current technologies, trends, devices, procedures, and products in the industry.

Detailed analysis of the market segmentation, depending on the types, devices, and products.

Government regulations and economic factors affecting the growth of the market.

An insight into the leading manufacturers.

Regional demographics of the market.

Who should buy this report?

Venture capitalists, Investors, financial institutions, Analysts, Government organizations, regulatory authorities, policymakers ,researchers, strategy managers, and academic institutions looking for insights into the market to determine future strategies

Enquire more details of the report at:https://www.adroitmarketresearch.com/researchreport/purchase/165

About Us:

Adroit Market Research is an India-based business analytics and consulting company incorporated in 2018. Our target audience is a wide range of corporations, manufacturing companies, product/technology development institutions and industry associations that require understanding of a markets size, key trends, participants and future outlook of an industry. We intend to become our clients knowledge partner and provide them with valuable market insights to help create opportunities that increase their revenues. We follow a code Explore, Learn and Transform. At our core, we are curious people who love to identify and understand industry patterns, create an insightful study around our findings and churn out money-making roadmaps.

Read the rest here:
Global Biologics Market 2019 Research Analysis, Current Trends, Regional Demand, Production Growth, Detailed Overview and Forecast Outlook by 2025 -...

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market (3rd Edition), 2019-2030" report has been added to ResearchAndMarkets.com's offering.

The Gene Therapy Market (3rd Edition), 2019-2030 report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, and genome editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

Since the approval of the first therapy, Genedicine in 2003, the gene therapy domain has evolved significantly. Specifically, in 2019, three gene therapies, namely Zolgensma (US), Zynteglo (Europe) and Beperminogene Perplasmid (Japan), have received approval / conditional approval, leading to a marked upward surge in the interest in this field.

In fact, the growing popularity can be correlated to the substantial increase (more than 90%) in the number of patents that have been filed/granted in the last three years. Moreover, in the same time period, more than USD 12.5 billion in the capital has been invested by various private and public investors to fund research activities. Presently, there are more than 10 approved gene therapies in the market, while many others are being investigated across various phases of clinical research.

Over time, the efforts of industry stakeholders and clinical researchers have led to the discovery of novel molecular targets, thereby, strengthening the research pipelines of companies involved in the development of gene therapies. Further, several technology developers have designed innovative ways to improve the efficacy and safety of gene therapies and introduced advanced therapy development and vector engineering platforms.

It is also worth mentioning that, in the last 4-5 years, there has been a marked rise in the M&A activity in this domain, including the involvement of several big pharma players as well. The capability of such therapies to target diverse disease indications is considered to be amongst the most prominent growth drivers of this market. Backed by promising clinical results and several therapy candidates in late phases of development, we believe that the overall market is expected to witness tremendous growth in the coming decade.

One of the key objectives of the report was to estimate the existing market size and the future opportunity for gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates, and expected pricing, we have provided informed estimates on the evolution of the market for the period 2019-2030.

The report also features the likely distribution of the current and forecasted opportunity across:

In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry's growth.

Amongst other elements, the report features:

Key Topics Covered:

1 Preface

2 Executive Summary

3 Introduction

4 Gene Delivery Vectors

5 Regulatory Landscape And Reimbursement Scenario

6 Competitive Landscape

7 Marketed Gene Therapies

8 Key Commercialization Strategies

9 Late Stage (Phase Ii/Iii And Above) Gene Therapies

10 Emerging Technologies

11 Promising Therapeutics Areas

12 Patent Analysis

13 Mergers And Acquisitions

14 Funding And Investment Analysis

15 Cost Price Analysis

16 Big Pharma Players: Analysis Of Gene Therapy Related Initiatives

17 Market Forecast And Opportunity Analysis

18 Vector Manufacturing

19 Case Study: Gene Therapy Supply Chain

20 Conclusion

21 Interview Transcripts

22 Appendix 1: Tabulated Data

23 Appendix 2: List Of Companies And Organizations

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

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Global Gene Therapy Market, 2019-2030: Current Market Landscape of Gene Augmentation-Based Therapies, Oncolytic Viral Therapies, and Genome Editing...

Gene editing will be tested in UW Medicine labs on kidney organoids tiny, kidney-like structures grown from stem cells as part of a federally funded effort to develop safe, effective genome editing technologies and therapies.

The National Institutes of Health today, Oct. 1, announced the next set of grant awards for the Somatic Cell Genome Editing consortium, created in 2018. Somatic cells make up the bodys tissues and organs, such as the lungs or blood, in contrast to reproductive cells, like fertilized eggs. Alterations made to somatic cell DNA are not passed down to the next generation.

In the latest round of SCGE funding, twenty-four grants, totaling about $89 million over four years, been awarded across the country. They will fund studies to address the promises and challenges of genome editing in the search for new treatment or cures for a number of genetic disorders.

The human genome contains thousands of genes responsible for making proteins. In many inherited disorders, a variation in the DNA code means that an important protein is not made, or is not made correctly. The missing or faulty protein could result in serious health problems. Genetic editing would aim to change the DNA to enable cells to make a sufficient amount of the proper protein.

For one of the new SCGE projects, collaborative research will take place between the University of Washington School of Medicine lab of kidney disease researcher Benjamin Beno Freedman, assistant professor of medicine, Division of Nephrology, and the University of California Berkeley lab of Jennifer Doudna, professor of molecular and cellular biology.

As a group, Freedman and his fellow researchers bring together expertise in kidney organoids, kidney cell biology, and kidney diseases. Their collaborators at UC Berkeley are leaders in the field of genome editing, including CRISPR-Cas9 gene editing technology to cut and paste portions of DNA in living cells.

Freedmans lab at the UW Medicine Institute for Stem Cell and Regenerative Medicine grow stem cell-derived organoids to study how kidney diseases begin and how they might be treated. Human kidney organoids and kidney-on-a-chip technologies (in which some functions of kidneys are simulated with living cells in tiny chambers) are providing useful medical information. For example, researchers have found new molecules that can reduce the signs of disease in these laboratory models.

Human kidney organoid showing podocytes (red) and proximal tubules (green) developed in the Freedman lab

Freedman explains the importance of exploring responsible gene-editing therapies for inherited kidney diseases: Genetic kidney diseases impact more than half a million people in the United States alone. If we can learn to safely repair the mutation that causes the disease, we can offer a way to treat patients that is much more effective than any current intervention.

Freedman emphasizes that dialysis and transplants two of the most common treatments for kidney diseases are expensive and hard on patients. Kidney transplants are in short supply; donor organs become available to less than 20 % of the patients who need them each year.

The shortcomings of dialysis and transplants make gene therapy an appealing area of research because it might get to the root of the problem.

One of the primary aims of the NIH-funded somatic cell genome editing explorations are to reduce the chances that gene editing produces unintended side effects that do more harm than good. In their collaborative project with UCBerkeley, the UW Medicine team will screen different gene therapies for their effects on normal kidney function and for risks of renal cancer or autoimmune disease.

Our hypothesis is that gene editing will cause adverse effects, but that these effects are predictable and controllable, says Freedman. Our goal is to prove this using laboratory models like organoids and kidneys on chips so we know the approach is safe before we ever involve a human patient.

Freedmans lab is in the Division of Nephrology, Department of Medicine, at the UW School of Medicine, and his lab is also part of the Kidney Research Institute, a collaboration between Northwest Kidney Centers and UW Medicine.

Joining Freedman on the UW Medicine research team are Institute for Stem Cell and Regenerative Medicine colleagues Hannele Ruohola-Baker, professor in biochemistry, and Julie Mathieu, assistant professor of comparative medicine, both at the UW School of Medicine.

Ruohola-Baker will investigate how genome-editing therapies affect cell metabolism. Mathieu adds CRISPR expertise to the UW research team. Several faculty members from other departments are also on the team.

How broad are the implications of developing responsible genome-editing methods?

This is a new paradigm for therapy development, says Freedman. Were looking at the kidney. But the liver, heart, and lungs all have similar challenges. Our hope is to create a model for doing this work in human organoids, which are faster and more humane than animal models, and can be more directly compared to human patients.

Genome editing has extraordinary potential to alter the treatment landscape for common and rare diseases, said Christopher P. Austin, director of the National Center for Advancing Translational Sciences and SCGE Program Working Group chair. The field is still in its infancy, and these newly funded projects promise to improve strategies to address a number of challenges, such as how best to deliver the right genes to the correct places in the genome efficiently and effectively. Together, the projects will help advance the translation of genome-editing technologies into patient care.

Nearly 40 million Americans have chronic kidney disease, a family of progressive conditions that can come with widespread health complications, including a higher risk for heart disease. When kidneys fail, the primary interventions, dialysis and kidney transplants, are not cures. These treatments come with significant side effects and a heavy economic burden. Medicare costs average $114 billion a year total for the care of the nations patients with kidney failure. Altogether, kidney disease is the ninth leading cause of death in the United States.

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Thatcher Heldring of the Institute for Stem Cell and Regenerative Medicine contributed to this news report.

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Genome editing to be tested in kidney organoids - UW Medicine Newsroom

For the past five years, there have been more than 400 mergers and acquisitions with companies in the gene therapy, immuno-oncology, microbiome and orphan drugs therapeutic space in North America and that trend is likely to continue, according to a new analysis.

In its Deal-Making Trends in Pharma analysis, GlobalDatas research shows that in North America, the pharmaceutical industry saw twice the number of M&S activity that the European market saw. When compared to the Asian-Pacific (APAC) market, the U.S. market had seven times the number of M&A deals. Europe had three times as many acquisitions than APAC, GlobalData said.

From 2014 to the first half of 2019, there were 2,882 deals that fell within the gene therapy, immuno-oncology, microbiome and orphan drug space. These deals were worth more than $1 trillion, according to GlobalData. During this time frame, the largest deal types were in the immuno-oncology space with 270 deals in 2018 alone, GlobalData said. That represents a growth of 63% since 2014. Gene therapy showed similar growth of 62% with 133 deals in 2018 compared to 82 deals in 2014.

Jesus Cuaron, associate director of Cardiovascular and Metabolic Disease and Gender Health Pharma at GlobalData, said North America is not only the top region for mergers and acquisitions, but also across each therapeutic theme.

Acquisitions revolving around immuno-oncology assets were the most prominent across the three geographic regions, while acquisitions of microbiome therapies remained low across the board. In the APAC region, no acquisitions were made that involved significant microbiome assets, Cuaron said in a statement.

While many of the M&A deals were on the small side, there have been a number of mega-mergers over the past few years. Among those are Takedas $60 billion acquisition of Shire; Bristol-Myers Squibbs $74 billion deal for Celgene; and AbbVies $63 billion plan to acquire Allergan. Other notable deals include Novartis $8 billion deal for gene therapy company AveXis and Roches $4.8 billion bid for Spark Therapeutics. Other mergers and acquisitions over the past few years can be found here.

Pointing to the mega-deals like BMS bid for Celgene, Cuaron said large-value deals are becoming more common, which signals a new strategy in which stakeholders capitalize on the strengths of other companies. These large-value deals are game-changers for the pharmaceutical industry, Cuaron said, and will cause a ripple of activity within the top pharmaceutical companies.

According to its latest analysis, GlobalData said it anticipates the pharmaceutical industry will continue to be attractive for high volumes of deal activity in the future. That prediction, the analyst said, is based on the fact that the pharmaceutical industry has a track record of making deals with companies that focus on highly specialized biologics and other high-value therapies.

Looking at the potential future of M&A activity, Cuaron said companies are looking to boost their pipelines with more innovative assets and technologies. While licensing agreements are less expensive routes for companies to take to gain an asset, Cuaron said acquisitions are more desirable for most companies because those deals allow the acquiring company to gain complete control over the assets of interest. Cuaron noted that pharmaceutical companies that have few revenue drivers or face patent expiration or generic competition, will be the ones to more quickly flex their M&A muscle. For example, AbbVie has significantly benefited from Humira, which last year generated nearly $30 billion in revenue. However, Humira is facing a patent cliff in 2023 and was seeking a transformational move when it made the bid for Botox-maker Allergan.

At the time Allergan struck the deal, AbbVie said: Smaller bolt-on acquisitions provide opportunities for future growth, but also require significant R&D investment amid scientific and clinical uncertainty. This transaction offers immediate compelling financial and strategic value to our shareholders with a much lower risk profile, which echoes what Cuaron said.

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M&A Will Continue to Be a Mainstay for Growth in the Pharma Industry - BioSpace

From the NIH Directors Blog by Dr. Francis Collins

Its a race against time when someone suffers a stroke caused by a blockage of a blood vessel supplying the brain. Unless clot-busting treatment is given within a few hours after symptoms appear, vast numbers of the brains neurons die, often leading to paralysis or other disabilities. It would be great to have a way to replace those lost neurons. Thanks to gene therapy, some encouraging strides are now being made.

In a recent study in Molecular Therapy, researchers reported that, in their mouse and rat models of ischemic stroke, gene therapy could actually convert the brains support cells into new, fully functional neurons.1 Even better, after gaining the new neurons, the animals had improved motor and memory skills.

For the team led by Gong Chen, Penn State, University Park, the quest to replace lost neurons in the brain began about a decade ago. While searching for the right approach, Chen noticed other groups had learned to reprogram fibroblasts into stem cells and make replacement neural cells.

As innovative as this work was at the time, it was performed mostly in lab Petri dishes. Chen and his colleagues thought, why not reprogram cells already in the brain?

They turned their attention to the brains billions of supportive glial cells. Unlike neurons, glial cells divide and replicate. They also are known to survive and activate following a brain injury, remaining at the wound and ultimately forming a scar. This same process had also been observed in the brain following many types of injury, including stroke and neurodegenerative conditions such as Alzheimers disease.

To Chens NIH-supported team, it looked like glial cells might be a perfect target for gene therapies to replace lost neurons. As reported about five years ago, the researchers were on the right track.2

The Chen team showed it was possible to reprogram glial cells in the brain into functional neurons. They succeeded using a genetically engineered retrovirus that delivered a single protein called NeuroD1. Its a neural transcription factor that switches genes on and off in neural cells and helps to determine their cell fate. The newly generated neurons were also capable of integrating into brain circuits to repair damaged tissue.

There was one major hitch: the NeuroD1 retroviral vector only reprogrammed actively dividing glial cells. That suggested their strategy likely couldnt generate the large numbers of new cells needed to repair damaged brain tissue following a stroke.

Fast-forward a couple of years, and improved adeno-associated viral vectors (AAV) have emerged as a major alternative to retroviruses for gene therapy applications. This was exactly the breakthrough that the Chen team needed. The AAVs can reprogram glial cells whether they are dividing or not.

In the new study, Chens team, led by post-doc Yu-Chen Chen, put this new gene therapy system to work, and the results are quite remarkable. In a mouse model of ischemic stroke, the researchers showed the treatment could regenerate about a third of the total lost neurons by preferentially targeting reactive, scar-forming glial cells. The conversion of those reactive glial cells into neurons also protected another third of the neurons from injury.

Studies in brain slices showed that the replacement neurons were fully functional and appeared to have made the needed neural connections in the brain. Importantly, their studies also showed that the NeuroD1 gene therapy led to marked improvements in the functional recovery of the mice after a stroke.

In fact, several tests of their ability to make fine movements with their forelimbs showed about a 60% improvement within 20 to 60 days of receiving the NeuroD1 therapy. Together with study collaborator and NIH grantee Gregory Quirk, University of Puerto Rico, San Juan, they went on to show similar improvements in the ability of rats to recover from stroke-related deficits in memory.

While further study is needed, the findings in rodents offer encouraging evidence that treatments to repair the brain after a stroke or other injury may be on the horizon. In the meantime, the best strategy for limiting the number of neurons lost due to stroke is to recognize the signs and get to a well-equipped hospital or call 911 right away if you or a loved one experience them. Those signs include: sudden numbness or weakness of one side of the body; confusion; difficulty speaking, seeing, or walking; and a sudden, severe headache with unknown causes. Getting treatment for this kind of brain attack within fourhours of the onset of symptoms can make all the difference in recovery.

This research was supported in part by NIA grant AG045656.

References:

[1] Chen Y-C, et al. A NeuroD1 AAV-based gene therapy for functional brain repair after ischemic injury through in vivo astrocyte-to-neuron conversion. Molecular Therapy. 2019. Epub Sept. 6.

[2] Guo Z, et al. In vivo direct reprogramming of reactive glial cells into functional neurons after brain injury and in an Alzheimers disease model. Cell Stem Cell. 2014;14(2):188-202. doi: 10.1016/j.stem.2013.12.001.

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Gene therapy shows promise repairing brain tissue damaged by stroke - National Institute on Aging

When Michael Pirovolakis was born eight days before Christmas in 2017, his green eyes glowed bright like two snow globes.

He had 10 fingers, 10 toes and a full head of hair matted to his head. Michael was a cooing, crying, healthy baby boy.

What else could we ask for, said his father, Terry.

Terry, 39, and his wife Georgia, 41, became a family of five, tucked away in a suburb east of Torontos core.

Michael grew like any ordinary infant, his peanut-coloured hair becoming longer and curlier by the day.

But six months into his life, worry arrived.

Terry says he and Georgia looked at photos of Michaels siblings and began to realize he wasnt reaching the same developmental milestones. Michael wasnt using his hands, didnt crawl and hadnt formed words.

That observation led the family down countless hospital hallways, into waiting rooms and handshakes with many experts in white lab coats.

First, pediatricians noticed Michaels smaller head and a floppiness arising from missing muscle tone. Physiotherapy should have solved the problem, but there was no sign of improvement two months later.

Then, specialists at Torontos Sick Kids Hospital suspected Michael may have contracted the Zika virus from his fathers travels in South America and Puerto Rico for work.

It was pretty scary because they told us he might be blind and deaf, Terry said.

It was a false alarm, but the tests didnt stop. A neurologist said although it was nothing to worry about, the white matter that moves neural signals across Michaels brain was underdeveloped.

It was another dead end, but then came genetic testing and when the results arrived this spring, they contained the answer to every question keeping Michaels parents up at night.

April 2 was doomsday, Terry said.

It was the worst day of my life I saw on the doctors face something was wrong, so they sat us down and told us what it was. It was just an acronym, but then we started reading words like paralyzed, quadriplegic, limited brain function. I honestly dont even know how we got home.

We curled up in a ball and cried for a couple of hours.

SPG50

Michael became one of 61 people on the planet and the only child in Canada known to have spastic paraplegia 50, also called SPG50.

Its a rare disease that will leave Michael with a progressive cerebral palsy effect in his limbs while his mind will fail like a Parkinsons patient, according to Terry, all because of a missing protein in the AP4M1 gene, starving his sons brain.

Hes got this double whammy of stuff happening to him right now.

Michael has suffered seizures and as he ages, his muscles will morph from missing tone to having too much, robbing him of his ability to move. His brain, too, will lose function.

Terry and Georgia didnt waste anytime starting to scour the internet for any bit of information about the disease.

They tracked down a child in Boston living with a similar condition.

For two weeks, that family took care of us, Terry said. We took that information and started running.

He started flying around the world to speak with specialists and pharmaceutical companies, while attending conferences to learn how they could beat the race against time.

I asked them all the same question: If this was your kid, what would you do? he said.

If this was your kid, what would you do?

Many of them pointed to gene therapy and researchers from the University of Texas. After Terry begged them to look at Michael, the team of specialists agreed, saying they could create an experimental therapy to try to cure his son but it would cost $3 million.

Weve liquidated everything weve reversed our mortgage and have taken everything out to try to save our kid, Terry said.

Terry also started a GoFundMe campaign for his child, which went viral, raising $695,032 since it was posted at the start of May.

THE $3 MILLION CURE

On top of the money needed for the experimental treatment, Michael also needs daily physical therapy and occupational therapy, but the government cant cover all the sessions.

If the therapy wasnt experimental, theres a chance the government would fund it and if the condition wasnt as rare as it is, there might be more researchers in Canada searching for a cure but the only option is out of the country, creating another barrier for any kind of financial help.

Terry said he tried contacting Ontario Premier Doug Ford and Prime Minister Justin Trudeau, but didnt receive any support.

Canada as a whole needs to come up with something to help parents, he said. There has to be a mechanism for families like us that are desperate, spending their life savings.

As people around the world organize fundraisers, events, yard sales and even lemonade stands to help Michael, Steven Miller, the head of neurology at SickKids, said Terry is one of the parents leading what could be a revolution within labs.

Were seeing a movement now, especially in Canada, where researchers are working with parents to identify what the next steps are in research, Miller said.

As we wait for the new therapies to evolve, its important not to lose sight for what we can do to help kids with diseases for which there is no cure.

Terry said he spends his mornings talking to the media before going to work for the day and coming home to his family in the evening before researching online until 3 a.m.

Almost two years old now, Michael is walking with assistance and still smiling on his own.

And his family isnt quitting.

Email: bhristova@postmedia.com | Twitter:

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Race against time for family of toddler with ultra-rare disease, whose only chance at life costs $3 million - National Post

MALVERN, Pa, & TIANJIN, China--(BUSINESS WIRE)--Ocugen, Inc., (NASDAQ: OCGN), a clinical stage biopharmaceutical company focused on innovative therapies that address rare and underserved eye diseases, has entered into a strategic partnership with CanSino Biologics (CanSinoBIO)(6185.HK) on Ocugens gene therapy pipeline product candidates for inherited retinal diseases, which are currently in development with Schepens Eye Research Institute of Massachusetts Eye and Ear, an affiliate of Harvard Medical School.

Under this strategic collaboration, CanSinoBIO will provide all CMC development and clinical supplies for the development of OCU400, Ocugens first gene therapy product candidate in its modifier gene therapy platform. CanSinoBIO maintains the option to support commercial manufacturing for Ocugen. The agreement also provides commercialization rights to CanSinoBIO in Greater China.

We believe our modifier gene therapy platform, and OCU400 as its first product candidate, has the potential to treat many inherited retinal diseases with one product, said Shankar Musunuri, Ph.D., MBA, Chairman, CEO and Co-Founder of Ocugen. A reliable manufacturing partnership is critical for gene therapy clinical trials and commercialization. Partnership with CanSinoBIO, with their state-of-the-art facilities and world class team, provides us a clear path to advance our development and manufacturing processes to reach the clinic.

OCU400 has received two different orphan drug designations (ODD) from the U.S. FDA. The first, for the treatment of NR3E3 mutation-associated retinal degeneration and, most recently, for the treatment of CEP290 mutation-associated retinal disease.

We are delighted to partner with Ocugen as they advance their portfolio of AAV-based gene therapies for rare retinal diseases, said Dr. Xuefeng Yu, the Chairman and Chief Executive Officer of CanSinoBIO. Our expertise in viral vector platform technologies, product development and manufacturing capabilities will play critical roles to advance OCU400 to the clinic and ultimately to serve patients in desperate need for retinal disease therapies.

About OCU400

OCU400 is a novel gene therapy with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited degenerative retinal diseases. OCU400 is the first program that Ocugen is advancing based on its novel modifier gene therapy platform, developed by Neena Haider, PhD, Associate Professor of Ophthalmology at Harvard Medical School and Associate Scientist at the Schepens Eye Research Institute of Massachusetts Eye and Ear, from which Ocugen obtained an exclusive worldwide license to develop and commercialize ophthalmology products based on the platform. Consisting of a functional copy of the nuclear hormone receptor (NHR) gene NR2E3, OCU400 is delivered to target cells in the retina using an adeno-associated viral (AAV) vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, stabilizing cells and potentially rescuing photoreceptors from degeneration.

About Ocugen, Inc.

Ocugen, Inc. is a clinical stage biopharmaceutical company focused on discovering, developing and commercializing a pipeline of innovative therapies that address rare and underserved eye diseases. The Company offers a robust and diversified ophthalmology portfolio that includes novel gene therapies, biologics, and small molecules and targets a broad range of high-need retinal and ocular surface diseases. Ocugen is leveraging its groundbreaking modifier gene therapy platform to address genetically diverse inherited retinal disorders and dry AMD, based on nuclear hormone receptor genes NR2E3 (OCU400) and RORA (OCU410), respectively. OCU400 has received two orphan drug designations (ODD) targeting two distinct IRDs. Ocugen is also developing novel biologic therapies for wet-AMD, DME and diabetic retinopathy (OCU200), as well as for retinitis pigmentosa (OCU100). The Companys late-stage Phase 3 trial for patients with ocular graft versus host disease (oGVHD)(OCU300) leverages Ocugens patented OcuNanoE Ocugens ONE Platform technology to enhance the efficacy of topical ophthalmic therapeutics. OCU300 is the first and only therapeutic with ODD for oGVHD, providing certain regulatory and economic benefits. For more information, please visit http://www.ocugen.com.

About CanSino Biologics Inc.

Incorporated in 2009 in Tianjin, China, CanSinoBIO (6185.HK) commits to research, development, production and commercialization of innovative vaccines for China and global public health. It possesses four integrated platform technologies including viral vectors, conjugation, protein design and recombination and formulation. As of today, it has established a robust pipeline of 15 candidate vaccines covering 12 diseases, including a globally innovative Ebola virus disease vaccine approved for emergency use and stockpile in 2017. For more information, please visit http://www.cansinotech.com.

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the Companys current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in Histogenics Registration Statement on Form S-4 (Reg. No. 333-232147), as amended, filed with the SEC. Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release. The Company assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

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Ocugen and CanSinoBIO Enter Strategic Partnership for Gene Therapy Co-Development and Manufacturing - Business Wire

By Adam Vaughan

Bloomberg/Getty Images

Boris Johnson has said the UK can become carbon neutral by the middle of the century and beat the sceptics, in his speech to the Conservative party conference.

The prime minister touched repeatedly on climate change, green technology and science in his address, and suggested that nuclear fusion which the Tories promised 200 million in extra funding over the weekend was on the brink of a breakthrough.

They are on the verge of creating commercially viable miniature fusion reactors for sale around the world, he said of the JET fusion research centre at Culham in Oxfordshire.

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Now I know they have been on the verge for some time. It is a pretty spacious kind of verge, he joked, to laughter in the audience. While the UK is considered a world leader in fusion research, its continued role in a multibillion European fusion project in France has been called into question by Brexit.

In response to Johnsons speech, Tom Nicholas at the University of York says: I would say thats definitely a stretch. Culhams best case would be full technology demonstration by the 2040s, he adds, although even that wouldnt be a commercially viable fusion plant.

Johnson said that while people used to suggest that wind and solar power werent fit for the UK, on some days they were now supplying more than half of the countrys electricity needs.

People years ago said wind turbines would not pull the skin off a rice pudding, Johnson said. He didnt mention that the phrase was his own, which he used when he told The Telegraph in 2013 that the UK should back shale gas, not wind power. Solar power has stalled in the UK and onshore wind farms are no longer being built on the mainland because of policy changes brought in by the Conservative government in 2015.

The prime minister also said the UK was leading on low carbon and battery technology, and said the UK would build a gigafactory, a reference to the sort of large-scale battery factory thatTesla has built in the US. The Labour party used its conference last month to promise three such gigafactories. Battery industry figures have told New Scientistthat the UK is an unlikely candidate for such a facility in Europe, with Germany seen as a much more likely location.

Johnson also promised more zero emission buses and better road and rail links, though he notably didnt mention the High Speed 2 rail project, which is under a government-commissioned review.

The prime minister praised UK leadership in science, saying Manchester led on genomics, gene therapy was being used to cure blindness, and the UK was building two spaceports, one in Newquay and one in Sutherland.

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Boris Johnson jokes about UK being 'on the verge' of nuclear fusion - New Scientist News

MARTINSRIED, Germany--(BUSINESS WIRE)--

CEO Christian Thirion to Present at Cell and Gene Meeting on the Mesa, California

Exhibition Booth at BioJapan and European Biotech and Pharma Partnering Conference, Japan

Exhibition at ESGCT Annual Conference in Conjunction with ABC of AAV, Spain

SIRION Biotech GmbH (SIRION) today announced its participation in major international conferences this October. SIRION, offering the most comprehensive portfolio of custom viral vectors for preclinical and clinical studies, will present at the Cell and Gene Meeting on the Mesa in Carlsbad, California on October 2 4, and exhibit at the European Biotech and Pharma Partnering Conference in Osaka, Japan on October 8, BioJapan in Pacifico Yokohama, Japan on October 9 11, and the ESGCT 27th Annual Congress on October 22 - 25 in Barcelona, Spain.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20191001005658/en/

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is the sectors foremost annual conference gathering industry leaders and decision-makers to bring forward the state-of-the-art research developments to medicines. Dr. Christian Thirion, CEO, will discuss novel therapeutic viral vectors and technology platforms based on lentivirus, adenovirus, and adeno-associated viruses, which expedite gene therapy research and advances drug development, during his company presentation on Wednesday, October 2 at 4:45 p.m. in the Cognate Bioservices Ballroom at the Park Hyatt Aviara Resort in Carlsbad, CA. A live video webcast of all company presentations will be available at: http://www.meetingonthemesa.com/webcast and will also be published on the conference website shortly after the event.

The Osaka European Biotech and Pharma Partnering Conference offers business contacts to industry professionals who are looking for potential partners in the EU and Japan to ensure sustainable business development and growth. BioJapan is Asias premier partnering event for the global biotech industry. SIRION will be exhibiting at both of these meetings, at the Pacifico Yokohama at D-53. Organized by European Society of Gene and Cell Therapy, the ESGCT Annual Congress in Barcelona promotes basic and clinical research in gene therapy, cell therapy and genetic vaccines by facilitating education, the exchange of information and technology, and by serving as a professional adviser to stakeholder communities and regulatory bodies in Europe. SIRION will exhibit at this meeting at the Barcelona INTERNATIONAL Convention Centre at level P2, booth 20 as part of the ABC of AAV, offering everything you need for gene therapy in one place.

About SIRION Biotech GmbH SIRION Biotech founded in 2005 in Martinsried near Munich, Germany with the goal to skyrocket a new generation of viral vector technologies for gene & cell therapy as well as vaccine development. SIRION advances novel therapeutic viral vectors and uses proprietary technology platforms based on lenti-, adeno-, and adeno-associated viruses, to facilitate its partners successes in drug development. SIRIONs LentiBOOSTTM transduction reagent is actively used to enhance, among others, hematopoietic cell transductions in clinical trials. NextGen AAV capsid evolution projects focus on tissue targeting and immune escape of capsids to accelerate a next generation therapeutics for gene therapy companies. For additional information, please visit http://www.sirion-biotech.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191001005658/en/

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SIRION Biotech to Participate in Major International Industry Conferences - Yahoo Finance

In an attempt to fulfill the lofty ambitions of the original Human Genome Project, Duke University engineers, scientists and physicians are joining forces in a new initiative to develop the missing technologies needed to understand the tangled genetic webs underpinning many of humanitys most stubborn diseases and unearth new drug targets and gene therapies to fight them.

The complexity of human biology and the big data stemming from it have all but walled off efforts to comprehensively understand the functions of most of the human genome. To scale these walls, the Duke Center for Advanced Genomic Technologies (CAGT) will build upon its facultys long track record of pioneering tools and techniques to probe the human genome, with a particular focus on teasing out the secrets of the so-called dark genomethe vast but largely unexplored territory of non-protein-coding DNA thought to hold the keys to treating common, complex disorders such as cancer and neurological disease.

There is an amazing team of engineers, scientists and clinicians at Duke coalescing around the challenges of studying and manipulating the human genome to treat disease, said Charlie Gersbach, a leader in the development of CRISPR-based technologies who will direct the CAGT. The mission of the CAGT is to build on their strengths, integrate their expertise, and support their entrepreneurial efforts to maximize impact of tackling grand challenges in genomics and epigenomics.

Launched with the support of the Pratt School of Engineering, the Trinity College of Arts and Sciences and the School of Medicine, the CAGT will focus its efforts on five key areas: genome structure and function, gene regulation, epigenomics of disease, genomics of drug response, and genome engineering technologies. Its faculty are already noted for pioneering advances in genomic tools and technologies, including next-generation DNA sequencing, CRISPR-based genome editing, high-throughput assays of genome structure and gene regulation, and single-cell genomics, which together provide a powerful toolkit for exploring these frontiersespecially the dark genome that makes up 98 percent of our shared blueprint.

In a clear sign of their potential, many of the Duke-born technologies developed for this work formed the basis of a Duke biotech startup called Element Genomics, which was acquired in 2018 by UCB, a global pharmaceuticals company with a focus on neurology and immunology.

Researchers know that the dark genome harbors the genetic regulators responsible for more than 90 percent of susceptibility to common diseases, including neurodegenerative, neuropsychiatric, autoimmune, and cardiovascular disorders, as well as predisposition to cancer. However, most studies of human gene function have completely ignored this gigantic piece of the puzzle, even though it is these mysterious genetic sites that represent a tremendous opportunity to attack diseases from a new direction. The lack of attention to the dark genome has historically been due to a lack of technologies for studying this biology.

With a number of world-leading biomedical engineers and scientists, Duke is emerging as a powerhouse in areas spanning gene therapy, stem cell and organoid technologies, microphysiological human tissue systems for drug testing, single-cell analysis tools, and innovative computational methods for analyzing large genomic datasets. A major goal of the center is to develop and integrate these otherwise disparate technologies and disease areas into a novel, concentrated approach to genomic discovery.

This center is unique in that its focused on decoding the dark genome, said Ravi Bellamkonda, Vinik Dean of the Pratt School of Engineering. This is crucial to understanding complex diseases like schizophrenia, cancer and Alzheimers, and Duke is one of the few places with the depth and breadth of expertise to believe such an outrageously ambitious project might just work.

Moreover, such technologies can be applied to any range of diseases or biological questions. Aside from big plans to tackle the dark genome, a few of the centers first projects aim to identify the gene regulatory networks that distinguish healthy versus diseased immune responses; understand how drugs used in oncology, inflammatory diseases, neurology and genetic diseases impact gene regulation in disease states; and define the function of genetic regulatory elements in different cells and environmental contexts. Identifying the right collaborations to answer the right questions at the right time will be an ongoing process within the CAGT and as part of the international genomics research community.

The CAGT is an important part of our strategic plan to unify and elevate recognition of Dukes tremendous expertise in cutting-edge genomic technologies, said Colin Duckett, vice dean for basic sciences in the Duke University School of Medicine. The opportunities created by building around existing successes of our faculty and integrating their efforts with complementary genomics activities through the new Duke Precision Genomics Collaboratory were clear.

The CAGT will leverage and integrate the range of unique expertise in genomic sciences here at Duke, added Svati Shah, associate dean of genomics in the School of Medicine and director of the Duke Precision Genomics Collaboratory. Its a really exciting endeavor, taking a deeper dive into better understanding the human genome and tackling how these poorly understood areas of the genome can help us better understand rare and common diseases.

The CAGT is directed by Gersbach, the Rooney Family Associate Professor of Biomedical Engineering and associate professor in surgery, who has focused on developing CRISPR technologies for both gene therapy and manipulating the web of biomolecules that determines which genes each cell activates and to what degree. This allows researchers to probe the roles of genes, but also provides methods for perturbing the dark genome in its natural context for the first time.

Additional center leadership includes a steering committee with expertise spanning CAGT focus areas:

Many of us have been doing really fun and interesting work that could have a major impact on human health with support from very different sources like the National Institutes of Health, industry and philanthropy, said Gersbach. Coordinating that collaboration through a focused center is a way to amplify and accelerate what grew organically across the Duke schools and departments and drive our efforts toward improving human health.

For more information, visit cagt.pratt.duke.edu.

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New Duke Center Takes Aim at the Dark Genome - Duke Today

To develop Ocugens orphan drug designated gene therapy candidate in its modifier gene therapy platform

Ocugen, Inc., a UK based clinical stage biopharmaceutical company focused on innovative therapies that address rare and underserved eye diseases, has entered into a strategic partnership with China based CanSino Biologics on Ocugens gene therapy pipeline product candidates for inherited retinal diseases, which are currently in development with Schepens Eye Research Institute of Massachusetts Eye and Ear, an affiliate of Harvard Medical School.

Under this strategic collaboration, CanSinoBIO will provide all CMC development and clinical supplies for the development of OCU400, Ocugens first gene therapy product candidate in its modifier gene therapy platform. CanSinoBIO maintains the option to support commercial manufacturing for Ocugen. The agreement also provides commercialization rights to CanSinoBIO in Greater China.

OCU400 has received two different orphan drug designations (ODD) from the U.S. FDA. The first, for the treatment ofNR3E3mutation-associated retinal degeneration and, most recently, for the treatment ofCEP290mutation-associated retinal disease.

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CanSino partners with Ocugen to work on gene therapy - BSA bureau

Astellas Pharma Inc. will invest $12.5 million in two innovation incubators operated by LabCentral, a U.S.-based lab facility for next-generation biotech start-ups. Astellas has previously committed more than $1 billion to drive innovation in Massachusetts to accelerate the discovery and development of potential breakthrough therapies in areas of significant unmet need.

"Astellas has a long-standing commitment to the Boston-area life sciences ecosystem, where world-class talent are dedicated to turning innovative science into value for patients," said Kenji Yasukawa, Ph.D., President and CEO, Astellas. "Our presence in the greater Boston area comprises over 200 professionals across several locations driving innovation in regenerative medicine, immuno-oncology, mitochondrial function, genetic regulation and beyond. Accelerating early-stage scientific innovation in areas such as cell and gene therapy is a strategic focus for Astellas, and is superbly aligned with the mission of LabCentral to serve as a launching-pad for cutting-edge biotech and life sciences start-ups."Astellas will invest $12.5 million to become the only pharma/biopharma company among five Founding Sponsors of a new incubator, which will feature a core lab space where companies can easily conductprocess development studies and a non-GMP pilot plant, being developed by LabCentral in Cambridge. The investment provides support to start-up companies and entrepreneurial founders seeking to create scientific innovation in areas of unmet need such as cell and gene therapy. The new incubator is expected to be operational in 2021.Astellas will also invest at least $450,000 over three years to become a Gold Sponsor of LabCentral's existing incubator located at 700 Main Street in Cambridge.By supporting these incubators, Astellas can select, support and access innovation from leading start-ups creating healthcare solutions in its areas of focus.Since 2010, Astellas has invested more than $800 million in, and committed nearly $500 million more to, Massachusetts-based innovation through the acquisitions of Ocata Therapeutics, Inc., Mitobridge, Inc. and Potenza Therapeutics, Inc., as well as the construction of a state-of-the-art headquarters for the Astellas Institute for Regenerative Medicine (AIRM) in Westborough, MA. The new facility, expected to open in 2020, will enable AIRM to accelerate research and development in the field of regenerative medicine and cell therapy.

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Astellas to Fund Boston-Area Cell and Gene Therapy Start-Up - Contract Pharma

SAN FRANCISCO--(BUSINESS WIRE)--Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases, today announced its planned presentations at the 24th International Annual Congress of the World Muscle Society in Copenhagen, Denmark, including new data from ASPIRO, the clinical trial evaluating AT132 in patients with X-linked Myotubular Myopathy. The oral presentation will be given by Dr. James J. Dowling, Hospital for Sick Children, Toronto, Canada, and will be held during the Clinical Trial Highlights 7 session of the conference on Saturday, October 5 beginning at 1:00pm Central European Summer Time (CEST)/7:00am Eastern Time (ET).

Dr. Dowling will present new efficacy and safety data from the ASPIRO Phase 1/2 dose escalation cohorts (10 treated patients and 2 controls). Efficacy analyses will include assessments of ventilator dependence and achievement of developmental motor milestones.

We are excited to share new efficacy and safety data from our ASPIRO study, stated Natalie Holles, President and Chief Operating Officer. The data from these dose escalation cohorts, along with results from our ongoing pivotal expansion cohort, will form the basis of the AT132 BLA submission planned in mid-2020, and MAA submission planned for the second half of 2020.

Audentes is planning several additional presentations during the conference, including a company-sponsored symposium that will provide an in-depth review of XLMTM and the ASPIRO study results. Following are details for each presentation:

Oral Presentation:

ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy FindingsSession: Clinical Trial Highlights 7Date and time: Saturday, October 5, 1:00-2:00pm Central European Summer Time (CEST)/7:00am-8:00am Eastern Time (ET)Abstract number: O.39

Poster Presentation:

INCEPTUS Pre-Phase 1, Prospective, Non-Interventional, Natural History Run-in Study to Evaluate Subjects Aged 4 Years and Younger with X-Linked Myotubular Myopathy (XLMTM)Session: Congenital myopathies: centronuclear and othersDate and time: October 2, 2019, 4:45pm - 6:45pm Central European Summer Time (CEST)/10:45am 12:45pm Eastern Time (ET)Abstract number: P.105

Audentes-Sponsored Symposium:

Altering the Treatment Paradigm: Gene Therapy for Neuromuscular DisordersDate and time: Friday, October 4, 1:15pm2:45pm Central European Summer Time (CEST)/7:15am 8:45am (ET)Location: Axelborg Hall

Agenda and Speakers:AAV Gene Therapy for Neuromuscular DisordersBenedikt Schoser, MD (Chair)Friedrich-Baur Institute, Ludwig-Maximilians University, Munich, Germany

Gene Therapy for XLMTM: The ASPIRO Study

The Potential of AAV for Neuromuscular DisordersEdward Conner, MDSenior Vice President and Chief Medical Officer, Audentes Therapeutics, San Francisco, CA, USA

Improvements in XLMTM Muscle Pathology and BiomarkersMichael W Lawlor, MD, PhDDepartment of Pathology and Laboratory Medicine and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI, USA

Achieving Ventilator Independence with AT132 in XLMTMRobert Graham, MDDivision of Critical Care Medicine, Boston Childrens Hospital, Boston, MA, USA

Attaining Motor Developmental Milestones in Children with XLMTMLaurent Servais, MD, PhDMuscular Dystrophy UK Oxford Neuromuscular Centre, Oxford, UK and Lige University, Lige, Belgium

About X-linked Myotubular Myopathy

X-linked Myotubular Myopathy (XLMTM) is a serious, life-threatening, rare neuromuscular disease that is characterized by extreme muscle weakness, respiratory failure, and early death. Mortality rates are estimated to be 50 percent in the first 18 months of life, and for those patients who survive past infancy, there is an estimated additional 25% mortality by the age of 10. XLMTM is caused by mutations in the MTM1 gene that lead to a lack or dysfunction of myotubularin, a protein that is needed for normal development, maturation, and function of skeletal muscle cells. The disease affects approximately 1 in 40,000 to 50,0000 newborn males.

XLMTM places a substantial burden of care on patients, families and the healthcare system, including high rates of healthcare utilization, hospitalization and surgical intervention. More than 80 percent of XLMTM patients require ventilator support, and the majority of patients require a gastrostomy tube for nutritional support. In most patients, normal developmental motor milestones are delayed or never achieved. Currently, only supportive treatment options, such as ventilator use or a feeding tube, are available.

About AT132 for the treatment of XLMTM

Audentes is developing AT132, an AAV8 vector containing a functional copy of the MTM1 gene, for the treatment of X-linked Myotubular Myopathy (XLMTM). AT132 may provide patients with significantly improved outcomes based on the ability of AAV8 to target skeletal muscle and increase myotubularin expression in targeted tissues following a single intravenous administration.

Audentes has reported promising safety, efficacy, and muscle biopsy data from ASPIRO, an ongoing, multicenter, ascending dose clinical study designed to evaluate the safety and efficacy of AT132. The preclinical development of AT132 was conducted in collaboration with Genethon (www.genethon.fr).

AT132 has been granted Regenerative Medicine and Advanced Therapy (RMAT), Rare Pediatric Disease, Fast Track, and Orphan Drug designations by the U.S. Food and Drug Administration (FDA), and Priority Medicines (PRIME) and Orphan Drug designations by the European Medicines Agency (EMA).

About Audentes Therapeutics, Inc.

Audentes Therapeutics (Nasdaq: BOLD) is a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases. We are leveraging our AAV gene therapy technology platform and proprietary manufacturing expertise to develop programs across three modalities: gene replacement, vectorized exon skipping, and vectorized RNA knockdown. Our product candidates are showing promising therapeutic profiles in clinical and preclinical studies across a range of neuromuscular diseases. Audentes is a focused, experienced and passionate team driven by the goal of improving the lives of patients.

For more information regarding Audentes, please visit http://www.audentestx.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, the timing and nature of the ASPIRO pivotal expansion and the ASPIRO clinical data results and the timing and nature of regulatory filings for AT132. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although the company believes that the expectations reflected in such forward-looking statements are reasonable, the company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company's ability to advance its product candidates and obtain regulatory approval of and ultimately commercialize its product candidates, the timing and results of preclinical and clinical trials, the company's ability to fund development activities and achieve development goals, the company's ability to protect intellectual property and other risks and uncertainties described under the heading "Risk Factors" in documents the company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

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Audentes Announces Upcoming Presentations at the 24th International Annual Congress of the World Muscle Society, Including New Data From ASPIRO, the...

A study has revealed that using DNA-like molecules to repair gene mutations in models could act as a successful therapy for patients.

Researchers have used a promising new therapeutic to treat Duchenne muscular dystrophy (DMD) in human muscle cells and mice models. According to the researchers, their treatment could aid as many as 47 percent of patients with the condition.

The study, conducted by the University of Alberta, US, investigated whether using a group of DNA-like molecules would result in regrowth of a protein called dystrophin, which supports muscle strength. Patients with DMD often severely lack this protein.

In muscle, if there is no dystrophin there is no support of muscle membrane and the muscle cells will become easily damaged or destroyed, said Toshifumi Yokota, a professor of medical genetics at the university. Our DNA-like molecules restore the production of dystrophin so it can support the muscle cell membrane.

The team used a mix of DNA-like molecules, known as antisense oligonucleotides, to act in the manner of a stitch to repair a specific gene mutation in patients.

Our treatment produces a shorter dystrophin protein than the drug being used now. This shorter protein is associated with extremely mild symptoms in some of the muscular dystrophy patients. Some have almost no symptoms at all, explained Yokota.

The researchers are now working to reduce the number of DNA-like molecules in the mixture to reduce both cost and regulatory hurdles moving forward. They hope to progress the work to a clinical trial in the future.

The results were published in Molecular Therapy.

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Researchers find DNA therapy could treat patients with DMD - Drug Target Review