Monthly Archives: October 2022

Massachusetts-based bluebird bio (BLUE Quick QuoteBLUE - Free Report) is a biotechnology company focused on developing gene therapies for severe genetic diseases and cancer.

bluebirds performance has been impressive after the company reported several positive news over the past two months.

In September, the company received an accelerated FDA approval for Syskona (elivaldogene autotemcel), also known as eli-cel, as a treatment for patients younger than 18 years with early, active cerebral adrenoleukodystrophy (CALD).

The company also announced that the clinical hold previously imposed by the FDA on the eli-cel development program in August 2021 has now been lifted on Sep 15, 2022, before the review of the Syskona biologics license application (BLA).

However, we remind investors that the continued approval of Syskona for CALD may depend upon verification and description of clinical benefit in a confirmatory study.

Thus, as a condition of Syskonas accelerated approval, bluebird will have to provide confirmatory long-term clinical data to the FDA from the ongoing long-term follow-up study (LTF-304), which follows patients treated in clinical trials for 15 years and from commercially treated patients.

With the accelerated approval in place, the company expects to launch Syskona commercially in the United States by the end of 2022 to enable patient access to gene therapy as soon as possible.

Syskona is the second ex-vivo lentiviral vector gene therapy to be approved by the FDA in the United States.

The first is bluebirds Zynteglo (betibeglogene autotemcel), also known as beti-cel, which the FDA approved in August as the first cell-based gene therapy. The therapy treats beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions across all genotypes.

The FDA approvals boost bluebird bios growth prospects, but the successful commercialization of these therapies holds the key. Moreover, gene therapies are complex by nature.

bluebird will face stiff competition in the target market. CRISPR Therapeutics (CRSP Quick QuoteCRSP - Free Report) and its partner Vertex Pharmaceuticals (VRTX Quick QuoteVRTX - Free Report) announced that the FDA granted a rolling review to the gene therapy exagamglogene autotemcel (exa-cel), formerly known as CTX001. The companies are developing the therapy for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Vertex intends to submit its BLA for exa-cell for a rolling review by the FDA by the start of November and expects to complete the process by the end of first-quarter 2023.

These approvals have provided bluebird two priority review vouchers, which are transferable. These vouchers can help shorten the review process of a new drug application (NDA) from 10 months to six months. With the intent of exploring additional financing opportunities, bluebird intends to monetize these vouchers in the near term.

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bluebird's (BLUE) Gene Therapy Approvals to Drive the Top Line - Zacks Investment Research

Acquisition of select PACT assets enables AmplifyBio to expand beyond safety, efficacy, and toxicology services to enable "living medicine" developers to shorten timelines and mitigate risk when moving to the clinic

WEST JEFFERSON, Ohio and SOUTH SAN FRANCISCO, Calif., Oct. 3, 2022 /PRNewswire/ --AmplifyBio, a contract research organization (CRO) focused on accelerating innovation across pharmaceutical modalities; today announced the acquisition of select assets fromPACT Pharma, Inc., a privately held biopharmaceutical company developing neoantigen-specific T cell receptor cell therapies. The deal will provide AmplifyBio with advanced characterization platforms, bioinformatics capabilities, and 40 drug development experts to enhance their cell and gene therapy service offerings. AmplifyBio will also acquire the South San Francisco advanced laboratory space.

With the acquisition of these assets, AmplifyBio aims to provide an early, consistent characterization of a treatment's purity, potency, and viability throughout the life cycle of therapeutic development. Unlike small molecules, there is no single, consistent process for cell and gene therapy companies to research, develop, and test their therapeutics. The gap that exists in characterization between the discovery phase and preclinical testing leads to material changes in a therapeutic during development, which can in turn create manufacturing inconsistencies and safety concerns during scale-up.

"Many biologics developers have adopted the phrase 'the process is the product' to describe how their therapeutic is differentiated based on a unique development process," said AmplifyBio Chief Executive Officer (CEO) and President J. Kelly Ganjei. "Rather than create our own, individual technique, AmplifyBio aims to replace that saying with a new one: 'the product is the product. Our acquisition of these assets from PACT Pharma means that cell and gene therapies can now be differentiated based on safety and efficacy profiles and specific product characteristics, not development processes."

"This deal allows PACT to retain its core intellectual property and continue our mission of developing novel, neoantigen-targeted T-Cell Therapies," added Scott Garland, PACT Pharma's CEO. "At the same time, we're working with AmplifyBio to leverage our platforms to offer a unique combination of optimization, characterization, safety and efficacy services to a wider range of clients seeking to better understand the immunology of their adoptive cell therapies."

AmplifyBio was spun out in 2021 from Battelle, a not-for-profit organization that advances science and technology to have the greatest impact on our society and economy. Following today's acquisition of the South San Francisco facility, AmplifyBio plans to add a third site in New Albany, Ohio that consists of 350,000 square feet of multi-function lab spaces. There, AmplifyBio will build on its advanced therapy services by adding capabilities for complex genotypic and phenotypic characterization analysis for late-stage development. The company expects to add additional development platforms and partnerships to become a commercial accelerator delivering safe, effective, reproducible advanced therapies to patients.

About AmplifyBioAmplifyBio is a leading preclinical CRO focused on toxicology, safety, and pharmacology testing to advance therapeutics for the betterment of human health. Spun out of Battelle in May of 2021, AmplifyBio's mission is to continue to provide exceptional CRO study services in an agile environment better suited to commercial goals and expand analytic capabilities to serve the dynamic needs of advanced therapy development. Clients of AmplifyBio enjoy the peace of mind that comes from decades of experience in GLP and non-GLP study design and execution, combined with rapid investment in technology, expertise, and infrastructure that together provide the critical components of a reliable, agile partnership.

Media Contact AmplifyBio[emailprotected]For Inquiries to PACT[emailprotected]

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AMPLIFYBIO ACQUIRES PACT PHARMA ASSETS TO ENHANCE CELL AND GENE THERAPY CHARACTERIZATION CAPABILITIES - PR Newswire

Theres a handful of reasons why the Philadelphia region has been (perhaps unfortunately) dubbed Cellicon Valley in the last few years, and a new report from the Chamber of Commerce for Greater Philadelphia and economic consulting firm Econsult Solutions has IDd them all.

In the report, which looked at 14 large cell and gene therapy hubs in the US, Philadelphia ranked as the runner up, just behind Boston, as the top spot for research and innovation in this space. Other metro areas such as New York and San Francisco scored the third and fourth spots on the list. The report shouts out early local work, including the first FDA-approved gene (Luxturna) and cell (Kymriah) therapies developed here at Spark Therapeutics and the University of Pennsylvania, respectively.

The Philadelphia region is increasingly attracting new and expanding cell and gene therapy companies because it checks all the boxes, but its the regions research infrastructure as defined by NIH-funded cell and gene therapy research and its large number of research institutions that give it the edge, said Claire Marrazzo Greenwood, executive director and CEO of Council for Growth and SVP of economic competitiveness for the Chamber, in a statement.

The study compared cell and gene therapy hubs for their research infrastructure, human capital, innovation output, commercial activity and value proposition. Heres why Philly ranked high:

Because Philly is home to four Tier 1 universities, 93 higher ed institutions, and tons of hospitals and research institutions, it scored second in research infrastructure. The region scored first for most National Health Institute funding, and the report said 302 gene or cell therapy patents had been approved in the last decade. In 2021, the region was home to 15,400 jobs in pharmaceutical manufacturing, and it pulled in $4.2 billion in venture capital funding since 2018.

The talent coming from the high number of universities and colleges and more than 450,000 students in the region also ranked the region high for human capital. Of this, a whopping 54% stay in the region. R&D jobs in the field have also increased more than 100% in the last five years.

Philadelphia also scored high for its innovation output, meaning the region produces a large amount of intellectual property in the cell and gene therapy space. As the birthplace of the industry, the report says, the region is currently home to 302 granted patent and 130 clinical trials now underway.

The large amount of attention cell and gene therapy has gotten from investors in the last four years also ranked the region high in commercial activity. Within the past few years, two local cell and gene therapy companies Passage Bio and Cabaletta Bio have also completed IPOs, raising more than $260 million combined. Cell and gene therapy companies also make up a significant portion of Phillys commercial real estate, leasing about 12 million square feet, with about 9 million planned in construction projects.

And Philadelphias value proposition, or cost to do business, helped the region rank so highly, the report said. The region attracts families and talent with cultural institutions, culinary scene and schools. Plus, life sciences office space rentals (averaging about $58 per square foot) were very affordable next to cities like San Francisco (at $78 per square foot).

Greater Philadelphia is an extremely livable region, boasting some of the worlds best museums, top-notch restaurants, and large open spaces at a comparatively affordable price, Econsult said in its summary.

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Why Philly ranks #2 among best cell and gene therapy hubs in the US - Technical.ly

CAMBRIDGE, Mass., Oct. 03, 2022 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc.(Nasdaq: VYGR), a gene therapy and neuroscience company developing life-changing treatments and next-generation adeno-associated virus (AAV) capsids, today announced that it will present three posters at the upcoming 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), taking place October 11-14, 2022, in Edinburgh, Scotland.

Poster Presentation Details:

Presentation Title: Identification of a Cell Surface Receptor Utilized by an Engineered BBB-Penetrant Capsid Family withEnhanced Brain Tropism in Non-Human Primates and MicePoster Number: P024Presenting Author: Brett Hoffman, Ph.D., Senior Scientist, Capsid Discovery

Presentation Title: Dose-Response Evaluation of 9P801, an Engineered AAV Capsid with High BBB Penetration and CNS Transduction in Non-Human PrimatesPoster Number: P015Presenting Author: Mathieu Nonnenmacher, Ph.D., Vice President, Capsid Discovery

Presentation Title: Evaluation of an Early, Late, Very Late Expressed Rep in a Recombinant Baculovirus to Produce a More Potent AAV-based Gene Therapeutic in Insect CellsPoster Number: P065Presenting Author: Jeffrey Slack, Ph.D., Principal Scientist, Cell Culture Development

AboutVoyager TherapeuticsVoyager Therapeutics(Nasdaq: VYGR) is leading the next generation of AAV gene therapy to unlock the potential of the modality to treat devastating diseases. Proprietary capsids born from the Companys TRACER discovery platform are powering a rich early-stage pipeline of programs and may elevate the field to overcome the narrow therapeutic window associated with conventional gene therapy vectors across neurologic disorders and other therapeutic areas. voyagertherapeutics.com LinkedIn Twitter

Voyager Therapeutics is a registered trademark, and TRACER is a trademark, ofVoyager Therapeutics, Inc.

ContactsInvestorsInvestors@vygr.com

MediaPeg Rusconiprusconi@vergescientific.com

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Voyager Therapeutics Announces Data Presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy - GlobeNewswire

Researchers at the National Eye Institute have designed a gene therapy approach that could help prevent blindness in children with Leber congenital amaurosis, a rare form of blindness.

A discovery by the National Eye Institute (NEI), part of the National Institutes of Health, could lead to a second gene therapy for a rare form of blindness. The researchers discovered that a type of Leber congenital amaurosis (LCA) is caused by mutations in the NPHP5 (also called IQCB1) gene and leads to severe defects in the primary cilium, a structure found in nearly all cells of the body. Primary cilia play a role in cell cycle regulation. In the eye, cilia play important roles in maintaining normal eye function.

Leber congenital amaurosis is an eye disorder that affects the tissue at the back of the eye that detects light and color. It is also associated with sensitivity to light, involuntary movements of the eye, and extreme farsightedness. Leber congenital amaurosis affects 2 to 3 per 100,000 newborns and is one of the most common causes of blindness in children.

There are at least 13 types of Leber congenital amaurosis, according to the National Library of Medicine. The types are distinguished by their genetic cause, patterns of vision loss, and related eye abnormalities. One gene therapy has already been approved to treat a degenerative eye disease. It is available for blindness associated with a mutation of RPE65, which provides the instructions for making a protein important for normal vision. In 2017, the FDA approved Spark Therapeutics Luxturna (voretigene neparvovec-rzyl), the first one-time gene therapy for patients with RPE65 mutation-associated retinal dystrophy and viable retinal cells.

The type of Leber congenital amaurosis caused by mutations in NPHP5 is relatively rare. In a healthy eye, NPHP5 protein is believed to help filter proteins that enter the cilium. Previous studies in mice have shown that NPHP5 is involved in the cilium, but researchers didnt know the exact role of NPHP5.

NPHP5 deficiency causes early blindness in its milder form, and in more severe forms, many patients also exhibit kidney disease along with retinal degeneration, the studys lead investigator, Anand Swaroop, Ph.D., senior investigator at the NEI Neurobiology Neurodegeneration and Repair Laboratory, said in a press release. Weve designed a gene therapy approach that could help prevent blindness in children with this disease and one that, with additional research, could perhaps even help treat other effects of the disease.

Three post-doctoral fellows, Kamil Kruczek, Ph.D., Zepeng Qu, Ph.D., and Emily Welby, Ph.D., at the National Eye Institute collected stem cell samples from two patients with NPHP5 deficiency at the NIH Clinical Center. These stem cell samples were used to generate retinal organoids, cultured tissue clusters that possess many of the structural and functional features of actual, native retina.

The found reduced levels of NPHP5 protein within the patient-derived retinal organoid cells, as well as reduced levels of another protein called CEP-290, which interacts with NPHP5 and forms the primary cilium gate. They also found that photoreceptor outer segments in the retinal organoids were missing and the opsin protein a light sensitive protein that should have been localized to the outer segments was instead found elsewhere in the photoreceptor cell body.

Researchers introduced an adeno-associated viral (AAV) vector a virus that is used mechanism to deliver the gene containing a functional version of NPHP5. The retinal organoids showed a restoration of opsin protein concentrated in the proper location in outer segments. The findings also suggest that functional NPHP5 may have stabilized the primary cilium gate.

The study was funded by the NEI Intramural program.

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Researchers Develop Potential Gene Therapy to Treat Blindness - Managed Healthcare Executive

New York, Sept. 29, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market by Scale of Operation, Type of Vector, Application Area, Therapeutic Area, and Geographical Regions : Industry Trends and Global Forecasts, 2022-2035" - https://www.reportlinker.com/p06323417/?utm_source=GNW In fact, in 2021, cell and gene therapy developers raised capital worth more than USD 20 billion, registering an increase of 19% from the amount raised in 2020 (~USD 17 billion). It is worth highlighting that, in February 2022, the USFDA approved second CAR-T therapy, CARVYKTI, developed by Johnson and Johnson, which can be used for the treatment of relapsed or refractory multiple myeloma. Additionally, close to 1,500 clinical trials are being conducted, globally, for the evaluation of cell and gene therapies. Over time, it has been observed that the clinical success of these therapies relies on the design and type of gene delivery vector used (in therapy development and / or administration). At present, several innovator companies are actively engaged in the development / production of viral vectors and / or non-viral vectors for cell and gene therapies. In this context, it is worth mentioning that, over the past few years, multiple viral vector and non-viral vector based vaccine candidates have been developed against COVID-19 (caused by novel coronavirus, SARS-CoV-2) and oncological disorders; this is indicative of lucrative opportunities for companies that have the required capabilities to manufacture vectors and gene therapies.

The viral and non-viral vector manufacturing landscape features a mix of industry players (well-established companies, mid-sized firms and start-ups / small companies), as well as several academic institutes. It is worth highlighting that several companies that have the required capabilities and facilities to manufacturing vectors for both in-house requirements and offer contract services (primarily to ensure the optimum use of their resources and open up additional revenue generation opportunities) have emerged in this domain. Further, in order to produce more effective and affordable vectors, several stakeholders are integrating various novel technologies; these technologies are likely to improve the scalability and quality of the resultant therapy. In addition, this industry has also witnessed a significant increase in the partnership and expansion activities over the past few years, with several companies having been acquired by the larger firms. Given the growing demand for interventions that require genetic modification, the vector and gene therapy manufacturing market is poised to witness substantial growth in the foreseen future.

SCOPE OF THE REPORTThe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market (5th Edition) by Scale of Operation (Preclinical, Clinical and Commercial), Type of Vector (AAV Vector, Adenoviral Vector, Lentiviral Vector, Retroviral Vector, Plasmid DNA and Others), Application Area (Gene Therapy, Cell Therapy and Vaccine), Therapeutic Area (Oncological Disorders, Rare Disorders, Neurological Disorders, Sensory Disorders, Metabolic Disorders, Musco-skeletal Disorders, Blood Disorders, Immunological Diseases, and Others), and Geographical Regions (North America, Europe, Asia Pacific, MENA, Latin America and Rest of the World): Industry Trends and Global Forecasts, 2022-2035 report features an extensive study of the rapidly growing market of vector and gene therapy manufacturing, focusing on contract manufacturers, as well as companies having in-house manufacturing facilities. The study presents an in-depth analysis of the various firms / organizations that are engaged in this domain, across different regions of the globe. Amongst other elements, the report includes:An overview of the current status of the market with respect to the players engaged (both industry and non-industry) in the manufacturing of viral, non-viral and other novel types of vectors and gene therapies. It features information on the year of establishment, company size, location of headquarters, type of product manufactured (vector and gene therapy / cell therapy / vaccine), location of manufacturing facilities, type of manufacturers (in-house and contract services), scale of operation (preclinical, clinical and commercial), type of vector manufactured (AAV, adenoviral, lentiviral, retroviral, plasmid DNA and others) and application area (gene therapy, cell therapy, vaccine and others).An analysis of the technologies offered / developed by the companies enagaged in this domain, based on the type of technology (viral vector related platform, non-viral vector related platform and others), type of manufacturer (vector manufacturing, gene delivery, product manufacturing, transduction / transfection, vector packaging and other), scale of operation (preclinical, clinical and commercial), type of vector involved (AAV, adenoviral, lentiviral, retroviral, non-viral and other viral vectors), application area (gene therapy, cell therapy, vcaccine and others). It also highlights the most prominent players within this domain, in terms of number of technologies.A region-wise, company competitiveness analysis, highlighting key players engaged in the manufacturing of vectors and gene therapies, across key geographical areas, featuring a four-dimensional bubble representation, taking into consideration supplier strength (based on experience in this field), manufacturing strength (type of product manufactured, number of manufacturing facilites and number of application areas), service strength (scale of operation, number of vectors manufactured and geographical reach) and company size (small, mid-sized and large).Elaborate profiles of key players based in North America, Europe and Asia-Pacific (shortlisted based on proprietary criterion). Each profile features an overview of the company / organization, its financial performance (if available), information related to its manufacturing facilities, vector manufacturing technology and an informed future outlook.Tabulated profiles of the other key players headquartered in different regions across the globe (shortlisted based on proprietary criterion). Each profile features an overview of the company, its financial performance (if available), information related to its manufacturing capabilities, and an informed future outlook.An analysis of partnerships and collaborations established in this domain since 2015; it includes details of deals that were / are focused on the manufacturing of vectors, which were analyzed on the basis of year of partnership, type of partnership (manufacturing agreement, product / technology licensing, product development, merger / acqusition, research and development agreement, process development / optimization, service alliance, production asset / facility acquisition, supply agreement and others), scale of operation (preclinical, clinical and commercial), type of vector involved (AAV, adenoviral, lentiviral, retroviral, plasmid and others), region and most active players (in terms of number of partnerships).An analysis of the expansions related to viral vector and non-viral vector manufacturing, which have been undertaken since 2015, based on several parameters, such as year of expansion, type of expansion (new facility / plant establishment, facility expansion, technology installation / expansion, capacity expansion, service expansion and others), type of vector (AAV, adenoviral, lentiviral, retroviral, plasmid and others), application area (gene therapy, cell therapy, vaccine and others) and geographical location of the expansion.An analysis evaluating the potential strategic partners (comparing vector based therapy developers and vector purification product developers) for vector and gene therapy product manufacturers, based on several parameters, such as developer strength, product strength, type of vector, therapeutic area, pipeline strength (preclinical and clinical).An overview of other viral / non-viral gene delivery approaches that are currently being researched for the development of therapies involving genetic modification.An in-depth analysis of viral vector and plasmid DNA manufacturers, featuring three schematic representations, a three dimensional grid analysis, representing the distribution of vector manufacturers (on the basis of type of vector) across various scales of operation and type of manufacturer (in-house operations and contract manufacturing services), a heat map of viral vector and plasmid DNA manufacturers based on the type of vector (AAV, adenoviral vector, lentiviral vector, retroviral vector and plasmid DNA) and type of organization (industry (small, mid-sized and large) and non-industry), and a schematic world map representation, highlighting the headquarters and geographical location of key vector manufacturing hubs.An analysis of the various factors that are likely to influence the pricing of vectors, featuring different models / approaches that may be adopted by product developers / manufacturers in order to decide the prices of proprietary vectors.An estimate of the overall, installed vector manufacturing capacity of industry players based on the information available in the public domain, and insights generated via both secondary and primary research. The analysis also highlights the distribution of the global capacity by company size (small, mid-sized and large), scale of operation (clinical and commercial), type of vector (viral vector and plasmid DNA) and region (North America, Europe, Asia Pacific and the rest of the world).An informed estimate of the annual demand for viral and non-viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.A discussion on the factors driving the market and various challenges associated with the vector production process.A qualitative analysis, highlighting the five competitive forces prevalent in this domain, including threats for new entrants, bargaining power of drug developers, bargaining power of vector and gene therapy manufacturers, threats of substitute technologies and rivalry among existing competitors.

One of the key objectives of this report was to evaluate the current market size and the future opportunity associated with the vector and gene therapy manufacturing market, over the coming decade. Based on various parameters, such as the likely increase in number of clinical studies, anticipated growth in target patient population, existing price variations across different types of vectors, and the anticipated success of gene therapy products (considering both approved and late-stage clinical candidates), we have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2022-2035. In order to provide a detailed future outlook, our projections have been segmented on the basis of scale of operation (preclinical, clinical and commercial), type of vector (AAV vector, adenoviral vector, lentiviral vector, retroviral vector, plasmid DNA and others), application area (gene therapy, cell therapy and vaccine), therapeutic area (oncological disorders, rare disorders, neurological disorders, sensory disorders, metabolic disorders, musco-skeletal disorders, blood disorders, immunological diseases, and others) and geographical region (North America, Europe, Asia Pacific, MENA, Latin America and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The research, analysis and insights presented in this report are backed by a deep understanding of key insights generated from both secondary and primary research. For the purpose of the study, we invited over 300 stakeholders to participate in a survey to solicit their opinions on upcoming opportunities and challenges that must be considered for a more inclusive growth. The opinions and insights presented in this study were also influenced by discussions held with senior stakeholders in the industry. The report features detailed transcripts of interviews held with the following industry and non-industry players:Menzo Havenga (Chief Executive Officer and President, Batavia Biosciences)Nicole Faust (Chief Executive Officer & Chief Scientific Officer, CEVEC Pharmaceuticals)Cedric Szpirer (Former Executive & Scientific Director, Delphi Genetics)Olivier Boisteau, (Co-Founder / President, Clean Cells), Laurent Ciavatti (Former Business Development Manager, Clean Cells) and Xavier Leclerc (Head of Gene Therapy, Clean Cells)Alain Lamproye (Former President of Biopharma Business Unit, Novasep)Joost van den Berg (Former Director, Amsterdam BioTherapeutics Unit)Bakhos A Tannous (Director, MGH Viral Vector Development Facility, Massachusetts General Hospital)Eduard Ayuso, DVM, PhD (Scientific Director, Translational Vector Core, University of Nantes)Colin Lee Novick (Managing Director, CJ Partners)Semyon Rubinchik (Scientific Director, ACGT)Astrid Brammer (Senior Manager Business Development, Richter-Helm)Marco Schmeer (Project Manager, Plasmid Factory) and Tatjana Buchholz (Former Marketing Manager, Plasmid Factory)Brain M Dattilo (Business Development Manager, Waisman Biomanufacturing)Beatrice Araud (ATMP Key Account Manager, EFS-West Biotherapy)Nicolas Grandchamp (R&D Leader, GEG Tech)Graldine Gurin-Peyrou (Director of Marketing and Technical Support, Polypus Transfection)Naiara Tejados, Head of Marketing and Technology Development, VIVEBiotech)Jeffery Hung (Independent Consultant)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGYThe data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market may evolve across different regions and technology segments. Wherever possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include:Annual reportsInvestor presentationsSEC filingsIndustry databasesNews releases from company websitesGovernment policy documentsIndustry analysts views

While the focus has been on forecasting the market over the period 2022-2035, the report also provides our independent view on various technological and non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWEREDWho are the leading players (contract service providers and in-house manufacturers) engaged in the development of vectors and gene therapies?Which regions are the current manufacturing hubs for vectors and gene therapies?Which type of vector related technologies are presently offered / being developed by the stakeholders engaged in this domain?Which companies are likely to partner with viral and non-viral vector contract manufacturing service providers?Which partnership models are commonly adopted by stakeholders engaged in this industry?What type of expansion initiatives are being undertaken by players in this domain?What are the various emerging viral and non-viral vectors used by players for the manufacturing of genetically modified therapies?What are the strengths and threats for the stakeholders engaged in this industry?What is the current, global demand for viral and non-viral vector, and gene therapies?How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES

Chapter 2 is an executive summary of the insights captured in our research. It offers a high-level view on the likely evolution of the vector and gene therapy manufacturing market in the short to mid-term, and long term.

Chapter 3 is a general introduction to the various types of viral and non-viral vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages, limitations and applications of the currently available gene delivery vehicles. The chapter also features the clinical and approved pipeline of genetically modified therapies. Further, it includes a review of the latest trends and innovations in the contemporary vector manufacturing market.

Chapter 4 provides a detailed overview of close to 150 companies, featuring both contract service providers and in-house manufacturers that are actively involved in the production of viral vectors and / or gene therapies utilizing viral vectors. The chapter provides details on the year of establishment, company size, location of headquarters, type of product manufactured (vector and gene therapy / cell therapy / vaccine), location of manufacturing facilities, type of manufacturer (in-house and contract services), scale of operation (preclinical, clinical and commercial), type of vector manufactured (AAV, adenoviral, lentiviral, retroviral, plasmid DNA and others) and application area (gene therapy, cell therapy, vaccine and others).

Chapter 5 provides an overview of close to 70 industry players that are actively involved in the production of plasmid DNA and other non-viral vectors and / or gene therapies utilizing non-viral vectors. The chapter provides details on the the year of establishment, company size, location of headquarters, type of product manufactured (vector and gene therapy / cell therapy / vaccine), location of plasmid DNA manufacturing facilities, type of manufacturer (in-house and contract services), scale of operation (preclinical, clinical and commercial) and application area (gene therapy, cell therapy, vaccine and others).

Chapter 6 provides an overview of close to 90 non-industry players (academia and research institutes) that are actively involved in the production of vectors (both viral and non-viral) and / or gene therapies. The chapter provides details on the year of establishment, type of manufacturer (in-house and contract services), scale of operation (preclinical, clinical and commercial), location of headquarters, type of vector manufactured (AAV, adenoviral, lentiviral, retroviral, plasmid DNA and others) and application area (gene therapy, cell therapy, vaccine and others).

Chapter 7 features an in-depth analysis of the technologies offered / developed by the companies engaged in this domain, based on the type of technology (viral vector and non-viral vector related platform), purpose of technology (vector manufacturing, gene delivery, product manufacturing, transduction / transfection, vector packaging and other), scale of operation (preclinical, clinical and commerical), type of vector involved (AAV, adenoviral, lentiviral, retroviral, non-viral and other viral vectors), application area (gene therapy, cell therapy, vaccine and others) and leading technology providers.

Chapter 8 presents a detailed competitiveness analysis of vector manufacturers across key geographical areas, featuring a four-dimensional bubble representation, taking into consideration supplier strength (based on its experience in this field), manufacturing strength (type of product manufactured, number of manufacturing facilities and number of application area), service strength (scale of operation, number of vectors manufactured and geographical reach) and company size (small, mid-sized and large).

Chapter 9 features detailed profiles of some of the key players that have the capability to manufacture viral vectors / plasmid DNA in North America. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience and an informed future outlook.

Chapter 10 features detailed profiles of some of the key players that have the capability to manufacture viral vectors / plasmid DNA in Europe. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience and an informed future outlook.

Chapter 11 features detailed profiles of some of the key players that have the capability to manufacture viral vectors / plasmid DNA in Asia-Pacific. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience and an informed future outlook.

Chapter 12 features tabulated profiles of the other key players that have the capability to manufacture viral vectors / plasmid DNA. Each profile features an overview of the company, its financial performance (if available), information related to its manufacturing capabilities, and an informed future outlook.

Chapter 13 features in-depth analysis and discussion of the various partnerships inked between the players in this market, during the period, 2015-2022, covering analysis based on parameters such as year of partnership, type of partnership(manufacturing agreement, product / technology licensing, product development, merger / acquisition, research and development agreement, process development / optimization, service alliance, production asset / facility acquisition, supply agreement and others), scale of operation (preclinical, clinical and commercial) and type of vector (AAV, adenoviral, lentiviral, retroviral, plasmid and others) most active players (in terms of number of partnerships).

Chapter 14 features an elaborate discussion and analysis of the various expansions that have been undertaken, since 2015. Further, the expansion activities in this domain have been analyzed on the basis of year of expansion, type of expansion (new facility / plant establishment, facility expansion, technology installation / expansion, capacity expansion, service expansion and others), geographical location of the facility, type of vector (AAV, adenoviral, lentiviral, retroviral, plasmid and others) and application area (gene therapy, cell therapy, vaccine and others).

Chapter 15 highlights potential strategic partners (vector based therapy developers and vector purification product developers) for vector and gene therapy product manufacturers, based on several parameters, such as developer strength, product strength, type of vector, therapeutic area, pipeline strength (clinical and preclinical). The analysis aims to provide the necessary inputs to the product developers, enabling them to make the right decisions to collaborate with industry stakeholders with relatively more initiatives in the domain.

Chapter 16 provides detailed information on other viral / non-viral vectors. These include alphavirus vectors, Bifidobacterium longum vectors, Listeria monocytogenes vectors, myxoma virus based vectors, Sendai virus based vectors, self-complementary vectors (improved versions of AAV), minicircle DNA and Sleeping Beauty transposon vectors (non-viral gene delivery approach) and chimeric vectors, that are currently being utilized by pharmaceutical players to develop gene therapies, T-cell therapies and certain vaccines, as well. This chapter presents overview on all the aforementioned types of vectors, along with examples of companies that use them in their proprietary products. It also includes examples of companies that are utilizing specific technology platforms for the development / manufacturing of some of these novel vectors.

Chapter 17 presents a collection of key insights derived from the study. It includes a grid analysis, highlighting the distribution of viral vectors and plasmid DNA manufacturers on the basis of their scale of operation and type of manufacturer (fulfilling in-house requirement / contract service provider). In addition, it consists of a heat map of viral vector and plasmid DNA manufacturers based on the type of vector (AAV, adenoviral vector, lentiviral vector, retroviral vector and plasmid DNA) and type of organization (industry (small, mid-sized and large) and non-industry). The chapter also consists of six world map representations of manufacturers of viral / non-viral vectors (AAV, adenoviral, lentiviral, retroviral vectors, and plasmid DNA), depicting the most active geographies in terms of the presence of the organizations. Furthermore, we have provided a schematic world map representation to highlight the geographical locations of key vector manufacturing hubs across different continents.

Chapter 18 highlights our views on the various factors that may be taken into consideration while pricing viral vectors / plasmid DNA. It features discussions on different pricing models / approaches that manufacturers may choose to adopt to decide the prices of their proprietary products.Chapter 19 features an informed analysis of the overall installed capacity of the vectors and gene therapy manufacturers. The analysis is based on meticulously collected data (via both secondary and primary research) on reported capacities of various small, mid-sized and large companies, distributed across their respective facilities. The results of this analysis were used to establish an informed opinion on the vector production capabilities of the organizations by company size (small, mid-sized and large), scale of operation (clinical and commercial), type of vector (viral vector and plasmid DNA) and region (North America, Europe, Asia Pacific and the rest of the world).

Chapter 20 features an informed estimate of the annual demand for viral and non-viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies. This section offers an opinion on the required scale of supply (in terms of vector manufacturing services) in this market. For the purpose of estimating the current clinical demand, we considered the active clinical studies of different types of vector-based therapies that have been registered till date. The data was analyzed on the basis of various parameters, such as number of annual clinical doses, trial location, and the enrolled patient population across different geographies. Further, in order to estimate the commercial demand, we considered the marketed vector-based therapies, based on various parameters, such as target patient population, dosing frequency and dose strength.

Chapter 21 presents a comprehensive market forecast analysis, highlighting the likely growth of vector and gene therapy manufacturing market till the year 2030. We have segmented the financial opportunity on the basis of type of vector (AAV vector, adenoviral vector, lentiviral vector, retroviral vector, plasmid DNA and others), application area (gene therapy, cell therapy and vaccine), therapeutic area (oncological disorders, rare disorders, neurological disorders, sensory disorders, metabolic disorders, musco-skeletal disorders, blood disorders, immunological diseases, and others), scale of operation (preclinical, clinical and commercial) and geography (North America, Europe, Asia Pacific, MENA, Latin America and rest of the world). Due to the uncertain nature of the market, we have presented three different growth tracks outlined as the conservative, base and optimistic scenarios.

Chapter 22 highlights the qualitative analysis on the five competitive forces prevalent in this domain, including threats for new entrants, bargaining power of drug developers, bargaining power of vector and gene therapy manufacturers, threats of substitute technologies and rivalry among existing competitors.

Chapter 23 provides details on the various factors associated with popular viral vectors and plasmid DNA that act as market drivers and the various challenges associated with the production process. This information has been validated by soliciting the opinions of several industry stakeholders active in this domain.

Chapter 24 presents insights from the survey conducted on over 300 stakeholders involved in the development of different types of gene therapy vectors. The participants, who were primarily Director / CXO level representatives of their respective companies, helped us develop a deeper understanding on the nature of their services and the associated commercial potential.

Chapter 25 summarizes the entire report, highlighting various facts related to contemporary market trend and the likely evolution of the viral vector, non-viral vector and gene therapy manufacturing market.

Chapter 26 is a collection of transcripts of the interviews conducted with representatives from renowned organizations that are engaged in the vector and gene therapy manufacturing domain. In this study, we spoke to Menzo Havenga (Chief Executive Officer and President, Batavia Biosciences), Nicole Faust (Chief Executive Officer & Chief Scientific Officer, CEVEC Pharmaceuticals), Cedric Szpirer (Former Executive & Scientific Director, Delphi Genetics), Olivier Boisteau, (Co-Founder / President, Clean Cells), Laurent Ciavatti (Former Business Development Manager, Clean Cells) and Xavier Leclerc (Head of Gene Therapy, Clean Cells), Alain Lamproye (Former President of Biopharma Business Unit, Novasep), Joost van den Berg (Former Director, Amsterdam BioTherapeutics Unit), Bakhos A Tannous (Director, MGH Viral Vector Development Facility, Massachusetts General Hospital), Eduard Ayuso, DVM, PhD (Scientific Director, Translational Vector Core, University of Nantes), Colin Lee Novick (Managing Director, CJ Partners), Semyon Rubinchik (Scientific Director, ACGT), Astrid Brammer (Senior Manager Business Development, Richter-Helm), Marco Schmeer (Project Manager, Plasmid Factory) and Tatjana Buchholz (Former Marketing Manager, Plasmid Factory), Brain M Dattilo (Business Development Manager, Waisman Biomanufacturing), Beatrice Araud (ATMP Key Account Manager, EFS-West Biotherapy), Nicolas Grandchamp (R&D Leader, GEG Tech), Graldine Gurin-Peyrou (Director of Marketing and Technical Support, Polypus Transfection), Naiara Tejados, Head of Marketing and Technology Development, VIVEBiotech) and Jeffery Hung (Independent Consultant)

Chapter 27 is an appendix, which provides tabulated data and numbers for all the figures in the report.

Chapter 28 is an appendix that provides the list of companies and organizations that have been mentioned in the report.Read the full report: https://www.reportlinker.com/p06323417/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Excerpt from:
Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market by Scale of Operation, Type of Vector, Application...

FLORHAM PARK, N.J., Oct. 04, 2022 (GLOBE NEWSWIRE) -- Real Endpoints, the leading market-access platform and advisory firm, announced a collaboration with bluebird bio, inc. (Nasdaq: BLUE), to provide multiple health plans with access to an innovative, outcomes-based agreement for ZYNTEGLO (betibeglogene autotemcel) through the Real Endpoints (RE) Marketplace.

These plans cover nearly 16 million individuals across the U.S.; while treatment-dependent beta-thalassemia is a rare disease, together these plans comprise a significant portion of the patient population in the U.S. ZYNTEGLO is currently the only FDA-approved gene therapy for people with beta-thalassemia who require regular red blood cell transfusions.

Through a single contract, the plans in RE Marketplace can take immediate advantage of bluebirds innovative agreement, which offers rebates of up to 80% if treatment with ZYNTEGLO does not enable a patient to achieve and maintain transfusion independence in the two years following therapy.

RE Marketplace performs all the required analytics and financial reconciliation as an expert, independent third-party. RE Marketplace provides participating plans and manufacturers with end-to-end capabilities for efficient, scalable value-based contracting and does so with complete financial and data transparency.

bluebirds ZYNTEGLO is a giant step forward for medicine, commented Jane Barlow, MD, Chief Clinical Officer at Real Endpoints. The plans in RE Marketplace are thrilled to be able to easily access bluebirds innovative risk-sharing agreement, which speeds the delivery of both clinical and economic innovations. That is a win for both patients and the broader health system, she said.

About RE Marketplace

The RE Marketplace platform provides members of mid-sized and smaller health plans speedier access to innovative treatments such as rare disease drugs, cell and gene therapies, and digital medicines. From four founding member plans, RE Marketplace now represents several mid-sized and regional plans approaching nearly 16 million beneficiaries across all lines of business. Both industry and payer participants benefit from the efficiency and flexibility of RE Marketplace, which can support a range of innovative contracts through a standard contracting process. There is also the potential for more generous rebate opportunities without additional Medicaid Best Price risk. RE Marketplace performs all the critical analytics and financial reconciliation transparently and with full audit rights, using a highly robust, secure, HIPAA-compliant system already tested and used in multiple value-based agreements. For more about RE Marketplace, please visit this link: https://realendpoints.com/products/re-marketplace/

About Real Endpoints

Real Endpoints solutions create patient access to meaningful medical innovations and prepare companies for competition in the value-based economy. Working collaboratively with biopharma, diagnostic and medical device companies, RE provides unique answers across a wide range of coverage and reimbursement issues from pricing and distribution to patient support services. RE is also the leading advisor to the industry on innovative contracting, including the evaluation, structuring, negotiating, and third-party management of the analytics and financial reconciliation of value-based contracts. For more information about Real Endpoints, visit http://www.realendpoints.com.

Website: http://www.realendpoints.comLinkedIn: https://www.linkedin.com/company/real-endpoints/

Contact: Aurore Duboille Email: aduboille@realendpoints.comPhone: 973-805-2300

Link:
Real Endpoints Marketplace announces collaboration with bluebird bio to help scale delivery of a first-of-its-kind value-based contract for one-time...

Sanofi will partner with the Californian biotechnology company Scribe Therapeutics in a deal that extends its exploration of new ways to build cancer cell therapies.

Under a partnership announced Tuesday, Sanofi will pay Scribe $25 million upfront to gain access to the five-year-old startups gene editing technology. The pharmaceutical company is also promising more than $1 billion in additional payments based on unspecified development and commercial milestones, although that money may never be paid out.

In return, Sanofi gets non-exclusive rights to use Scribes CRISPR-based gene editing technology to develop cancer treatments constructed from modified natural killer, or NK, cells. A type of immune defender, NK cells have drawn increasing interest from cancer drugmakers looking for alternatives to the T cells used in CAR-T treatments for leukemia, lymphoma and multiple myeloma.

This collaboration with Scribe complements our robust research efforts across the NK cell therapy spectrum and offers our scientists unique access to engineered CRISPR-based technologies as they strive to deliver off-the-shelf NK cell therapies and novel combination approaches that improve upon the first generation of cell therapies, said Frank Nestle, Sanofis head of research and chief scientific officer, in a statement.

Sanofi missed the first wave of cancer cell therapy development, which companies like Novartis, Gilead and, more recently, Bristol Myers Squibb have led. But it appears interested in making up ground with bets on newer technologies.

In November 2020, Sanofi bought Kiadis Pharma and its pipeline of donor-derived NK cell therapies. Five months later, the company acquired Tidal Therapeutics, which was attempting to use messenger RNA to reprogram immune cells in the body to attack cancers.

While a much smaller financial commitment, the partnership with Scribe could help Sanofi better develop NK cells therapies. Scribes gene editing technology relies on the CRISPR framework pioneered by its cofounder Jennifer Doudna, but the company has developed its own DNA-cutting enzymes, too.

Scribe raised $100 million in a Series B round last spring and in March hired ex-Barclays banker David Parrot as its chief financial officer. In an interview with CFO Dive, Parrot said he had been brought on to help eventually launch an initial public offering, but noted the company would focus first on inking partnerships as public markets remain cool to IPOs.

The deal with Sanofi is the second Scribe has disclosed publicly. Its also working with Biogen on a research collaboration focused on ALS and another undisclosed disease.

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Sanofi partners with Scribe to gain gene editing tools for cell therapy work - BioPharma Dive

By the end of March, Vertex Pharmaceuticals and CRISPR Therapeutics expect to have submitted a U.S. approval application for a gene editing medicine designed to treat two rare blood disorders.

On Tuesday, the companies said the Food and Drug Administration is allowing a so-called rolling review of their medicine, named exa-cel, for the treatment of sickle cell disease and beta thalassemia. Filing is slated to begin in November, with a completed application anticipated some time in the first quarter of next year. In Europe, where Vertex and CRISPR are also seeking approval, the companies said theyre on track to file by the end of this year.

If approved, exa-cel would become the first marketed therapy based on the CRISPR gene editing technology that won a Nobel Prize in 2020. Data generated in clinical studies have so far shown that, for most patients, a one-time treatment with exa-cel significantly alleviates the symptoms and burdens of sickle cell and beta thalassemia.

We continue to work with urgency to bring forward the first CRISPR therapy for a genetic disease, and one that holds potential to transform the lives of patients, said Nia Tatsis, Vertexs chief regulatory and quality officer, in a statement.

Vertex previously aimed to submit a full application by the end of 2022, wrote Brian Abrahams, an analyst at the investment firm RBC Capital Markets, in a note to clients.Still, Abrahams and his team wouldnt expect a few months of difference in expected filing time to be material.

More concerning, according to the RBC team, is the potential sales outlook for exa-cel.

Several companies, including deep-pocked players like Pfizer, Novartis and Novo Nordisk, are trying to develop new medicines for sickle cell and beta thalassemia. And just last month, Massachusetts-based Bluebird bio secured FDA approval of a gene therapy another one-time, long-lasting treatment for patients with severe beta thalassemia who require blood transfusions. Bluebird is developing a gene therapy for sickle cell, too.

Additionally, the way exa-cel is administered could affect how many patients seek it out.

The medicine is made with a patients own stem cells, which are engineered and then implanted back into the bone marrow. The process requires patients be conditioned with busulfan, a chemotherapy-based regimen that can be difficult to tolerate. For example, one patient in the exa-cel clinical trial experienced bleeding in the brain that researchers attributed to this regimen.

CRISPR has said its exploring alternative conditioning procedures that dont involve chemotherapy. Even so, some analysts remain skeptical. Luca Issi, an RBC analyst who covers Beam Therapeutics, another company developing a gene-editing treatment for sickle cell, believes the commercial prospects for Beams program would be capped by its use of busulfan conditioning.

We remain cautious on exa-cel's ultimate commercial opportunity given our prior [conversations with doctors and patients], at least not until the much longer term once less toxic pre-conditioning regimens can be deployed, Abrahams wrote.

Vertex, meanwhile, has appeared more confident in exa-cels sales potential. Last year, the company paid CRISPR $900 million to amend their partnership so Vertex receives a greater portion of the profits should exa-cel come to market.

Excerpt from:
Vertex given green light to seek US approval of CRISPR-based therapy - BioPharma Dive

MIAMI--(BUSINESS WIRE)--The 2022 Cell & Gene Meeting on the Mesa annual conference will be held in Carlsbad, California, on October 11-13, 2022, bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures. Tackling the commercialization hurdles facing the cell and gene therapy sector today, this meeting covers a wide range of topics from clinical trial design to alternative payment models to scale-up and supply chain platforms for advanced therapies. Meet with the OrganaBio team to learn about our reliable supply of high-quality, ethically sourced tissue and cellular raw materials with clear paths to clinical translation, and the advanced processing and characterization capabilities we offer to speed up novel therapeutic development.

OrganaBios CEO, Justin Irizarry, and VP of Corporate Development, Dr. Priya Baraniak, will join the over 1,700 attendees, and will be available for one-on-one meetings to discuss available solutions to cell therapy developers.

http://www.organabio.com

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Meet with the OrganaBio executives in-person at The Cell & Gene Meeting on the Mesa - Business Wire