Archive for the ‘Gene Therapy Research’ Category

The worldwide for Gene Therapy marketis expected to grow at a CAGR of roughly +33% over the next five years.

Gene therapy is a test treatment that includes bringing genetic material into an individuals phones to battle or counteract ailment. Specialists are reading gene therapy for various maladies, for example, extreme joined immuno-insufficiencies, hemophilia, Parkinsons infection, disease and even HIV, through various methodologies. A gene can be conveyed to a cell utilizing a bearer known as a vector. The most widely recognized kinds of vectors utilized in gene therapy are infections. The infections utilized in gene therapy are modified to make them safe, albeit a few dangers still exist with gene therapy. The innovation is still in its infancy

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Scope of the Report:

The Gene Therapy market is currently in a phase of transition as mobile operators seek to address increasing mobile traffic demands amidst economic uncertainties. This paradigm shift is bringing new challenges and opportunities to infrastructure vendors.

The key players covered in this study:

Market segment by Type, the product can be split into

Market segment by Application, split into

Different global regions such as North America, Latin America, Europe, Asia-Pacific, Africa, and India have been examined to get a better understanding of the competitive landscape.

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Key points of Gene Therapy Market Report

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Hormonal therapy industry market is considered to be a very small market because of the unlimited scopes as well as Limited market size which is only focused to was helping with being a substitute of the biotechnology and Clinical Research

Global Prescription Digital Therapeutics (DTx) Market 2020 research report presents analysis of market size, share, and growth, trends, cost structure, statistical and comprehensive data of the global market. The Market report offers noteworthy data regarding industrys growth parameters, the current

Global Pet Cancer Therapeutics Market Size, Status and Forecast 2020-2027 The Global Pet Cancer Therapeutics Market report mainly elaborates market size, share, trends, and growth analysis on the basis of different parameters. The Global Pet Cancer Therapeutics Industry analysis is

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Gene Therapy Market Aims to Expand at Double-Digit Growth Rate|Novartis AG, Gilead Sciences Inc., UniQure NV, Spark Therapeutics LLC, Bluebird Bio,...

ReportsnReports recently added a detailed overview and industry professional survey report on the global Stem Cell and Regenerative Therapy Market. In this report, titled Stem Cell and Regenerative Therapy Market Size, Share and Industry Analysis by Technologies, By Product, By Application, By Distribution Channel, and Regional Forecast 2019-2026.

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The scope of the report encompasses the major types of Stem Cell and Regenerative Therapy Market that have been used, as well as the major applications being developed by industry, academic researchers and their commercialization offices, and government agencies. It analyzes the current market status, examines future market drivers, and presents forecasts of growth over the next five years. Technology developments, including the latest trends, are discussed. Other influential factors such as screening strategies for pharmaceuticals have also been included.

The global Stem Cell and Regenerative Therapy Market is comprehensively profiled in the report, including a detailed study of the markets key drivers and restraints, major market players, and leading segments.

Report Scope:

The scope of this report is broad and covers various type of product available in the stem cell and regenerative medicines market and potential application sectors across various industries. The current report offers a detailed analysis of the stem cell and regenerative medicines market.

The report highlights the current and future market potential of stem cell and regenerative medicines and provides a detailed analysis of the competitive environment, recent development, merger and acquisition, drivers, restraints, and technology background in the market. The report also covers market projections through 2024.

The report details market shares of stem cell and regenerative medicines based on products, application, and geography. Based on product the market is segmented into therapeutic products, cell banking, tools and reagents. The therapeutics products segments include cell therapy, tissue engineering and gene therapy. By application, the market is segmented into oncology, cardiovascular disorders, dermatology, orthopedic applications, central nervous system disorders, diabetes, others

The market is segmented by geography into the following regions: North America, Europe, Asia-Pacific, South America, and the Middle East and Africa. The report presents detailed analyses of major countries such as the U.S., Canada, Mexico, Germany, the U.K. France, Japan, China and India. For market estimates, data is provided for 2018 as the base year, with forecasts for 2019 through 2024. Estimated values are based on product manufacturers total revenues. Projected and forecasted revenue values are in constant U.S. dollars, unadjusted for inflation.

Report Includes:

28 data tables An overview of global markets for stem cell and regenerative medicines Analyses of global market trends, with data from 2018, estimates for 2019, and projections of compound annual growth rates (CAGRs) through 2024 Details of historic background and description of embryonic and adult stem cells Information on stem cell banking and stem cell research A look at the growing research & development activities in regenerative medicine Coverage of ethical issues in stem cell research & regulatory constraints on biopharmaceuticals Comprehensive company profiles of key players in the market, including Aldagen Inc., Caladrius Biosciences Inc., Daiichi Sankyo Co. Ltd., Gamida Cell Ltd. and Novartis AG

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Summary:

The global market for stem cell and regenerative medicines was valued at REDACTED billion in 2018. The market is expected to grow at a compound annual growth rate (CAGR) of REDACTED to reach approximately REDACTED billion by 2024. Growth of the global market is attributed to the factors such as growingprevalence of cancer, technological advancement in product, growing adoption of novel therapeuticssuch as cell therapy, gene therapy in treatment of chronic diseases and increasing investment fromprivate players in cell-based therapies.

In the global market, North America held the highest market share in 2018. The Asia-Pacific region is anticipated to grow at the highest CAGR during the forecast period. The growing government funding for regenerative medicines in research institutes along with the growing number of clinical trials based on cell-based therapy and investment in R&D activities is expected to supplement the growth of the stem cell and regenerative market in Asia-Pacific region during the forecast period.

Reasons for Doing This Study

Global stem cell and regenerative medicines market comprises of various products for novel therapeutics that are adopted across various applications. New advancement and product launches have influenced the stem cell and regenerative medicines market and it is expected to grow in the near future. The biopharmaceutical companies are investing significantly in cell-based therapeutics. The government organizations are funding research and development activities related to stem cell research. These factors are impacting the stem cell and regenerative medicines market positively and augmenting the demand of stem cell and regenerative therapy among different application segments. The market is impacted through adoption of stem cell therapy. The key players in the market are investing in development of innovative products. The stem cell therapy market is likely to grow during the forecast period owing to growing investment from private companies, increasing in regulatory approval of stem cell-based therapeutics for treatment of chronic diseases and growth in commercial applications of regenerative medicine.

Products based on stem cells do not yet form an established market, but unlike some other potential applications of bioscience, stem cell technology has already produced many significant products in important therapeutic areas. The potential scope of the stem cell market is now becoming clear, and it is appropriate to review the technology, see its current practical applications, evaluate the participating companies and look to its future.

The report provides the reader with a background on stem cell and regenerative therapy, analyzes the current factors influencing the market, provides decision-makers the tools that inform decisions about expansion and penetration in this market.

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The latest Stem Cell and Regenerative Therapy Market report provides readers with a deeper understanding of potential target consumers to create a lucrative marketing strategy for the 2019-2026 forecast period. For entrepreneurs seeking information about potential customers, it will be particularly helpful. Selective statements provided by leading vendors would allow entrepreneurs to gain a deeper understanding of the local market and prospective customers.

Table of Contents:

Chapter 1 Introduction

Study Background

Study Goals and Objectives

Reasons for Doing This Study

Scope of Report

Methodology and Information Sources

Geographic Breakdown

Market Breakdown

Analysts Credentials

.Continued

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Stem Cell and Regenerative Therapy Market Covid-19 Impact Analysis, Size, Share & Trends Analysis Report by Component, By Enterprise Size, By End...

By 2027, the global gene therapy market is estimated to reach a staggering value of $6.6B. With the number of successfully approved gene therapies increasing, the sector has moved from hype to hope. At the heart of gene therapy lie viral vectors, which are used to transport a gene into a target cell. Here we explore the current bottleneck in viral vector production, why viral vectors still outshine non-viral vector solutions, and what we can expect in the future.

The gene therapy field is gaining momentum. Investments are pouring in. The FDA is estimating that by 2025 it will approve between 10 and 20 cell and gene therapy products every year. This shows that a treatment, which started out as a hype, is now a real hope, says Ratish Krishnan, Associate Director of Cell and Gene Therapy Bioprocessing at MilliporeSigma*.

Today, a number of treatments have been approved, such as Spark Therapeutics LUXTURNA, the first FDA approved in vivo gene therapy, or Novartis Zolgensma, which gained US approval in 2019 and European approval in March 2020.

One of the key ingredients of gene therapies is the viral vector, which is used to transfer a gene of interest into a target cell. The most commonly used vector is that of the adeno-associated virus (AAV). But the manufacturing of viral vectors and scaling up their production remain difficult.

In upstream development, one challenge is the way viral vectors are produced. In a process called transient transfection, plasmids carrying the DNA of interest are introduced into host cells that will produce the viral vectors.

But host cells are commonly grown in adherent cell cultures, which are usually harder to reproduce at a large scale. So to scale-up and achieve high titers of virus particles, researchers are working on growing cells in suspension using large bioreactors instead.

We are facing several challenges at the moment and that is what keeps us on our toes, Krishnan adds. In upstream development, there is a desire to move towards suspension. Most processes use transient transfection methods using plasmids and the transfection step at a production scale of 500L or 2000L is extremely challenging.

In downstream development, researchers are studying the purity of capsids. The capsid is the protective shell of the virus enclosing the gene of interest. Related to this is an important discussion on the purity of viral vectors.

When you produce vectors, you will generally have a population of empty capsids, which is the viral AAV assembly without the genetic material inside, or partially-full capsids with only part of the DNA inside, Krishnan explains. We have to better understand the role of empty capsids inside the body. Are they needed as immune decoys or are they strictly considered impurities? In theory, you only need the ones with all the DNA inside, the full capsids.

But researchers have yet to discover the correct percentage of full capsids in a drug substance or product, Krishnan says. We dont have the answer yet. Typically, the strategy leans towards the enrichment of a percentage of full capsids as high as possible, while taking into account the data from clinical trials.

Questions about purity are difficult to answer because there is little or no regulatory guidance. For instance, compared to monoclonal antibodies (mAbs) for which the regulatory environment is well understood, the regulatory landscape for gene therapies remains largely unclear.

Through decades of research, mAb production also works through well-established and standardized platforms, whereas viral vectors are still in their infancy and cannot be produced using platform technologies yet although much is being done in this field and they are catching up fast.

Because of their longstanding history, we already have a lot of knowledge and research about mAbs, Krishnan explains. Lets say I want to start a biotech company or a CDMO that develops mAbs. I can rely on existing templates and get up and running much quicker than if I were developing viral vectors.

AAVs come in many different serotypes distinguishable strains which impact platform development. The scale-up is also challenging since the indications can be strikingly contrasting. For example, ocular indications need smaller viral drug substances compared to a muscular indication.

Manufacturing mAbs is better understood than manufacturing viral vectors. Viruses are a whole lot bigger and can be more complex than antibodies. While antibodies usually have a size around 10 nm, AAVs measure around 20nm and Lentiviruses around 100nm in size. Not only are antibodies produced at a larger scale than viral vectors, but their scalability is also more predictable due to the established platform technologies.

Despite these challenges, the increase in popularity, investments flooding in, and the promise of essential cures have led to a bottleneck in viral vector production for gene therapies. But many companies working in the gene therapy field are small or emerging biotechs that do not have the necessary resources and expertise in-house to tackle the challenges of viral vector production.

Lacking the facilities to do it themselves, small and emerging biotechs therefore turn to experienced contract development and manufacturing organizations (CDMOs), such as Merck BioReliance, to produce their gene therapies.

There are only a handful of CDMOs that have the capability and expertise to take on the complexities of gene therapy projects, Krishnan says. But there is currently a bottleneck in manufacturing slots. Manufacturing facilities can only work 24 hours a day, and if you are a small company and a CDMO has other clients waiting in line before you to have their therapies manufactured, you have no other option but to wait.

The problem with waiting, of course, is that the biotech runs the risk of falling behind its competition. Krishnan emphasizes, time is of the essence in gene therapy. There is no silver medal for developing a therapy for the same indication.

The key, says Krishnan, lies in much planning and close engagement with the CDMO partner. Biotechs should do an analysis of what they can perform in-house versus what they have to outsource early on. Engaging a CDMO is the route you want to take.

With decades of experience, Merck can support its biotech sponsors all the way to the clinic. We are big on the concept of integrated solutions, Krishnan explains. From clone to clinic to commercialization, Merck has the expertise, knowledge network, product, and services to help guide any customer to the finish line. We have the CDMO expertise with BioReliance, with testing services, gene therapy expertise, and regulatory support.

To circumvent the manufacturing bottleneck for viral vectors, some biotechs are looking at non-viral vector solutions for gene therapies. While traditional gene therapies use a viral vector, like AAV, to transfer a gene of interest into the patient, non-viral gene therapy deploys an alternative delivery system for the gene of interest.

Examples for non-viral delivery systems include physical force to deliver the gene through the cell membrane; injecting the gene with a needle into the target region; electroporation, which uses an electric current to produce pores in the cell membrane through which the gene can be inserted into the cell; and chemical vectors, such as lipid-, polymer-, or peptide-based particles.

Nevertheless, viral vectors, such as the AAV, remain the preferred path for most companies. The efficiency of delivery for non-viral vectors remains questionable. This means that there might be reactions in the immune system that get triggered, eliciting a dangerous, adverse response. AAVs, on the other hand, are well-engineered and safe, despite being novel.

Viral vectors have recently demonstrated success, Krishnan adds. Scientists are making advances in the non-viral area of gene therapy but they also come with a unique set of challenges. Questions, such as how do they interact with serum components in the body, how do they involve the immune system before reaching the target tissue, how do they interact with the surfaces of cells, remain.

Despite unaddressed challenges, gene therapy has definitely shifted from being a hope to carrying an expectation. This is reflected in the number of investments pouring into the sector.

Big pharma and biotech companies are heavily investing their resources into gene therapy, Krishnan says. Typically, companies have vaccines or mAbs in their portfolio. Now, gene therapy is becoming a major modality of interest as well.

While we are still far away from reaching the smooth manufacturing processes we have in place for antibodies, many companies are also looking into platform approaches for gene therapy. We would take a quantum leap if we developed a platform approach for upstream and downstream processes. That would significantly reduce the time to the clinic. Platform approaches are definitely being explored, Krishnan adds.

Vendors like Merck are playing a big role in developing fit-for-purpose products for gene therapies, Krishnan says. At the R&D level, researchers are also working on advances in capsid engineering. We already have synthetic capsids, and there are other tremendous advancements in capsid engineering, design, and purity, which are going to continue to evolve.

But, as Krishnan puts it, We are running a marathon at sprint speed. The journey is exciting and challenging, identical to the ride on a rollercoaster, but we are barely even at the tip of the iceberg. There are patients waiting for life-saving treatment, so gene therapies will definitely continue to be in the limelight, and for good reason.

Are you fighting to solve the bottleneck in viral vector production? Get in touch with the expert team at Merck and view their webinar on this topic!

*The life science business of Merck operates as MilliporeSigma in the U.S. and Canada.

Images via Shutterstock.com and Elena Resko

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The Roller Coaster of Gene Therapy... - Labiotech.eu

Genespire and SR-Tiget announce strategic alliance for the development of transformative gene therapies for genetic diseases and disclose collaboration focus

Pre-clinical data from SR-Tiget, included in the alliance with Genespire, to be presented at ASGCT 23rd Annual Meeting

Italy, Milan, 13 May 2020: The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), one of the worlds leading gene therapy research institutes jointly managed by Fondazione Telethon and Ospedale San Raffaele and Genespire, a gene therapy company developing transformative therapies for genetic diseases, and spin-out of SR-Tiget, announced today their alliance on the research and development of candidate therapeutic products for people affected by primary immunodeficiencies and metabolic diseases based on novel gene editing and lentiviral vector technologies developed by SR-Tiget.

Genespire was co-founded in March 2020 by SR-Tiget director and gene therapy pioneer Prof. Luigi Naldini and Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. Genespire recently raised 16 million in a Series A financing round from Sofinnova Partners.

Under the terms of the alliance, Genespire and SR-Tiget will study and further develop novel gene therapies, which have the unique potential to address severe unmet medical need and exploit gene editing and lentiviral vector technologies developed by SR-Tiget.

Genespire was granted an exclusive global license for the research, development and commercialization of gene therapies for metabolic diseases based on SR-Tigets alloantigen free, microRNA-regulated lentiviral vectors, which allow for stable liver gene therapy even for diseases with early onset, requiring administration at a young age.

Genespire was also granted exclusive licenses and options to the results of a joint research and development program with SR-Tiget in the T-cell and Hematopoietic Stem Cells field to address genetic diseases, in particular primary immunodeficiencies, exploiting the ex vivo gene editing technology. SR-Tiget and Genespire will first collaborate to bring an ex-vivo autologous edited T-cell gene therapy for X-linked Hyper IgM syndrome (HIGM1) to the clinic, which becomes Genespires lead candidate product. HIGM1 is caused by inherited mutations of the CD40 ligand gene (CD40L), resulting in impaired antibody response and innate immunity, meaning that people find it difficult to fight off infections and eventually succumb to them. The treatment objective is to correct the defective gene through targeted editing of the endogenous locus, thereby maintaining physiological regulation of the CD40L gene, with the aim of improving the immune response of the patients.

Preclinical results of SR-Tiget on HIGM1 will be disclosed in an oral presentation at the American Society for Cell and Gene Therapy (ASGCT) 23rd Annual Meeting, taking place virtually from 12-15 May 2020 by SR-Tiget (details of Presentation 1 below). The presentation will outline the technology and its preclinical validation in the disease model and patient derived cells and discuss the potential of the gene edited T-cell treatment approach for patients with Hyper IgM.

Dr. Alessio Cantore will also present novel data related to the potential of the lentiviral vector platform for liver gene therapy in an oral presentation at ASGCT (details of Presentation 2 below). The presentation will focus on investigating the stability of lentiviral vector genetically modified liver cells following post-natal liver growth in mice, in view of its potential application to pediatric patients.

Luigi Naldini, Director of SR-Tiget and scientific co-founder of Genespire said: We are excited to have secured a path for bringing forward some of the gene therapy work pioneered at SR-Tiget to eventually help individuals affected by severe metabolic and immunodeficiency disorders. SR-Tigets alliance with Genespire will provide the means to progress effectively to clinical trials, with a strong view to develop efficacious and safe medicines ready for market access.

Julia Berretta, Chief Executive Officer of Genespire commented: SR-Tiget brings outstanding expertise and significant experience in developing gene therapies from bench to bedside. We believe that our strong partnership with SR-Tiget, led by internationally recognized experts Prof. Luigi Naldini and Dr. Alessio Cantore will be fundamental for Genespire to carry out its goal of translating pioneering science into transformative therapeutic solutionsfor patients.

-ENDS-

Oral presentation 1 details: Title: Modeling, Optimization and Comparative Efficacy of HSC- and T-cell Based Editing Strategies for Treating Hyper IgM Syndrome Authors: Valentina Vavassori, Elisabetta Mercuri, Genni Marcovecchio, Maria Carmina Castiello, Giulia Schiroli , Luisa Albano, Elena Fontana, Andrea Annoni, Valentina Capo, Carrie Margulies, Frank Buquicchio, Joseph Kovacs, Eugenio Scanziani, Cecilia Cotta-Ramusino, Anna Villa, Luigi Naldini, Pietro Genovese Date and time: May 14th 2020, 3:45 PM EDT Session: 354 Gene Therapies for Hemophilia and Immune Disorders Abstract #937 Oral Presentation 2 Details Title: Investigating the stability of lentiviral vector targeted liver cells during post-natal growth for in vivo gene therapy applications Authors: Michela Milani, Francesco Starinieri, Cesare Canepari, Tongyao Liu, Federica Moalli, Gioia Ambrosi, Tiziana Plati, Mauro Biffi, Cesare Covino, Timothy Nichols, Matteo Iannacone, Robert Peters, Luigi Naldini, Alessio Cantore Date and time: May 14th 2020, 4:15 pm EDT Session: 350 RNA Virus Vectors Abstract #911

Notes to Editors

About Hyper IgM Syndrome (HIGM)

Hyper IgM is a Primary Immune Deficiency affecting 1:250,000-500,000 patients. The disease is linked to mutations in the CD40L gene, which is expressed in activated CD4 T cells, and results in impaired antibody production and innate immunity. The current standard of care is constituted by continuous Ig replacement, and antibiotic-antifungal prophylaxis, but the disease is still linked to high morbidity and reduced life expectancy. Allogeneic hematopoietic stem cell transplant (HSCT) is potentially curative, but is limited by matched donor availability and is associated with high risk of graft versus host disease, infections and death. Thus, improved therapeutic alternatives are strongly needed.

About Genespire

Genespire is a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini and Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. It is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute and is backed by Sofinnova Partners. http://www.genespire.com

About SR-Tiget

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute has given a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

About Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the bestscientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit:www.telethon.it

About Ospedale San Raffaele

Ospedale San Raffaele (OSR) is a clinical-research-university hospital established in 1971 to provide international-level specialised care for the most complex and difficult health conditions. OSR is part ofGruppo San Donato, the leading hospital group in Italy. The hospital is a multi-specialty center with over 60 clinical specialties; it is accredited by the Italian National Health System to provide care to both public and private, national and international patients. Research at OSR focuses on integrating basic, translational and clinical activities to provide the most advanced care to our patients. The institute is recognized as a global authority in molecular medicine and gene therapy, and is at the forefront of research in many other fields. Ospedale San Raffaele is a first-class institute which treats many diseases and stands out for the deep interaction between clinical and scientific area. This makes the transfer of scientific results from the laboratories to the patients bed easier. Its mission is to improve knowledge of diseases, identify new therapies and encourage young scientists and doctor to grow professionally. For more information, please visit:www.hsr.it

Enquiries: Genespire Julia Berretta, CEOTel: +39 02 83991300info@genespire.com Consilium Strategic Communications Amber Fennell / Matthew Neal Tel: +44 (0) 20 3709 5700genespire@consilium-comms.com

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Genespire and SR-Tiget announce strategic alliance for the development of transformative gene therapies for genetic diseases and disclose...

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Cutting-edge research on rewriting the genes responsible for Tay-Sachs disease, sickle-cell anemia, and other disorders will be presented at this weeks online annual meeting of the American Society for Gene and Cell Therapy. Originally planned as a Boston gathering, the scientific groups get-together became a virtual one because of the Covid-19 pandemic.

From Tuesday through Friday, academic researchers will be presenting their latest data online, along with updates from firms like Bluebird Bio (ticker: BLUE), Voyager Therapeutics (VYGR), Fate Therapeutics (FATE), Beam Therapeutics (BEAM), Axovant Gene Therapies (AXGT), and many others. Patients and their families have found their way to clinical trials through the societys website.

Bluebird plans presentations on its cell therapy against the blood cancer known as multiple myeloma. Using a technology known as CAR-T, the company creates supercharged versions of a patients immune cells that have halted disease progression in some of the 18 patients enrolled in a continuing Phase 1 trial.

Featured on Friday will be reports on the first babies treated with gene therapy for the debilitating neurodegenerative disorder Tay-Sachs. The treatment is being developed by the London-based Axovant under license from the University of Massachusetts Medical School.

Voyager will discuss its preclinical mouse studies on treating neurological disorders like amyotrophic lateral sclerosis and Huntingtons disease by using techniques that block the rogue signals generated by defective genes.

Fate Therapuetics is scheduled to show a new off-the-shelf CAR-T technology that it hopes will allow the immune system to target a broad range of solid tumors as well as multiple myeloma. The approach is licensed from Harvard Universitys Dana-Farber Cancer Institute.

Beam, meanwhile, will detail success it has shown in preclinical editing of the genetic defect that causes sickle-cell anemia. The company is developing a sharper-edged way of rewriting faulty genes than the widely used Crispr technology that Beam licensed from researchers at the Broad institute of Harvard and MIT. Beam founder and Crispr pioneer Feng Zhang will give a featured lecture as part of the online meeting on Thursday.

Write to Bill Alpert at william.alpert@barrons.com

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Gene-Therapy Treatments for Tay-Sachs, Sickle Cell to Be Featured in Online Gathering - Barron's

Researchers at Washington University School of Medicine in St. Louis have demonstrated a new gene therapy that builds strength and muscle mass quickly while preventing obesity even when the recipient is eating a high-fat diet. This isnt some crazy new bodybuilding tool, though, but rather something that could be used as a physical therapy aid for people with muscle-weakening arthritis or those suffering from osteoarthritis-related joint pain.

We know that obesity and joint injury are the primary risk factors for osteoarthritis, Farshid Guilak, professor of orthopedic surgery and director of research at Shriners Hospitals for Children in St. Louis, told Digital Trends. However, in cases of severe obesity or muscle loss, it is extremely difficult if not impossible to lose weight or improve muscle strength through normal exercise and diet. The goal of this study was to show the importance of muscle strength in overriding many of the harmful effects of obesity on the joint.

So far, the gene therapy approach has only been demonstrated in mice. The researchers delivered the gene for a molecule called follistatin to the muscle of eight-week-old mice, via injection. The protein blocks myostatin, a molecule normally responsible for stopping muscle growth. The mice in the experiment gained around twice their normal muscle mass and were able to completely burn off all the extra energy from an unhealthy high-fat diet. This prevented almost all the metabolic complications of obesity, such as systemic inflammation and high blood sugar, while also reducing arthritis and pain significantly.

The researchers worried that the gene therapy could potentially have a negative effect on the mices hearts. However, these fears proved unfounded and heart function in the mice actually improved, along with overall cardiovascular health.

These first studies in mice have shown that the procedure has excellent efficacy and safety, Guilak said. The next steps will be to do longer-term studies in mice and possibly larger animals to ensure safety of this procedure.

Guilak said that, should these steps prove successful, the researchers would consider testing the approach in humans with the initial trials in those with severe, possibly life-threatening diseases of the muscles, such as muscular dystrophy.

A paper describing the work was recently published in the journal Science Advances.

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New gene therapy cuts fat and builds muscle with ease. But theres a catch - Digital Trends

PALO ALTO, Calif.andDURHAM Flush with cash, Kriya Therapeutics has big plans.

The biotech startup, with headquarters in Durham and Palo Alto, California, has secured $80.5 million in Series A financing to fund the development of its gene therapies for highly serious diseases.

Among them: type 1 and type 2 diabetes, severe obesity and other indications affecting millions of patients.

Series A investors include QVT, Dexcel Pharma, Foresite Capital, Bluebird Ventures (associated with Sutter Hill Ventures), Narya Capital, Amplo,Paul Manning, andAsia Alpha. This Series A round follows an initial seed financing completed by the company in the fourth quarter of 2019 led by Transhuman Capital, who also participated in the Series A round.

Kriya said financing proceeds would go towards supporting the development of the companys pipeline, internal discovery engine, and proprietary GMP manufacturing infrastructure.

There have been numerous successful gene therapies focused on rare monogenic diseases in recent years, said Shankar Ramaswamy, M.D., Co-Founder, Chairman, and CEO of Kriya Therapeutics, in a statement.

We see tremendous potential to expand the field and apply gene therapy to highly prevalent serious diseases. We are focused on designing gene therapies using algorithmic tools, scalable infrastructure, and proprietary technology to optimize the efficacy and durability of our treatments. We look forward to accelerating the development of our pipeline, platform technologies, and internal GMP manufacturing capability with the funds raised in this Series A financing.

Founded in 2019, the companys team includesformer senior leadership from Spark Therapeutics, AveXis, Sangamo Therapeutics, and other gene therapy companies.

Kriyas initial pipeline includes:

Kriya is building a leading team and cutting-edge infrastructure to engineer best-in-class gene therapies for severe chronic conditions and accelerate their advancement into human clinical trials, saidRoger Jeffs, Ph.D., Co-Founder and Vice Chairman of Kriya, in a statement.

The company is committed to incorporating the latest advancements in the field into the design and development of its therapeutic constructs. Through its R&D laboratory capabilities in the Bay Area and in-house process development and manufacturing infrastructure inResearch Triangle Park, I believe that Kriya will be uniquely positioned to become a leader in the gene therapy field.

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Durham's Kriya Therapuetics lands $80M to advance gene therapies for diabetes, severe obesity - WRAL Tech Wire

]]> The company signed gene therapy deals with Novartis and Sarepta Therapeutics. Credit: Gerd Altmann from Pixabay.

Biotechnology company Dyno Therapeutics has launched from stealth mode with focus on using artificial intelligence (AI) technology to develop adeno-Associated Virus (AAV) vectors.

The company signed gene therapy deals with Novartis and Sarepta Therapeutics.

Dyno Therapeutics and Novartis will create improved AAV vectors for research, development and commercialisation of gene therapies across ocular diseases.

The partnership will leverage Dynos CapsidMap AI platform in combination with Novartis gene therapy development and global commercialisation expertise.

Dyno will use AI technology and its suite of machine learning and experimental tools to design and identify AAV capsids with improved functional properties for gene therapy.

Later, Novartis will carry out preclinical, clinical and commercialisation activities for the gene therapy candidates developed using the AAV capsids.

Dyno will gain upfront consideration, research funding, licence fees, along with potential clinical, regulatory and sales milestone payments.

Dyno Therapeutics CEO and cofounder Eric Kelsic said: With their extensive ophthalmologic expertise, Novartis is an ideal partner to leverage Dynos platform to design AI-powered vectors to expand the impact of gene therapies for ocular diseases.

This collaboration is a major step forward in our plan to realise the potential of Dynos CapsidMap platform for gene therapies to improve patient health.

Meanwhile, the company will work with Sarepta Therapeutics to use its CapsidMap platform to develop next-generation AAV vectors for muscle diseases.

Under the deal, Dyno will design and discover AAV capsids for gene therapy while Sarepta will conduct preclinical, clinical and commercialisation for product candidates resulting from the alliance.

Dyno could get more than $40m in upfront, option and licence payments during the research phase. Also, if Sarepta develops and commercialises product candidates for various muscle diseases, Dyno will receive milestone payments.

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Dyno Therapeutics launches with deals from Novartis and Sarepta - Pharmaceutical Technology

Repeated setbacks aside, little Capricor has suggested it has generated some long-term data to support its pursuit to garner approval for its stem cell therapy for Duchenne muscular dystrophy, although some of the data appeared to underwhelmed investors.

The data from the small, placebo-controlled mid-stage study, HOPE-2, tracked the effects of the companys stem cell therapy CAP-1002, which is designed to temper the inflammation associated with DMD, in 8 boys and young men who are in advanced stages of DMD. The remaining 12 enrolled patients received the placebo.

The main goal of the study was a measure that evaluates shoulder, arm and hand strength in patients who are generally non-ambulant (performance of the upper limb (PUL) 2.0), as suggested by the FDA, Capricor said. It is one of several ways Capricor quantified skeletal muscle improvement in the trial.

The intravenous infusion of CAP-1002, given every 3 months, induced a statistically meaningful improvement of 2.4 points (p=0.05) versus the placebo group, in which patient declines were consistent with natural history data. However, on another measure of upper limb function, the trend was in favor of the Capricor drug, but did not hit statistical significance.

The companys shares $CAPR were down nearly 13% to $6.89 in morning trading.

Click on the image to see the full-sized version

Meanwhile, there were also some encouraging data on cardiac function the genetic condition is characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles.

As reflected above, CAP-1002 elicited an improvement across different measures of cardiac function, although the effect was not always statistically significant. In particular, the drug also caused a reduction in the levels of the biomarker CK-MB, an enzyme that is only released when there is cardiac muscle cell damage.

Armed with these data and an RMAT and orphan drug designation from the FDA, Capricor is now hoping to eke out a plan with the FDA for marketing approval.

LA-based Capricor initially set out to test the potential of technology that Eduardo Marbn, CEO Linda Marbns husband, developed at Johns Hopkins. But repeated setbacks clobbered the company, which in 2014 traded north of $14 a share. In 2017, J&J walked away from a collaboration on a stem cell therapy for damaged hearts after it flopped in the clinic.

In late 2018, the company voluntarily halted a DMD clinical trial, following a severe allergic reaction that occurred during infusion. In February 2019, the company said it is exploring strategic alternatives for one or more of its products and cutting 21 jobs to keep financially afloat, but had resumed dosing in its DMD trial.

The first batch of positive data on CAP-1002, which consists of progenitor cells derived from donor hearts and is designed to exude exosomes that initiate muscle repair by suppressing inflammation and driving immunomodulation, came last July when the company announced the drug had generated a positive effect at the interim analysis juncture of HOPE-2. Capricor is now working on to flexing its therapeutic muscle with CAP-1002 to fight the Covid-19 pandemic.

DMD is a rare muscle-wasting disease caused by the absence of dystrophin, a protein that helps keep muscle cells intact. It disproportionately affects boys and affects roughly 6,000 in the United States.

Patients are essentially treated with steroids. Sarepta Therapeutics now has two exon-skipping drugs designed to treat certain subsets of the disease, although the magnitude of their effect is controversial given that approvals were not based on placebo-controlled data. Meanwhile, Sarepta and others are also pursuing one-time cures in the form of gene therapies to replace the missing dystrophin gene in patients.

Social: Linda Marbn, Capricor CEO (Twitter)

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One year on, Capricor's stem cell therapy appears to help DMD patients in small study, but investors balk at the data - Endpoints News

Global Gene Therapy for Age-related Macular Degeneration Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level.

Gene Therapy for Age-related Macular Degeneration Market Size Covers Global Industry Analysis, Size, Share, CAGR, Trends, Forecast And Business Opportunity.

>>Need a PDF of the global market report? Visit: https://industrystatsreport.com/Request/Sample?ResearchPostId=11998&RequestType=Sample

Latest research report on Gene Therapy for Age-related Macular Degeneration Market delivers a comprehensive study on current market trends. The outcome also includes revenue forecasts, statistics, market valuations which illustrates its growth trends and competitive landscape as well as the key players in the business.

Macular degeneration is a condition in which, macula, a part of the retina, gets damaged or deteriorated. This condition usually affects individuals who are aged 50 years and above and therefore, it is called age-related macular degeneration (AMD). AMD is the leading cause of vision loss and is directly related to the advancement of age. But smoking also plays a vital role in causing AMD. AMD is characterized by the presence of a blurred area near the center of vision that leads to distorted vision. There are two different types of AMD, including dry (atrophic) AMD (dAMD) and wet (neovascular/exudative) AMD (wAMD). The dAMD is the most common type of AMD and accounts for almost 80%-90% of the overall AMD cases.

It has been observed that age-related macular degeneration (AMD) is one of the major causes for vision loss and is characterized by the formation of a blurred area near the center of vision, a condition that mostly affects the geriatric population. According to the CDC, almost 2 million individuals in the US suffer from AMD and by 2050, this number will reach more than 5 million. This will subsequently demand the need for the development of innovative treatments for AMD, driving the markets growth.

The market research analysts have predicted that with the introduction of techniques such as fluorescein angiography, the global age-related macular degeneration market will register a CAGR of more than 7% by 2020. With the unavailability of FDA-approved treatment for dry AMD (dAMD) and the treatment of wet AMD (wAMD) involving the need of intravitreal injections for an indefinite period, gene therapy is emerging as the most-efficient approach for the treatment of age-related macular degeneration (AMD).

According to this pipeline analysis report, most of the gene therapy molecules in the pipeline are being developed for wet AMD (wAMD). Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.

This report focuses on the global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China.

The key players covered in this study RetroSense Therapeutics REGENXBIO AGTC

Market segment by Type, the product can be split into Subretinal Intravitreal Unspecified

Market segment by Application, split into Monotherapy Combination Therapy

In this study, the years considered to estimate the market size of Gene Therapy for Age-related Macular Degeneration are as follows: History Year: 2014-2018 Base Year: 2018 Estimated Year: 2019 Forecast Year 2019 to 2025

Market segment by Regions/Countries, this report covers United States Europe China Japan Southeast Asia India Central & South America

The study objectives of this report are: To analyze global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. To present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China. To strategically profile the key players and comprehensively analyze their development plan and strategies. To define, describe and forecast the market by product type, market and key regions.

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.

Need a PDF of the global market report? Visit: https://industrystatsreport.com/Request/Sample?ResearchPostId=11998&RequestType=Methodology

Table of Content:

Market Overview: The report begins with this section where product overview and highlights of product and application segments of the Global Gene Therapy for Age-related Macular Degeneration Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company: Here, the competition in the Worldwide Global Gene Therapy for Age-related Macular Degeneration Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data: As the name suggests, this section gives the sales data of key players of the Global Gene Therapy for Age-related Macular Degeneration Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the Global Gene Therapy for Age-related Macular Degeneration Market.

Market Status and Outlook by Region: In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the Global Gene Therapy for Age-related Macular Degeneration Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User: This section of the research study shows how different end-user/application segments contribute to the Global Gene Therapy for Age-related Macular Degeneration Market.

Market Forecast: Here, the report offers a complete forecast of the Global Gene Therapy for Age-related Macular Degeneration Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion: This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

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Covid 19 Pandemic: Gene Therapy for Age-related Macular Degeneration Market Size is Thriving Worldwide- Demand and Analysis 2019-2025 - Cole of Duty

Doctors in Southern California are working with researchers in Arizona to better understand the bodys sometimes bizarre immune response to COVID-19 an antibody onslaught that may kill the patient, rather than kill the virus.

The nonprofit Translational Genomics Research Institute (TGen), an affiliate of City of Hope, is peering into specific proteins on the virus to see how they react with different antibodies a high-resolution view that might guide treatment, testing and vaccine development.

The hypothesis is that antibodies can make things worse, and thats whats killing some people, said John Altin, assistant professor in TGens infectious-disease branch. We want to understand how that might be different from an immune response that protects somebody.

As many critically ill patients are treated in clinical trials with convalescent plasma therapy that is, injecting antibodies from recovered COVID-19 patients into those who are very ill, in hopes of triggering protective immune responses its imperative to understand whats behind the differing reactions.

Usually, antibodies provide protection, but there may be a bit of an exception with this virus, Altin said. That is a serious concern.

To that end, TGen and the Center for Gene Therapy at City of Hope are cooperating on a COVID Immunity Study that aims to collect blood from COVID-19 survivors.

The researchers will analyze your blood and profile your immune memory, the study consent form explains.

Participants can use the TGen kit at home. Theyll get a study kit by mail and collect one small spot blood sample, via a finger-prick device, for two consecutive weeks. Then theyll mail the study kit back to TGen.

About 500 people are expected to participate through the course of the study, and researchers may reach out for additional samples, and/or with additional questions, to see how immune memory changes over time.

Participants must be U.S. residents, at least 18 years old, have tested positive for COVID-19, and then recovered. For more information, see https://covidimmunity.org/.

This will help us learn more about how, when and why we produce antibodies in response to a COVID-19 infection, said David Engelthaler, director of TGen North, in a prepared statement. One class of antibodies tackles the infection first, and then another comes in to finish the job. Knowing when these different immune responses occur, and how long they last, could help us understand if some patients gain a certain degree of immunity against reinfection. We need to know how that works.

While large-scale clinical trials involving convalescent plasma are under way all over the nation, this study aims not to treat the disease, but to better understand the mechanisms behind it.

TGen describes its approach as a high-resolution view of the antibody response. It seeks to not only map the viruss proteins in detail, but to also see which parts of those proteins are targeted by antibodies.

Our approach will not only tell you which proteins arebeing targeted, but also be able to tell which regions of each protein are being targeted, Altin said in a statement. Each protein can be recognized by many different types of antibodies. By looking at this level of detail, we then could see elements of the antibody response that others might be missing.

TGen hopes to tease out subtle differences that can help develop therapies, vaccines and better antibody testing.

Others are looking at responses to the entire protein. Our approach is a little different. When we look at the antibody response, we divide it up into thousands of pieces. Theres potential for that to tell us what a beneficial and un-beneficial response might look like, Altin said.

John Zaia, director of the Center for Gene Therapy at City of Hope, is working with TGen, and has other COVID-19-related projects happening as well.

Zaia is leading a research project at City of Hope, in collaboration with Altins lab, that could lead to development of a COVID-19 virus antibody neutralization test, which would quantify antibodies.

Zaia also has received a $750,000 grant from the California Institute for Regenerative Medicine for a clinical study on the use of blood plasma as a potential treatment for COVID-19.

Theyre doing what you could call qualitative and quantitative measurements of the nature of the antibody what does it actually bind to? Zaia said. The virus has this surface protein, the spike protein, but there are also other things the immune system might be seeing. It might be focused on one or more parts of the spike.

The CIRM project will focus on finding plasma donors to determine if theres any correlation between the outcome in the sick patient who received the plasma and the specific antibody that went in. It will focus on under-served areas.

Duarte-based City of Hope was founded in 1913 and is a founding member of the National Comprehensive Cancer Network. It has many sites throughout Southern California, and is investing $1 billion to establish clinics and a cancer center in Orange County. A clinic opened in Newport Beach in January, and a hospital dedicated to cancer treatment and research is slated for Irvine.

On the forefront of science, new discoveries are made every day and so much is still unknown.

I think the FDA said it best: Theres no way that one group could solve all the problems, do all the testing that needs to be done, Zaia said. The whole field is so new.

Theres a balance that must be struck between moving quickly and moving carefully, Altin said. We should know a lot in the next three months about how the antibody response looks, he said. Vaccine development will take much longer.

See original here:
Why does immune response to coronavirus save some, kill others? - East Bay Times

The Gene Therapies for Cancer Treatment market report provides a thorough analysis of this business landscape based on the consumption and production aspects. With respect to consumption, the report reviews the product consumption value as well as the product consumption volume alongside the individual sales trends of each product during the forecast period. In addition, details regarding the import and export graphs across the various geographies are also provided in the report.

According to Latest Research Report on Gene Therapies for Cancer Treatment Market size | Industry Segment by Applications (Cancer Research Centers,Diagnostic Laboratories,Cancer Hospitals andOthers), by Type (Somatic Cell Gene Therapy (SCGT) andGermline Gene Therapy (GGT), Regional Outlook, Market Demand, Latest Trends, Gene Therapies for Cancer Treatment Industry Share, Research Growth Forecast & Revenue by Manufacturers, The Leading Company Profiles, Growth Forecasts 2026.

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Based on the production aspect, the report covers the manufacturing of the product, its revenue, and gross margins garnered by the market majors. Variation in unit costs strategized by these manufacturers across various regional markets during the analysis period are also entailed in the report.

A brief of the regional outlook:

An overview of the product spectrum:

A gist of the application terrain:

Insights regarding the competitive terrain:

In summary, the Gene Therapies for Cancer Treatment market report is evaluated through several categorizations, including the basic industry definitions. Information pertaining the upstream raw materials, downstream buyers, and distribution channels of the competitors are discussed in the report. The study also examines the key drivers, restraints and opportunities that will impact the growth trends in the ensuing years.

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Gene Therapies for Cancer Treatment Market Report, History and Forecast 2015-2026, Breakdown Data by Manufacturers, Key Regions, Types and Application...

People who are overweight and suffering from joint pain caused by osteoarthritis are often reluctant to exercise, even though physical activity can boost muscle strength and relieve pain. A new study suggests gene therapy may someday be a good option for those peopleand it may help them shed pounds, too.

Researchers at the Washington University School of Medicine gave young mice a single injection of the gene that makes follistatin, a protein that normally blocks another protein called myostatin, which modulates muscle growth. The therapy caused a significant buildup of muscle mass in the mice while also preventing obesity, the team reported in the journal Science Advances.

We've identified here a way to use gene therapy to build muscle quickly, said senior investigator Farshid Guilak, Ph.D., professor of orthopaedic surgery and director of research at Shriners Hospitals for Children St. Louis, in a statement. It had a profound effect in the mice and kept their weight in check, suggesting a similar approach may be effective against arthritis, particularly in cases of morbid obesity."

In fact, the mice didnt just build muscle, they also nearly doubled their strength without exercising any more than they usually did. Despite being fed a high-fat diet, they had fewer metabolic issues and stronger hearts than did animals that did not receive the follistatin gene. Their joints were healthier, with less cartilage damage and inflammatory markers than their untreated counterparts, the researchers reported.

Whats more, the Washington University team discovered that the gene therapy promoted the beiging of white fat, meaning it turned some unhealthy white adipose tissue into brown fat, which positively correlates with increased triglyceride clearance, normalized glucose level, and reduced inflammation, the researchers wrote in the study. Therefore, delivering the follistatin gene could serve as a very promising approach to induce beiging of [white adipose tissue] in obesity, they wrote.

RELATED: Could gene therapy be the solution to obesity and diabetes?

This is not the first time gene therapy has been proposed as a potential treatment for obesity and other metabolic diseases. Australian researchers demonstrated that removing the gene RCAN1 from mice, for example, helped turn white fat into brown fat. And a team in South Korea used the gene editing system CRISPR to remove the FABP4 gene from mice that had been fed a high-fat diet, resulting in a 20% loss of body weight and a reduction in insulin resistance.

The Washington University teams approach is distinctive in that it focuses on building muscle. But the researchers noted theyll have to do further studies to determine whether the gene therapy has any negative effect on heart muscle. Even though heart health improved in the mice, any thickening of the hearts walls could be dangerous over time.

Still, Guilak and his colleagues believe that follistatin gene therapy could be a promising approach to treating several conditions, including muscular dystrophy and other diseases that cause muscle wasting, they said in the study.

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Gene therapy cuts fat and builds muscle in sedentary mice on unhealthy diets - FierceBiotech

California-based Kriya Therapeutics announced on Tuesday that it concluded an $80.5 million Series A financing round, led by QVT, Dexcel Pharma, Foresite Capital, Bluebird Ventures, Narya Capital, Amplo, Paul Manning, and Asia Alpha. The company, which was founded in 2019, has a pipeline that includes multiple AAV-based gene therapies for the treatment of type 1 and type 2 diabetes, as well as obesity.

"There have been numerous successful gene therapies focused on rare monogenic diseases in recent years," said Shankar Ramaswamy, M.D., Co-Founder, Chairman, and CEO of Kriya Therapeutics. "We see tremendous potential to expand the field and apply gene therapy to highly prevalent serious diseases. We are focused on designing gene therapies using algorithmic tools, scalable infrastructure, and proprietary technology to optimize the efficacy and durability of our treatments. We look forward to accelerating the development of our pipeline, platform technologies, and internal GMP manufacturing capability with the funds raised in this Series A financing."

Kriya focuses on developing gene therapies for conditions that impact millions of patients. Its goal is to design one-time gene therapies to express therapeutic proteins within specific human tissues. Kriyas pipeline includes KT-A112, KT-A522, and KT-A832, all of which are investigational gene therapies.

Kriyas leadership team is composed of experts who have experience designing, developing and manufacturing successful gene therapies as well.

"Kriya is building a leading team and cutting-edge infrastructure to engineer best-in-class gene therapies for severe chronic conditions and accelerate their advancement into human clinical trials," said Roger Jeffs, Ph.D., Co-Founder and Vice Chairman of Kriya. "The company is committed to incorporating the latest advancements in the field into the design and development of its therapeutic constructs. Through its R&D laboratory capabilities in the Bay Area and in-house process development and manufacturing infrastructure in Research Triangle Park, I believe that Kriya will be uniquely positioned to become a leader in the gene therapy field."

Another company with a focus on gene therapy that recently came out of stealth mode is Dyno Therapeutics. On Monday, the Massachusetts-based organization announced that it is now eligible for more than $2 billion in upfront payments, research support and various milestones and options fees through its research-and-development and collaboration deals.

Dyno, which launched in 2018 with approximately $9 million in financing, has a technology platform built on the intellectual property that came from the laboratory of George Church. Church, who is the Robert Winthrop Professor of Genetics at Harvard Medical School, is a cofounder of Dyno.

At Dyno, we see a vast opportunity to expand the treatment landscape for gene therapies, said Eric D. Kelsic, co-founder and chief executive officer of Dyno. The success of gene therapy relies on the ability of vectors to safely and precisely deliver a gene to the intended target cells and tissues. Our approach addresses the major limitations of naturally occurring AAV vectors and creates optimized, disease-specific vectors for gene therapies with great curative potential. Our portfolio of R&D programs and newly-announced collaborations with leading gene therapy developers reflect the applicability of our AI-powered approach to improve treatments for patients and expand the number of treatable diseases with gene therapies.

Dyno has announced partnerships with Novartis and Sarepta Therapeutics thus far. With Sarepta, Dyno will design and discover novel AAV capsids to improve gene therapy for muscle diseases. Along with Novartis, Dyno will focus on developing improved AAV vectors for gene therapies for ocular diseases.

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Kriya Therapeutics to Focus on Gene Therapy with $80.5M in Funding - BioSpace

A gene therapy developer emerging from stealth mode said today it could generate more than $2 billion through collaborations launched with Novartis and Sarepta Therapeutics to develop treatments based on a delivery platform developed in the lab of George Church, PhD, of Harvard Medical School.

Dyno Therapeutics said it will partner with Novartis on research, development, and commercialization of new gene therapies for eye diseases incorporating improved adeno-associated virus (AAV) vectors. Separately, Dyno will collaborate with Sarepta on gene therapies for muscle diseases.

Both collaborations will apply Dynos CapsidMap platform, which uses artificial intelligence (AI) to design novel capsids that confer improved functional properties to AAV vectors. At the core of CapsidMap, according to Dyno, are advanced search algorithms applying machine learning and the companys large quantities of experimental data.

CapsidMap is designed to integrate DNA library synthesis and next generation DNA sequencing to measureinvivogene delivery properties in high throughput.The capsid platform is designed to expand the universe of diseases treatable via gene therapy by improving upon present-day AAV vectors, which are limited by delivery, immunity, packaging size, and manufacturing challenges.

Our portfolio of R&D programs and newly-announced collaborations with leading gene therapy developers reflect the applicability of our AI-powered approach to improve treatments for patients and expand the number of treatable diseases with gene therapies, Eric Kelsic, PhD, a former postdoc of Church who is Dynos CEO and Co-founder, said in a statement. We see a vast opportunity to expand the treatment landscape for gene therapies.

Dyno has an exclusive option to enter into a license agreement with Harvard University for the CapsidMap technology, which was developed in the lab of Church, a co-founder of Dyno and chairman of its scientific advisory board. Church is the Robert Winthrop Professor of Genetics at Harvard Medical School and a Core Faculty member at Harvards Wyss Institute for Biologically Inspired Engineering.

Church and other co-founders and members of his lab at HMS and the Wyss Institute carried out work that underpin Dynos approach to AAV capsid engineering, described in Comprehensive AAV capsid fitness landscape reveals a viral gene and enables machine-guided design, a paper published November 29, 2019, in the journal Science.

In that paper, Church, Kelsic, and two co-authors detailed how they mutated one-by-one each of the 735 amino acids within the AAV2 capsid, including all possible codon substitutions, insertions and deletions at each position. They generated a virus library containing about 200,000 variants and identified capsid changes that both maintained AAV2s viability and improved its homing potential (tropism) to specific organs in mice.

Unexpectedly, the team also discovered a new accessory protein hidden within the capsid-encoding DNA sequence that binds to the membrane of target cells, playing a significant role in viral production.

Our comprehensive, machine-guided design strategy generated viable mutants in a principled and high-throughput manner and is generalizable to other proteins and engineering challenges, the researchers reported. Applied to AAV, such methods now enable the systematic optimization of natural capsids into synthetic variants with enhanced properties for emerging gene therapies.

Under its partnership with Novartis, Dyno will be responsible for using AI technology and its suite of machine learning and experimental tools for the design and discovery of novel AAV capsids. Novartis agreed to conduct preclinical, clinical, and commercialization activities for the gene therapy product candidates created with the novel AAV capsids.

Dyno and Novartis did not disclose specific financial terms of their collaboration. They did say, however, that Novartis agreed to pay Dyno an upfront fee plus committed research funding and license fees. Dyno will be eligible to receive clinical, regulatory and sales milestone payments. Dyno will also receive royalties on worldwide net sales of any commercial products developed through the partnership.

Many eye diseases are ideally suited to being treated with gene therapies, and more opportunities can be opened with new and improved AAV vectors, Kelsic added. With their extensive ophthalmologic expertise, Novartis is an ideal partner to leverage Dynos platform to design AI-powered vectors to expand the impact of gene therapies for ocular diseases.

Under its collaboration with Sarepta, Dyno agreed to oversee the design and discovery of novel AAV capsids with improved functional properties for gene therapy, while Sarepta agreed to take responsibility for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the novel capsids.

Our agreement with Dyno provides us with another valuable tool to develop next-generation capsids for gene therapies to treat rare diseases, stated Doug Ingram, Sareptas president and CEO. By leveraging Dynos AI platform and Sareptas deep expertise in gene therapy development, our goal is to advance next-generation treatments with improved muscle-targeting capabilities.

Sarepta agreed to pay Dyno a total $40 million in in upfront, option, and license payments during the research phase of their collaboration. Should Sarepta develop and commercialize multiple candidates for multiple muscle diseases, Dyno said, it will be eligible for additional significant future milestone payments. Dyno will also receive royalties on worldwide net sales of any commercial products developed through the collaboration.

Dyno was launched in late 2018 with a $9 million financing co-led by Polaris Partners and CRV. Alan Crane, a co-founder of Dyno and entrepreneur partner at Polaris Partners, and Dylan Morris, general partner at CRV, have joined Dynos board, with Crane serving as Dynos executive chairman.

Dyno said it did not anticipate the need for additional fundraising, based on the significant financial resources made available through its collaborations.

Link:
Dyno Inks Up to $2B+ in Gene Therapy Partnerships with Novartis, Sarepta - Genetic Engineering & Biotechnology News

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders, today presents new clinical data supporting longer-term efficacy and durability of gene therapy for Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held virtually May 12-15, 2020. Two oral presentations highlight updates from the companys Phase 1/2 study of RP-L201 for the treatment of severe LAD-I and the Phase 1/2 study of RP-L102 Process A for the treatment of FA.

The latest additional data from both our LAD-I and FA programs demonstrate sustained engraftment and durable clinical impact, said Jonathan D. Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. These results further support the viability of gene therapy in LAD-I and FA, disorders in which bone marrow transplant is the primary curative option and is associated with high rates of toxicity.

Patients with severe LAD-I have neutrophil CD18 expression of less than 2% of normal, with extremely high mortality in early childhood, said Dr. Schwartz. In this first patient treated with RP-L201 using Process B, an increase from less than 1% to 47% CD18 expression sustained over six months demonstrates that RP-L201 has the potential to correct the neutrophil deficiency that is the hallmark of LAD-I. We are also pleased with the continued visible improvement of multiple disease-related skin lesions. These results lend further support to the applicability of Process B across the lentiviral portfolio. The second patient has also recently been treated, and we look forward to completing the Phase 1 portion of the registrational trial for this program.

Dr. Schwartz continued, In our FA program, patients followed for a year or more after treatment with RP-L102 Process A continue to demonstrate durable engraftment and hematologic correction, without the use of pre-treatment conditioning regimens. All six patients who received minimally adequate drug product and were followed for more than one year display sustained and progressive engraftment. Notably, hemoglobin levels have normalized to baseline in two patients treated. Todays update not only gives us confidence as we transition to our improved Process B drug product, but also supports the potential of gene therapy in the absence of any conditioning regimen as a definitive hematologic treatment for FA. The ability to treat patients without the side effects associated with allogeneic transplant or the use of genotoxic conditioning, and to restore blood cell counts is a major milestone for the FA scientific community.

Details on Rockets oral presentations at ASGCT:

Title: A Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Initial Results from the First Treated Patient Session: HSPC Gene Therapies for Blood and Immune DisordersPresenter: Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and principal investigator of the Phase 1 trialDate: Tuesday May 12, 2020Time: 4:30 p.m. - 4:45 p.m. EDT

Additional results from the first patient treated with RP-L201 for LAD-I continue to demonstrate evidence of safety and potential efficacy. Analyses of peripheral vector copy number (VCN) and CD18-expressing neutrophils were performed six months post treatment with RP-L201 to evaluate engraftment and phenotypic correction. The patient demonstrated peripheral blood VCN levels of 1.3 and CD18-expression of 47%, which is sustained from the 45% expression observed three months post treatment; pretreatment CD18 expression was <1%. The drug product VCN was 3.8. Additionally, the patient continues to display visible improvement of skin lesions. No safety or tolerability issues related to RP-L201 administration have been identified to date.

RP-L201 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacin Sanitaria Fundacin Jimnez Daz (IIS-FJD). The lentiviral vector was developed in a collaboration between The University College of London (UCL) and CIEMAT.

Title: Updated Results of a European Gene Therapy Trial in Fanconi Anemia Patients, Subtype ASession: HSPC Gene Therapies for Blood and Immune DisordersPresenter: Juan A. Bueren, Ph.D., Scientific Director of the FA gene therapy program and Head of the Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBERER / IIS-FJD Date: Tuesday May 12, 2020Time: 4:45 p.m. - 5:00 p.m. EDT

Nine pediatric patients have been enrolled and treated in the Phase 1/2 clinical trial of RP-L102 Process A for the treatment of Fanconi Anemia, seven of whom are evaluable at or beyond the one year mark following treatment. The first four patients (02002, 02004, 02005 and 02006) exhibit robust and durable engraftment, continued hematologic correction and blood count stabilization. Importantly, hemoglobin levels for patients 02002 and 02006 have increased to a healthy, normal range; these patients received more optimal product consistent with the minimal dose criteria established for the Process B registrational program. Two additional patients (02008 and 02013) who have been followed for a year or more after treatment display early evidence of engraftment, as measured by increases in peripheral blood VCNs. Patient 02007 received a lower than optimal dose and is beginning to demonstrate preliminary signs of engraftment. Blood counts are not yet available in these patients. Two patients, patients 01003 and 02009, have not been included in this analysis. Patient 02009 is only six months post treatment and will continue to be followed. Patient 01003 received a drug product that did not meet full release criteria due to a technical issue this was a one-time lab-specific issue that was addressed. To date, no patients in this trial have undergone allogeneic bone marrow transplant.

RP-L102 is being developed in conjunction with CIEMAT, CIBERER and IIS-FJD.

The presentations will be made available on Rockets website at http://www.rocketpharma.com/asgct-presentations/ following presentation at the conference.

About Leukocyte Adhesion Deficiency-ISevere Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Fanconi AnemiaFanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking StatementsVarious statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, filed May 8, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Presents Positive Updates on FA and LAD-I Gene Therapy Programs at the 23rd Annual Meeting of the American Society of Gene and...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a pioneer in T-cell immunotherapy leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with severe diseases including solid tumors, hematologic cancers and autoimmune diseases, today announced the appointment of immunology and cell & gene therapy expert Maria Grazia Roncarolo, MD, to the Board of Directors.

Dr. Roncarolo is the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and Medicine, Director of the Center for Definitive and Curative Medicine, and Co-Director of the Institute for Stem Cell Biology and Regenerative Medicine at Stanford University.

In 2014, Dr. Roncarolo established the Stanford Center for Definitive and Curative Medicine. The center, which is dedicated to the development of innovative stem cell and gene therapies for patients with currently incurable diseases, spans a wide range of bench and clinical research activities from basic biology through translational research and features its own GMP cell processing and Phase 1 study units.

I am thrilled to have one of the worlds leading experts in immunology and T cells, Dr. Roncarolo, bringing to our Board her experience and strategic vision in cell therapy and gene editing as well as her passion for transformative immunotherapies, said Pascal Touchon, President and Chief Executive Officer of Atara. She has dedicated her life to caring for patients with severe immunological and hematological diseases and has an impressive record in translating scientific discoveries in cell and gene therapy into novel treatments which aligns very well with Ataras mission.

Dr. Roncarolo has served as the primary investigator in several landmark trials involving the development of innovative stem cell- and gene-based therapies. She worked at DNAX Research Institute for Molecular and Cellular Biology in Palo Alto for several years, where she contributed to the discovery of novel cytokines, cell-signaling molecules that are part of the immune response. She studied the role of these cytokines in inducing immunological tolerance and in promoting stem cell growth and differentiation. As Director of the Telethon Institute for Cell and Gene Therapy and the San Raffaele Scientific Institute in Milan, Dr. Roncarolo was the principal investigator leading the successful gene therapy trial in SCID, a severe life threatening disorder in which patients lack an enzyme critical to DNA synthesis.

Beyond studying new therapies, Dr. Roncarolo has also helped elucidate drivers of disease at the molecular and cellular level, as she has investigated the mechanisms of immune-mediated diseases throughout her career and helped advance the understanding of immunological tolerance. Dr. Roncarolo was the recipient of the outstanding achievement award from the European Society of Gene and Cell Therapy (ESGCT) in 2010 and from the American Society of Gene and Cell Therapy (ASGCT) in 2017. She is currently the president of the Federation of Clinical Immunology Societies.

About Atara Biotherapeutics

Atara Biotherapeutics, Inc. (@Atarabio) is a pioneer in T-cell immunotherapy leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with severe diseases including solid tumors, hematologic cancers and autoimmune disease. With our lead program in Phase 3 clinical development, Atara is the most advanced allogeneic T-cell immunotherapy company and intends to rapidly deliver off-the-shelf treatments to patients with high unmet medical need. Our platform leverages the unique biology of EBV T cells and has the capability to treat a wide range of EBV-associated diseases, or other severe diseases through incorporation of engineered CARs (chimeric antigen receptors) or TCRs (T-cell receptors). Atara is applying this one platform to create a robust pipeline including: tab-cel (tabelecleucel) in Phase 3 development for Epstein-Barr virus-driven post-transplant lymphoproliferative disease (EBV+ PTLD); ATA188, a T-cell immunotherapy targeting EBV antigens as a potential treatment for multiple sclerosis; and multiple next-generation chimeric antigen receptor T-cell (CAR T) immunotherapies for both solid tumors and hematologic malignancies. Improving patients lives is our mission and we will never stop working to bring transformative therapies to those in need. Atara is headquartered in South San Francisco and our leading-edge research, development and manufacturing facility is based in Thousand Oaks, California. For additional information about the company, please visit atarabio.com and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains or may imply "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Because such statements deal with future events and are based on Atara Biotherapeutics' current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Atara Biotherapeutics could differ materially from those described in or implied by the statements in this press release. These forward-looking statements are subject to risks and uncertainties, including those discussed in Atara Biotherapeutics' filings with the Securities and Exchange Commission (SEC), including in the Risk Factors and Managements Discussion and Analysis of Financial Condition and Results of Operations sections of the Companys most recently filed periodic reports on Form 10-K and Form 10-Q and subsequent filings and in the documents incorporated by reference therein. Except as otherwise required by law, Atara Biotherapeutics disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

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Atara Biotherapeutics Announces Appointment of Cell & Gene Therapy Expert Maria Grazia Roncarolo, MD to Board of Directors - Business Wire

DUBLIN Dyno Therapeutics Inc., an early stage gene therapy firm applying artificial intelligence to advanced capsid engineering, has entered partnerships with Novartis AG and Sarepta Therapeutics Inc., in ophthalmic indications and muscle diseases, respectively, which have more than $2 billion in biobucks attached. Further financial details are scant, although it could receive more than $40 million in up-front, option and license payments during the research phase of the Sarepta alliance. The two deals are evidence of the market appetite for improved adeno-associated viral vectors. We had a significant amount of interest even before the company was formed, Dyno CEO and co-founder Eric Kelsic told BioWorld.

The phase III pivotal trial of mavacamten, an oral, allosteric cardiac myosin modulator for treating symptomatic, obstructive hypertrophic cardiomyopathy, from Myokardia Inc., of Brisbane, Calif., hit its primary and all secondary endpoints. The data show mavacamten was well-tolerated and demonstrated safety results comparable to placebo. Ninety-eight percent of patients enrolled completed the study. The company stock (NASDAQ:MYOK) was met by enthusiasm midday Monday, as shares swelled by 62%. Myokardia said it plans to submit an NDA in the first quarter of 2021. Myokardia is developing mavacamten to treat conditions in which excessive cardiac contractility and impaired diastolic filling of the heart are the underlying cause.

After being hit with the major market meltdown during March, public biopharmaceutical companies developing new medicines put that behind them with a dramatic surge in valuations in April. As a result, the BioWorld Drug Developers index recorded an almost 20% increase during the period, with that momentum continuing into early May.

By delivering the protein follistatin via gene therapy, researchers at Washington University in St. Louis were able to increase skeletal muscle mass, decrease inflammation and reverse obesity-related arthritis in mice who developed osteoarthritis as a result of a high-fat diet. They reported their results in the May 8, 2020, online issue of Science Advances.

HONG KONG The Japanese government is tightening its grip on its listed companies, including those working on promising COVID-19 treatments. On May 8, the Japanese Ministry of Finance releaseda list of 518 companies that would be subject to stricter restrictions on receiving foreign investments. Starting on June 7, foreign investors buying a stake of 1% or more in Japanese firms will be pre-screened, compared with the previous limit of 10%. In a statement, the ministry said the move was related to the degree of impact of the investment on maintaining the basis of production and technologies in the business sectors that relate to protection of national security, maintenance of public order, or safeguard of public safety.

DUBLIN Tolerogenixx GmbH is on track to move its cell-based immune tolerance induction therapy for kidney transplant recipients into a 200-patient phase IIb trial, following the publication of promising data from a phase Ib trial in 10 patients, in which all participants had a successful transplant at one-year follow-up, including those in a high-dose group on a reduced immunosuppression regimen.

Having a COVID-19 therapy approved through an emergency use authorization (EUA) is not the same as having access to it, even if its free. With one-third of the COVID-19 cases confirmed globally as of today and 28.5% of the deaths, the U.S. is getting 40% of the 1.5 million doses of remdesivir Gilead Sciences Inc. is donating worldwide. The federal government last week began doling out the 607,000 doses to the states, which are then charged with distributing them to the hospitals with the greatest need. But given the supply and manufacturing timeline for the first drug granted an EUA to treat severe COVID-19 cases, more than 300,000 eligible U.S. patients likely will not have access to the drug through the end of July, said Brian Abrahams, a senior analyst with RBC Capital Markets LLC.

HONG KONG South Koreas Hanmi Pharmaceutical Co. Ltd. has filed a new drug approval application for Rolontis (eflapegrastim) with the countrys Ministry of Food and Drug Safety. Rolontis, a biologic to treat neutropenia, is the first of its kind in South Korea and, according to Hanmi, the first to be developed using the companys Lapscovery platform.

About 15 months after closing its multibillion-dollar acquisition of Loxo Oncology Inc., Eli Lilly and Co. has secured an accelerated FDA approval for the first of the deal's headline assets, the RET kinase inhibitor selpercatinib, now branded as Retevmo. The green light, following a priority review, allows for marketing of the drug as a treatment for three types of tumors non-small-cell lung cancer, medullary thyroid cancer and other types of thyroid cancers in patients whose tumors have a rearranged during transfection alteration. The decision arrived well ahead of an earlier-projected third-quarter decision by the agency.

Three biopharma drugs are up for FDA approval this week, including one new chemical entity, dasotraline, from Sunovion Pharmaceuticals Inc. to treat binge eating disorder, and two other candidates that are part of supplemental filings for expanded oncology indications. Sunovions drug and Blueprint Medicines Corp.s Ayvakit (avapritinib) to treat fourth-line gastrointestinal stromal tumors (GIST), have PDUFA dates set for May 14, while Clovis Oncology Inc.s Rubraca (rucaparib) for prostate cancer is scheduled for May 15.

The articles from BioWorlds ongoing coverage of the COVID-19 coronavirus outbreak are available at http://www.bioworld.com/coronavirus. Note that we have added three critical tables which are constantly updated:

4D Pharma, 9 Meters, Abbvie, ADC, Amag, Appili, Artara, Astrazeneca, Bellerophon, Biomarin, Bluebird, Bridgebio, Burning Rock, Cellect, Cocrystal, Crispr, Daicchi, Dar, Faron, Histogen, Hummingbird, Infinity, Inmune, Intercept, Jazz, Kiniksa, Kura, Lilly, Merck, Mimedx, Myokardia, Navidea, Noxxon, Orexo, Oyster Point, Pliant, Plus, Portage, Protagonist, Protalix, Regeneron, Relief, Ritter, Strongbridge, Themis, Tiburio, Tolerogenixx, Tracon, Vertex

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Novartis, Sarepta join Dyno's enterprise to boldly go to new gene therapy frontier - BioWorld Online

The Cell and Gene Therapy Catapult (CGT Catapult) and Kyoto, Japan-based CiRA Foundation are launching a new collaborative research project focused on induced pluripotent stem (iPS) cell characterisation.

With the move, the groups are hoping to further the application of iPS cell technologies for the manufacture of regenerative medicine products.

The potential of distinct iPS cell lines for differentiation into specific cell types is usually biased towards some cell line-specificity which, the parties note, is very difficult to predict. As such, in order to select an appropriate iPS cell line for clinical trials it is necessary to differentiate several candidate cell lines, which is time-consuming.

CGT Catapult and CiRA plan to explore novel methods of evaluating cell differentiation and aim to establish reliable tests to predict the potential of iPS cell to differentiation bias, a capability that would help to advance the use of iPS cells for regenerative medicine products.

We are honoured to collaborate with CiRA Foundation, an organisation with world-leading capabilities in iPS cell technology, and to be the first group to utilise CiRAs innovative iPS cell lines outside of Japan, said CGT's chief executive Matthew Durdy

This is a truly exciting project to help further the application and manufacture of iPS cells into cell therapies. We look forward to progressing this promising research together, which has potential benefits for the global advanced therapies industry.

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Cell and Gene Therapy Catapult links with Japan's CiRA Foundation - PharmaTimes

For its first quarter ended March 31, 2020, the Maryland-based company, reported revenue of $1.9 million, compared to $0.4 million a year earlier

Inc (), a global biotech company focused on accelerating and transforming the delivery of cell and gene therapies, posted first-quarter results on Monday that saw its revenue soar 348% year-over-year driven by its Cell and Gene Therapy (CGT) Biotech platform.

For its first quarter ended March 31, 2020, the Germantown, Maryland-based company, reported revenue of $1.9 million, compared to $0.4 million in the first quarter of 2019.

Orgenesis achieved net income of $75.6 million, or $4.23 per share, reflecting the sale of subsidiary Masthercell Global Inc, a contract development manufacturing organization (CDMO).

READ:Orgenesis boss Vered Caplan makes top 20 list of inspirational leaders in advanced medicine

On February 11, Orgenesis completed the successful sale of its CDMO business to Somerset, New Jersey-based Catalent Pharma Solutions, for around $127 million.

As a result, Orgenesis reported cash and equivalents of $107.1 million as of March 31, 2020.

In a statement accompanying the numbers, Orgenesis CEO Vered Caplan said: Step by step, our CGT Biotech Platform is gaining traction within the market, as illustrated by the year-over-year growth.

In the first quarter of 2020, revenue increased to $1.9 million, or nearly an $8 million revenue run rate compared to $3.1 million for all of 2019. We believe our CGT Biotech Platform is poised for growth this year through industry partnerships that are currently underway with leading research institutes and hospitals around the world, she added.

The companys CGT Biotech platform consists of three core elements:point-of care Therapeutics, point-of care Technologies, and point-of care Network.

Caplan also noted that earlier this year, the company struck collaboration agreements with two leading healthcare research institutes in the US.

We plan to utilize our point-of-care Network to support their growing development and processing needs in order to advance and accelerate cell and gene-based clinical therapeutic research, said Caplan.

Orgenesis is using the Masthercell sale proceeds to expand the companys point-of-care cell therapy business. The biotech is currently focused on therapies which span a wide range of treatments.

In addition to our POCare Network, we are building our pipeline of POCare Therapeutics and Technologies, with an ultimate goal of providing life-changing treatments to large numbers of patients at reduced costs within the point-of-care setting, said Caplan.

Specifically, we are focusing on immune-oncology, metabolic and autoimmune diseases, as well as anti-viral therapies.

Orgenesis also recently completed the acquisition of Tamir Biotechnology and its broad-spectrum antiviral platform, ranpirnase in a cash and stock deal for roughly $21 million. The company will use ranpirnase to target human papillomavirus (HPV), which causes genital warts.

Ranpirnase has demonstrated clinical efficacy against HPV and other hard to target viruses based on its unique mechanism of action of killing the virus and modulating the immune system, said Caplan.

Going forward, Orgenesis plans to move the program through a Phase 2b trial in the US.

Meanwhile, the Orgenesis boss said the company has received a nod from regulators to keep research alive at its labs during the coronavirus (COVID-19) pandemic.

We are leveraging all our knowledge and expertise in the field of cell and gene therapy, including anti-viral technologies, in an attempt to find potential COVID-19 cures and therapies, said Caplan.

Importantly, we have a strong balance sheet and are strategically positioned to bring a variety of therapies to market in a cost-effective, high-quality and scalable manner.

At the start of April, Orgenesis teamed up with regenerative medicine and cell therapy firm RevaTis on a new joint venture to produce certain stem cells. The two firms plan to leverage Orgenesiss autologous CGT Biotech platform to advance clinical trials.

Under the deal, RevaTis and Orgenesis will use the formers patented technique to obtain muscle-derived mesenchymal stem cells (mdMSC) as a source of exosomes and various other cellular products.

Our plan is to combine RevaTis patented technique to obtain mdMSCs through a minimally invasive muscle micro-biopsy with our own automated/closed-systems, 3D printing, and bioreactor technologies, said Caplan.

The goal of this JV is to lower the costs and accelerate the timeline of bringing these innovative therapies through the clinic and into commercialization.

Contact the author Uttara Choudhury at [emailprotected]

Follow her on Twitter: @UttaraProactive

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Orgenesis sees 1Q revenue rocket driven by its Cell and Gene Therapy Biotech platform - Proactive Investors USA & Canada

Amber Freeds singular mission is to help scientists develop a treatment for her son. But the pandemic has put the success of that mission into question.

Freed is a mother to twins Riley and Maxwell, who just turned 3 this year. Riley is a healthy toddler. But Maxwell has a rare genetic disease that has led to delays in his development. Hes nonverbal, and he has a movement disorder. The condition responsible for these symptoms is called SLC6A1, after the gene thats affected, and Maxwell was only the 34th person in the world to get this diagnosis. if left untreated, the disease could soon cause debilitating epilepsy.

When doctors broke the news of the diagnosis to Freed, they told her there was no treatment or cure for her son, and that her family would have to prepare to live with Maxwells condition. But Amber didnt accept that.

Every instinct in my body said, Youll have your whole lifetime to cry for yourself, Freed said. At this exact moment, you put your feelings and sadness aside. This isnt about you. This is about Maxwell. And you fight like the third monkey on the loading deck to Noahs Ark, and its starting to rain.

This is how Freeds quest began. She taught herself microbiology, so she could understand her sons condition. She lobbied scientists to take up the research, which wasnt on their radar given how rare the disease is. She convinced scientists in China to make genetically engineered mice that could be sent to the US and act as a model of Maxwells disease. And she fundraised a necessary step given the high cost of the gene therapy research.

All told, shes raised more than $1 million. What were doing will be the building blocks of all gene therapies to come after us, Freed told Arielle Duhaime-Ross on an episode of Reset in January.

Even though she was racing against time, she was optimistic that Maxwell would be treated this year, as experiments on the genetically modified mice were about to begin.

Then the pandemic hit.

Covid-19 has brought most non-coronavirus medical research to a halt, and closed labs.

I never anticipated or thought something like this could happen, Freed said, but also it was a state of devastation like I felt when Maxwell was originally diagnosed that lightning hit us. The unthinkable happened. And here, lightning has hit again. A black swan event for the world that no one could have anticipated.

Freed came back to Reset to talk about the impact of the pandemic on Maxwell and her family as well as rare disease research as a whole. Listen to the full episode below.

The episode also features a conversation with BuzzFeed science reporter Dan Vergano, who first wrote about Ambers story and who talks about how the closure has impacted thousands of other kids with rare diseases, as well as clinical trials for other types of medical research.

Listen and read more:

Listen to the original Reset story from January

Dan Verganos reporting on this story for BuzzFeed

Back in January, Dans piece on how the mice were being used to develop a treatment

Milestones for Maxwell, Amber Freeds website

Original post:
A treatment for this toddlers rare genetic condition was in sight. Then the pandemic hit. - Vox.com

DetailsCategory: More NewsPublished on Wednesday, 06 May 2020 09:49Hits: 453

PRATTELN, Switzerland I May06, 2020 I Santhera Pharmaceuticals (SIX: SANN) announces the signing of two agreements with Rutgers, The State University of New Jersey as part of its program to advance gene therapy research for the treatment of LAMA2-deficient congenital muscular dystrophy (LAMA2MD or MDC1A). Under the agreements, Santhera gains rights to intellectual property developed at Rutgers on certain gene constructs that will be further studied under a collaboration agreement.

Santhera has entered into a license agreement with Rutgers, The State University of New Jersey and a collaboration with Prof. Peter Yurchenco, a pioneer in a novel gene therapy approach for the treatment of LAMA2MD. These agreements complement the ongoing collaboration of Santhera with Prof. Markus Regg from the Biozentrum of the University of Basel [1]. Previous collaborative work by Prof. Regg and Prof. Yurchenco has established the potential of this approach in animal models.

The novel gene therapy strategy developed by these leading experts uses two linker proteins that are composed of domains derived from extracellular matrix proteins agrin, laminin and nidogen [2-5]. In animal models for LAMA2MD, this approach has led to restoration of muscle fiber basement membranes, recovery of muscle force and size, increased overall body weight and markedly prolonged survival thus demonstrating strong evidence for disease modifying potential [2].

The coordinated work of both collaborations will further advance Santheras effort to bring this innovative gene therapy approach to patients with LAMA2MD.

Gene replacement is a promising therapeutic option for the treatment of LAMA2MD, said Peter D. Yurchenco, MD, PhD, Professor at Rutgers Robert Wood Johnson Medical School, USA. We have been working on continuously optimizing linker proteins engineered from extracellular matrix proteins which will aid in advancing such gene therapy approach towards clinical use.

Santhera is excited to extend its collaborative network for this therapeutic approach, now including experts from Rutgers University, added Kristina Sjblom Nygren, MD, Chief Medical Officer and Head of Development of Santhera. This will add value to our gene therapy program for LAMA2MD and complements the work already under way with the Biozentrum at the University of Basel, which was awarded a grant by Innosuisse in 2019. Both of our collaboration partners have pioneered this field and will work closely with Santhera, clinical experts and the patient community to establish the best way to bring this approach to clinical use.

About LAMA2MD (CMD Type 1A or MDC1A) and Emerging Therapy Approaches

Congenital muscular dystrophies (CMDs) are inherited neuromuscular diseases characterized by early-onset weakness and hypotonia alongside associated dystrophic findings in muscle biopsy. Progressive muscle weakness, joint contractures and respiratory insufficiency characterize most CMDs. Laminins are proteins of the extracellular matrix that help maintain muscle fiber stability by binding to other proteins. LAMA2-related muscular dystrophy (LAMA2MD, also called MDC1A), is one of the most common forms of CMD. It is caused by mutations in the LAMA2 gene encoding the alpha2 subunit of laminin-211. Most LAMA2MD patients show complete absence of laminin-alpha 2, are hypotonic (floppy) at birth, fail to ambulate, and succumb to respiratory complications.

Previous work has demonstrated that two linker proteins, engineered with domains derived from the extracellular matrix proteins agrin, laminin and nidogen, could compensate for the lack of laminin-alpha2 and restore the muscle basement membrane [2-5]. Through simultaneous expression of artificial linkers (SEAL), this gene therapy approach aims to overcome the genetic defect by substituting laminin-alpha2 deficiency with small linker proteins containing necessary binding domains to re-establish muscle fiber integrity. In a transgenic mouse model, the linker expression increased the lifespan of LAMA2-deficient mice 5-fold to a median of 81 weeks compared to 15.5 weeks in the disease model without the therapeutic linker expression [2]. Recently, it was demonstrated that such linker constructs could be applied by standard adeno-associated virus (AAV) vectors [6, 7]. First results using the AAV technology have been presented by Prof Regg [8].

References

[1] Santhera press release on gene collaboration with Biozentrum Basel (May 21, 2019), accessible here

[2] Reinhard et al. (2017). Sci Transl Med 9, eaal4649

[3] Moll et al. (2001). Nature 413, 302-307.

[4] Meinen et al. (2007) J. Cell Biol. 176, 979-993.

[5] McKee et al. (2017) J. Clin. Invest. 127, 1075-1089.

[6] Qiao et al. (2018) Mol Ther Methods Clin Dev 9, 47-56.

[7] Qiao et al. (2005) Proc. Natl. Acad. Sci. U. S. A. 102, 11999-12004.

[8] Reinhard, J. et al. (2019) Neuromuscular Disorders, Volume 29, S164

About Rutgers, The State University of New Jersey

Rutgers, The State University of New Jersey, is a leading national research university and the state of New Jerseys preeminent, comprehensive public institution of higher education. Established in 1766, the university is the eighth-oldest higher education institution in the United States. More than 71,000 students and 23,000 faculty and staff learn, work and serve the public at Rutgers University-New Brunswick, Rutgers University-Newark, Rutgers University-Camden, and Rutgers Biomedical and Health Sciences.

About Santhera

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical need. Santhera is building a Duchenne muscular dystrophy (DMD) product portfolio to treat patients irrespective of causative mutations, disease stage or age. A marketing authorization application for Puldysa (idebenone) is currently under review by the European Medicines Agency. Santhera has an option to license vamorolone, a first-in-class anti-inflammatory drug candidate with novel mode of action, currently investigated in a pivotal study in patients with DMD to replace standard corticosteroids. The clinical stage pipeline also includes lonodelestat (POL6014) to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases, as well as omigapil and an exploratory gene therapy approach targeting congenital muscular dystrophies. Santhera out-licensed ex-North American rights to its first approved product, Raxone (idebenone), for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group. For further information, please visit http://www.santhera.com.

SOURCE: Santhera Pharmaceuticals

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Santhera Signs Agreements in Gene Therapy Research for Congenital Muscular Dystrophy with Rutgers University | More News | News Channels -...

WORCESTER, Mass., May 12, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced two poster presentations at the virtual 23rd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), being held May 12-15, 2020.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are extremely pleased with the strides forward that our researchers have made in gaining greater insights into our innovative CS1 chimeric antigen receptor (CAR) T cell therapy (MB-104), which we previously licensed from City of Hope. We commend them on their poster presentations at ASGCT and look forward to learning more as they continue their research to optimize our clinical trials.

Details on the poster presentations are as follows:

Title: CS1 Targeted CAR-T Cells (MB-104) for the Treatment of Multiple Myeloma Shows Antitumor Activity Sparing Normal T-Cells Despite the Common Expression of CS1Session: Cell TherapiesAbstract number: 421Date and Time: Tuesday, May 12, 2020, 5:30 PM-6:30 PM ETRoom: Exhibit Hall C & DAuthors: Nathan Gumlaw, Aviva Joseph, James Edinger, Ekta Patel, Research and Translational Sciences, Mustang Bio, Worcester, MA

This poster describes researchers investigation into the impact of MB-104 on CS1 positive and negative cells in vitro, as well as T cells due to shared CS1 antigen expansion. The researchers demonstrated MB-104 does not confer biologically significant fratricide and can be successfully manufactured as evident by viability, growth kinetics and fold expansion, despite the shared antigen expression between tumor cells and T cells. CS1 positive T cells are present in culture during the expansion of MB-104, suggesting absence of fratricide. Finally, MB-104 can induce potent anti-tumor cell lysis and proliferates in response to tumor cells but not primary T cells expressing CS1. Taken together, their results demonstrate MB-104 is a novel CS1-targeting CAR T that shows potent anti-tumor cell lysis but spares normal T cells, despite the shared CS1 antigen expression.

Title: Development of an Immunohistochemistry Assay for the Detection of CS-1 Expression in Multiple Myeloma PatientsSession: Pharmacology/Toxicology Studies or Assay DevelopmentAbstract number: 897Date and Time: Wednesday, May 13, 2020, 5:30 PM-6:30 PM ETRoom: Exhibit Hall C & DAuthors: Bethany Biron Girard, James Edinger, Ekta Patel, Translational Sciences, Mustang Bio, Worcester, MA

This poster details a study in which researchers evaluated commercially available CS1 antibodies for IHC and identified the best clone with high specificity for CS1 to improve screening subjects for CS1 positive tumor expression prior to treatment and correlate efficacy with antigen expression. The researchers, for the first time, developed and optimized a robust immunohistochemistry assay for the assessment of CS1 expression in bone marrow core biopsy samples and plasmacytoma solid tumor samples from multiple myeloma (MM) patients, which can be used for enrollment into Mustangs CS1 CAR T clinical trials.

For more information, including abstracts, please visit the ASGCT meeting website at https://annualmeeting.asgct.org/am20/.

About Mustang BioMustang Bio, Inc. (Mustang) is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking Statements

This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

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Mustang Bio Announces Presentations at 23rd Annual Meeting of the American Society of Gene & Cell Therapy - GlobeNewswire

The latest report pertaining to The Future of Gene Therapy Market collated by Market Study Report, LLC, provides a detailed analysis regarding market size, revenue estimations and growth rate of the industry. In addition, the report illustrates the major obstacles and newest growth strategies adopted by leading manufacturers who are a part of the competitive landscape of this market.

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According to a new report, the global gene therapy market is anticipated to reach USD 4,300 million by 2021. The demand for gene therapy is primarily driven by continuous technological advancements and successful progression of several clinical trials targeting treatments with strong unmet need. Moreover, rising R&D spend on platform technologies by large and emerging biopharmaceutical companies and favorable regulatory environment will accelerate the clinical development and the commercial approval of gene therapies in the foreseeable future. Despite promise, the high cost of gene therapy represents a significant challenge for commercial adoption in the forecast period.

Gene therapy offers promise in the treatment of range of indications in cancer and genetic disorders. Large Pharmaceuticals and Biotechnology companies exhibit strong interest in this field and key among them include Allergan, Shire, Biomarin, Pfizer and GSK. The gene therapy space is witnessing a wave of partnerships and alliances. Pfizer has recently expanded its presence in gene therapy with the acquisition of Bamboo Therapeutics and Allergan entered the field, with the acquisition of RetroSense and its Phase I/II optogenetic program.

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North America holds a dominating position in the global gene therapy market which is followed by Europe and the Asia Pacific. The U.S. has maximum number of clinical trials ongoing followed by Europe. Moreover, the field of gene therapy in the U.S. and Europe continues to gain investor attention driven by success of high visible clinical programs and the potential of gene therapy to address strong unmet need with meaningful commercial opportunity. Moreover, the increasing partnerships and alliances and the disruptive potential of gene therapy bodes well for the sector through the forecast period.

Gene therapy involves inactivating a mutated gene that is not functioning properly and introducing a new gene to assist in fighting a disease. Overall, the field of gene therapy continues to mature and advance with many products in development and nearing commercialization. For instance, Spark Therapeutics received approval of Luxturna, a rare form inherited blindness in December 2017. Gene therapy market in late 2017 also witnessed the approvals of Gilead/Kite Pharmas Yescarta and Novartis Kymriah in the cancer therapeutic area.

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Key Findings from the study suggest products accessible in the market are much competitive and manufacturers are progressively concentrating on advancements to pick up an aggressive edge. Companies are in a stage of development of new items in order to guarantee simple implementation and connection with the current gene. The hospatility segment is anticipated to grow at a high growth rate over the forecast period with the expanding utilization of smart locks inferable from expanding security-related worries among clients amid their stay at the hotels. North America is presumed to dominate the global smart locks market over the forecast years and Asia Pacific region shows signs of high growth owing to the booming economies of India, and China.

The Future of Gene Therapy Market share byMajor regions included:

United StatesNorth AmericaAsia PacificEuropeMiddle East & Africa

Table of Contents

1.Gene Therapy Overview1.1.History and Evolution of Gene Therapies1.2.What is Gene Therapy1.3.Types of Gene Therapy1.4.Ex vivo and in vivo Approaches of Gene Therapy1.5.RNAi Therapeutics1.6.CAR-T Technology based Gene Therapy1.7.Types of Vectors used for Gene Therapy1.7.1.Viral1.7.2.Non-Viral2.Historical Marketed Gene Therapies [2003-2012]2.1.Rexin-G (Epeius Biotechnologies Corporation)2.2.Gendicine (SiBiono GeneTech Co., Ltd)2.3.Neovasculgen [Human Stem Cells Institute (HSCI))2.4.Glybera (UniQure Biopharma B.V.)3.First Countries to get an access to Gene Therapies3.1.Philippines for Rexin-G [2003]3.2.China for Gendicine [2003]3.3.Russia for Neovasculgen [2011]3.4.Selected European Countries for Glybera [2012]4.Marketed Gene Therapies [Approved in Recent Years]4.1.KYMRIAH (tisagenlecleucel)4.1.1.Therapy Description4.1.2.Therapy Profile4.1.2.1.Company4.1.2.2.Approval Date4.1.2.3.Mechanism of Action4.1.2.4.Researched Indication4.1.2.5.Vector Used4.1.2.6.Vector Type4.1.2.7.Technology4.1.2.8.Others Development Activities4.1.3.KYMRIAH Revenue Forecasted till 20214.2.YESCARTA (axicabtagene ciloleucel)4.2.1.Therapy Description4.2.2.Therapy Profile4.2.2.1.Company4.2.2.2.Approval Date4.2.2.3.Mechanism of Action

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Global and Regional The Future of Gene Therapy Market Research 2018 Report | Growth Forecast 2026 - Jewish Life News

MEMPHIS, Tenn. and BOTHELL, Wash., May 12, 2020 /PRNewswire/ -- Key Biologics and Astarte Biologics, together the leading provider of research- and clinical-grade human immune cells, blood products, and related services to the biopharmaceutical industry, today announced its rebranding to Cellero. The new Cellero brand better reflects the full capabilities of the organization, which serves customers across the entire cell and gene therapy lifecyclefrom concept to cure. In 2018, Key Biologics and Astarte Biologics merged to establish a comprehensive product and service offering that provides researchers critical access to biomaterial products, and the new Cellero brand represents the synergy and broad capabilities of the combined organizations.

"We are very excited to announce the launch of Cellero and the comprehensive, end-to-end product and service line we offer to our customers," said Jeffrey Allen, CEO. "Regardless of where organizations are in the continuum of discovery through cure, they can trust Cellero to recruit common and hard-to-find blood donors, isolate and characterize specific immune cells, deliver high-volume pure blood products, execute early-stage contract research and discovery projects, and collect from patients for autologous and allogeneic therapies."

In conjunction with the launch of the new brand, Cellero has also announced the grand opening of its new, state-of-the-art cell collection and CLIA-laboratory facility in Lowell, MA. The new facility, combined with the doubling of the company's capacity for collections at its Memphis site in late 2019, represent the completion of Phase One of Cellero's multi-year $50 million plan to invest in new facilities and capabilities to meet the growing demand for human cells for biopharmaceutical R&D and clinical development.

Allen continued, "Our mission is to fuel and accelerate advancements in the discovery, development, and administration of new treatments and cures. Our new facility in Lowell represents one of several steps to execute on this vision and meet the growing demand of our clinical and R&D customers for high-quality cell-based products. This new facility supports our commitment to sourcing and delivering a full range of fresh and frozen GMP-grade biomaterials to some of the most cutting-edge biopharmaceutical companies in Massachusetts, Europe, and elsewhere around the world. Even more exciting is that our new location will also provide apheresis collections for patients in the greater New England area, establishing Cellero as a critical player in the local community supporting patients receiving innovative, life-changing cell therapies."

The new 5,000 square foot facility in Lowell boasts state of the art collection stations utilizing TerumoBCT Spectra Optias for optimal blood collection and superior donor/patient safety and comfort. In addition to the collection suites, a CLIA-licensed laboratory is situated onsite to ensure the highest quality, most efficient operations for Cellero's donors, patients, and customers.

With locations in Seattle, Memphis, and Lowell, Cellero is able to quickly and reliably supply fresh and frozen leukapheresis products to customers across North America, Europe, and Asia. In addition to research and clinical leukapheresis products, the company offers cell characterization and processing services to support cell therapy manufacturers as well as cell-based research tools and services for drug discovery.

Get to know Cellero!

ABOUT CELLEROCellero is the most comprehensive end-to-end provider of donor and patient collection services, biomaterials, characterized immune cells, and custom research and clinical laboratory services for companies developing new drugs and therapies.

Cellero leverages immunology research expertise and coast-to-coast collection facilities and distribution centers to service academic and biopharmaceutical researchers around the world. Visit http://www.cellero.comto learn how Cellero can be your partner in discovery and development.

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Key Biologics and Astarte Biologics Rebrand as Cellero and Announce Completion of Phase One of $50 Million Multi-Year Expansion Plan - NBC Right Now