Archive for the ‘Gene Therapy Research’ Category

A biotechnology company launched Wednesday by life sciences venture firm ATP is the latest startup to debut with a new twist on genetic editing.

With $50 million in funding, Boston-based Ascidian Therapeutics claims its RNA exon editing approach could match the durability of gene therapy while avoiding some of the risks that come with editing DNA.

Its platform is designed to correct for mutations in exons the regions of DNA that contain information needed to make proteins. Ascidian aims to do this by replacing mutated exons with functional RNA copies as DNA is being converted into its chemical cousin.

The company will first target a genetic eye condition called Stargardt disease, which is the most common form of inherited macular degeneration and results in vision loss.

According to Ascidian, its technology can fix genetic errors that other editing approaches cant, and can be applied to widely varied genes. Its lead program can replace more than 20 exons at a time, said Romesh Subramanian, Ascidians CEO.

We are changing chapters in a book rather than whiting-out one letter at a time, Subramanian, said in an interview with BioPharma Dive. Subramanian came to Ascidian from Dyne Therapeutics, a biotech he founded and led as CEO. He previously founded RNA specialist Translate Bio, which was bought by Sanofi last year.

Subramanian claims that Ascidians approach, by focusing on RNA, maintains genome integrity and thereby sidesteps concerns around off-target edits. His company also doesnt rely on foreign enzymes to work, potentially easing immunogenicity risks, he added.

Along with Stargardt disease, Ascidian is looking at other eye conditions, neurological disorders and rare diseases. Subramanian declined to disclose how many drug research programs Ascidian plans to roll out.

Ascidians name is derived from a class of ocean-dwelling invertebrate creatures, which are sometimes known as sea squirts. These creatures use RNA trans-splicing to alter the RNA messengers used by their cells, a process that Ascidian plans to leverage to rewrite RNA for treating disease.

Ascidian is not ATPs first foray into genetic medicine. Last year, Ascidian co-founder and ATP venture partner Michael Ehlers, a former Biogen executive,launched a startup called Intergalactic Therapeuticsthat focuses on non-viral gene therapy. ATP has also built a company called Replicate, which is developing another kind of RNA medicine.

We think the RNA space is a big way of manipulating biology and treating disease across the board, and this approach we've taken to Ascidian defines a new class of RNA therapeutics, Ehlers said.

The company expects to spend the rest of 2022 and 2023 on pre-clinical studies for its lead program, along with developing proof of concept for other candidates targeting neurological and neuromuscular diseases.

Gene editing research was catalyzed by the discovery of CRISPR, which has now been extended and adapted to support several different gene editing technologies. But biotech companies are also exploring RNA editing, which in part appeals to scientists because it doesnt change the underlying DNA.

It has drawn in larger drugmakers, too: Roche and Eli Lilly have recently formed partnerships with Shape Therapeutics and ProQR Therapeutics, respectively, to develop treatments for a wide variety of diseases.

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Ascidian starts up with $50M and a twist on RNA editing - BioPharma Dive

ReportLinker

The Global Viral Vectors Market size was estimated at USD 1,291. 23 million in 2021 and expected to reach USD 1,464. 47 million in 2022, and is projected to grow at a CAGR 13. 67% to reach USD 2,785.

New York, Oct. 14, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Viral Vectors Market Research Report by Type, Disease, Application, End User, Region - Global Forecast to 2027 - Cumulative Impact of COVID-19" - https://www.reportlinker.com/p06342341/?utm_source=GNW 63 million by 2027.

Market Statistics:The report provides market sizing and forecast across 7 major currencies - USD, EUR, JPY, GBP, AUD, CAD, and CHF. It helps organization leaders make better decisions when currency exchange data is readily available. In this report, the years 2018 and 2020 are considered as historical years, 2021 as the base year, 2022 as the estimated year, and years from 2023 to 2027 are considered as the forecast period.

Market Segmentation & Coverage:This research report categorizes the Viral Vectors to forecast the revenues and analyze the trends in each of the following sub-markets:

Based on Type, the market was studied across Adeno-associated Viral Vectors, Adenoviral Vectors, and Retroviral Vectors.

Based on Disease, the market was studied across Cancers, Genetic Disorders, and Infectious Diseases.

Based on Application, the market was studied across Gene Therapy and Vaccinology.

Based on End User, the market was studied across Pharmaceutical & Biopharmaceutical Companies and Research Institutes.

Based on Region, the market was studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, Thailand, and Vietnam. The Europe, Middle East & Africa is further studied across Denmark, Egypt, Finland, France, Germany, Israel, Italy, Netherlands, Nigeria, Norway, Poland, Qatar, Russia, Saudi Arabia, South Africa, Spain, Sweden, Switzerland, Turkey, United Arab Emirates, and United Kingdom.

Cumulative Impact of COVID-19:COVID-19 is an incomparable global public health emergency that has affected almost every industry, and the long-term effects are projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlying COVID-19 issues and potential paths forward. The report delivers insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecasts, considering the COVID-19 impact on the market.

Cumulative Impact of 2022 Russia Ukraine Conflict:We continuously monitor and update reports on political and economic uncertainty due to the Russian invasion of Ukraine. Negative impacts are significantly foreseen globally, especially across Eastern Europe, European Union, Eastern & Central Asia, and the United States. This contention has severely affected lives and livelihoods and represents far-reaching disruptions in trade dynamics. The potential effects of ongoing war and uncertainty in Eastern Europe are expected to have an adverse impact on the world economy, with especially long-term harsh effects on Russia.This report uncovers the impact of demand & supply, pricing variants, strategic uptake of vendors, and recommendations for Viral Vectors market considering the current update on the conflict and its global response.

Competitive Strategic Window:The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies to help the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. It describes the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period.

FPNV Positioning Matrix:The FPNV Positioning Matrix evaluates and categorizes the vendors in the Viral Vectors Market based on Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Market Share Analysis:The Market Share Analysis offers the analysis of vendors considering their contribution to the overall market. It provides the idea of its revenue generation into the overall market compared to other vendors in the space. It provides insights into how vendors are performing in terms of revenue generation and customer base compared to others. Knowing market share offers an idea of the size and competitiveness of the vendors for the base year. It reveals the market characteristics in terms of accumulation, fragmentation, dominance, and amalgamation traits.

Competitive Scenario:The Competitive Scenario provides an outlook analysis of the various business growth strategies adopted by the vendors. The news covered in this section deliver valuable thoughts at the different stage while keeping up-to-date with the business and engage stakeholders in the economic debate. The competitive scenario represents press releases or news of the companies categorized into Merger & Acquisition, Agreement, Collaboration, & Partnership, New Product Launch & Enhancement, Investment & Funding, and Award, Recognition, & Expansion. All the news collected help vendor to understand the gaps in the marketplace and competitors strength and weakness thereby, providing insights to enhance product and service.

Company Usability Profiles:The report profoundly explores the recent significant developments by the leading vendors and innovation profiles in the Global Viral Vectors Market, including ABL Inc., Batavia Biosciences B.V., BioNTech IMFS GmbH, Biovian Oy, Cell and Gene Therapy Catapult, Cevec Pharmaceuticals GmbH, Creative Biogene, FinVector Vision Therapies, Fujifilm Diosynth Biotechnologies, GeneOne Life Science, Inc., Genezen Laboratories, Lonza Group AG, Merck KGaA, Miltenyi Biotec GmbH, Novasep Inc., Sirion-Biotech GmbH, Spark Therapeutics Inc., Thermo Fisher Scientific Inc., and Wuxi AppTec Co., Ltd..

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on the market offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyze penetration across mature segments of the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, certification, regulatory approvals, patent landscape, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and breakthrough product developments

The report answers questions such as:1. What is the market size and forecast of the Global Viral Vectors Market?2. What are the inhibiting factors and impact of COVID-19 shaping the Global Viral Vectors Market during the forecast period?3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Viral Vectors Market?4. What is the competitive strategic window for opportunities in the Global Viral Vectors Market?5. What are the technology trends and regulatory frameworks in the Global Viral Vectors Market?6. What is the market share of the leading vendors in the Global Viral Vectors Market?7. What modes and strategic moves are considered suitable for entering the Global Viral Vectors Market?Read the full report: https://www.reportlinker.com/p06342341/?utm_source=GNW

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Andrew P. Byrnes, Ph.D.

Office of Tissues and Advanced TherapiesDivision of Cellular and Gene TherapiesGene Transfer and Immunogenicity Branch

Andrew.Byrnes@fda.hhs.gov

Chief, Gene Transfer and Immunogenicity Branch

BS, MS, Yale University, Department of Molecular Biophysics and Biochemistry

PhD, Oxford University, Department of Human Anatomy

Postdoctoral Fellow, Johns Hopkins University, School of Hygiene and Public Health

Gene therapy holds great promise for treating cancer, inherited disorders, and other diseases. Gene therapy uses carriers called 'vectors' to deliver genes to tissues where they are needed. Researchers are currently investigating the safety and effectiveness of a variety of different gene therapy vectors in hundreds of clinical trials in the US.

We are studying one type of commonly-used gene therapy vector that is made from a disabled cold virus -- the adenovirus vector. While adenovirus vectors are very efficient at delivering genes, adenovirus vectors are not always easy to target to the correct tissue. In addition, adenovirus vectors can cause toxic effects that limit the amount of vector that doctors can give to patients. We are particularly interested in how to safely deliver large amounts of adenovirus vectors intravenously, with the goal of specifically targeting tumors and other tissues.

New adenovirus gene therapy vectors are tested in animals before human clinical trials begin, and it is important for both researchers and the FDA to know how well these animal studies can predict safety. Thus, another of our major goals is to develop animal models that reliably predict the safety and effectiveness of adenovirus vectors in humans.

Our studies will help us to understand the mechanisms for adenovirus vector targeting and toxicity, and the relevance of animal models to human outcomes. This new knowledge will enable researchers to design safer and more effective gene therapy vectors.

Adenovirus (Ad) vectors have shown considerable promise in animal models and are currently being used in numerous clinical trials, especially for the therapy of cancer. We are interested in improving the safety and efficacy of Ad vectors, especially when administered through the vascular system. Certain properties of Ad vectors make them hazardous to administer intravenously in large doses, and our laboratory is trying to understand and fix this problem.

One of our major areas of interest is the innate immune response to Ad vectors. These rapid responses can cause serious toxicity and may severely limit the doses of Ad vectors that are safe to use. In addition, we are also studying how cells in the liver such as Kupffer cells and hepatocytes recognize Ad, since the liver is the major site at which Ad vectors are cleared from the circulation. A better understanding of these mechanisms will help us to develop strategies to improve vector efficacy and reduce toxicity. We will also gain a better understanding of the advantages and disadvantages of using different animal species to predict the behavior of Ad vectors in humans, which is particularly relevant to the regulatory work of the FDA.

Recent work from our lab and others has shown that Ad vectors are heavily influenced by plasma proteins that rapidly opsonize the vectors after intravenous injection. We found that natural IgM antibodies bind to Ad vectors, activate complement, and reduce liver transduction. Intriguingly, the Ad hexon protein specifically binds to coagulation factor X (FX), and we found that recruitment of FX by Ad vectors protects them against neutralization by complement. These findings show that Ad vectors recruit a number of plasma proteins that interact in complex ways with each other and with cells, and that these host proteins ultimately help to determine whether the vector successfully reaches its target.

In the long run, a better fundamental understanding of Ad vector biology will facilitate the design of safer Ad vectors that are easier to target. Better animal models will be important for testing novel vectors for safety and efficacy.

02/22/2022

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Safety and Effectiveness of Gene Therapy | FDA

The cutting-edge Cellaca PLX system, designed by the companys Nexcelom unit, combines best-in-class image cytometer hardware, software, validated consumables and trackable data reporting all in one system without requiring complex calibration procedures or intense training requirements. To further streamline the customer experience, optimized reagent kits with validated antibodies from PerkinElmers BioLegend business are also part of the proprietary solution.

The new offering provides researchers expanded cell sample CQA analysis options beyond flow cytometry and staining methods, which historically have required a variety of different instruments and analytical methods. By combining these capabilities, researchers can now detect multiple markers simultaneously (multiplexing) and perform immunophenotyping and viability assays in seconds with an easy-to-use, modern user interface.

"Pharmaceutical companies have invested heavily in cell and gene therapy, but they struggle to assess the complex cell samples required to meet immense scientific demands and regulatory rigor across their research and manufacturing processes," said Alan Fletcher, senior vice president, Life Sciences, PerkinElmer. "While the Cellaca PLX Image Cytometer platform is therapeutic area agnostic, it is expected to be especially beneficial for researchers working in CAR-T cell therapy who want to streamline their phenotyping of immune cells for downstream processes."

PerkinElmers Nexcelom unit is a leading provider of automated cell counting technology and image cytometry products for cell analysis, including the original and widely adopted Cellaca MX high-throughput automated cell counter. Learn more about the new platform and other image cytometry instruments and reagents at BioProcessing International East from September 27-30 in Boston where PerkinElmer is showcasing the latest innovations across its extensive Life Science and cell and gene therapy portfolio in booths 625 and 631. Product demonstrations can be scheduled here.

About PerkinElmer

PerkinElmer is a leading, global provider of end-to-end solutions that help scientists, researchers and clinicians better diagnose disease, discover new and more personalized drugs, monitor the safety and quality of our food, and drive environmental and applied analysis excellence. With an 85-year legacy of advancing science and a mission of innovating for a healthier world, our dedicated team of more than 16,000 collaborates closely with commercial, government, academic and healthcare customers to deliver reagents, assays, instruments, automation, informatics and strategic services that accelerate workflows, deliver actionable insights and support improved decision making.

We are also deeply committed to good corporate citizenship through our dynamic ESG and sustainability programs. The Company reported revenues of approximately $5.0 billion in 2021, serves customers in 190 countries, and is a component of the S&P 500 index. Additional information is available at http://www.perkinelmer.com. Follow PerkinElmer on LinkedIn, Twitter, Facebook, Instagram, and YouTube.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220926005402/en/

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PerkinElmer Unveils Industry-first Cell Analysis Solution to Streamline ...

Biopharma focuses on streamlining biomanufacturing and supply chain issues to drive uptake of cell and gene therapies.

Cell and gene therapies (CGTs) offer significant advances in patient care by helping to treat or potentially cure a range of conditions that have been untouched by small molecule and biologic agents. Over the past two decades, more than 20 CGTs have been approved by FDA in the United States and many of these one-time treatments cost between US$375,00 and US$2 million a shot (1). Given the high financial outlay and patient expectations of these life-saving therapies, it is essential that manufacturers provide integrated services across the whole of the supply chain to ensure efficient biomanufacturing processes and seamless logistics to reduce barriers to uptake.

The following looks at the who, what, when, and why of biomanufacturing and logistics in CGTs in the bio/pharmaceutical industry in more detail.

According to market research, the global gene therapy market will reach US$9.0 billion by 2027 due to favorable reimbursement policies and guidelines, product approvals and fast-track designations, growing demand for chimeric antigen receptor (CAR) T cell-based gene therapies, and improvements in RNA, DNA, and oncolytic viral vectors (1).

In 2020, CGT manufacturers attracted approximately US$2.3 billion in investment funding (1). Key players in the CGT market include Amgen, Bristol-Myers Squibb Company, Dendreon, Gilead Sciences, Novartis, Organogenesis, Roche (Spark Therapeutics), Smith Nephew, and Vericel. In recent years, growth in the CGT market has fueled some high-profile mergers and acquisitions including bluebird bio/BioMarin, Celgene/Juno Therapeutics, Gilead Sciences/Kite, Novartis/AveXis and the CDMO CELLforCURE, Roche/Spark Therapeutics, and Smith & Nephew/Osiris Therapeutics.

Many bio/pharma companies are re-considering their commercialization strategies and have re-invested in R&D to standardize vector productions and purification, implement forward engineering techniques in cell therapies, and improve cryopreservation of cellular samples as well as exploring the development of off-the-shelf allogeneic cell solutions (2).

The successful development of CGTs has highlighted major bottlenecks in the manufacturing facilities, and at times, a shortage of raw materials (3). Pharma companies are now taking a close look at their internal capabilities and either investing in their own manufacturing facilities or outsourcing to contract development and manufacturing organizations (CDMOs) or contract manufacturing organizations (CMOs) to expand their manufacturing abilities (4). Recently, several CDMOsSamsung Biologics, Fujifilm Diosynth, Boehringer Ingelheim, and Lonzahave all expanded their biomanufacturing facilities to meet demand (5).

A major challenge for CGT manufacturers is the seamless delivery of advanced therapies. There is no room for error. If manufacturers cannot deliver the CGT therapy to the patient with ease, the efficacy of the product becomes obsolete. Many of these therapies are not off-the-shelf solutions and therefore require timely delivery and must be maintained at precise temperatures to remain viable. Thus, manufacturers must not only conform to regulations, but they must also put in place logistical processes and contingency plans to optimize tracking, packaging, cold storage, and transportation through the products journey. Time is of the essence, and several manufacturers have failed to meet patient demands, which have significant impacts on the applicability of these agents.

Several CAR T-cell therapies have now been approved; however, research indicates that a fifth of cancer patients who are eligible for CAR-T therapies pass away while waiting for a manufacturing slot (6). Initially, the manufacture of many of these autologous products took around a month, but certain agents can now be produced in fewer than two weeks (7). Companies are exploring new ways to reduce vein-to-vein time (collection and reinfusion) through the development of more advanced gene-transfer tools with CARs (such as transposon, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) among others, and the use of centralized organization with standardized apheresis centers (5). Others are exploring the use of the of allogeneic stem cells including Regen Biopharma, Escape Therapeutics, Lonza, Pluristem Therapeutics, and ViaCord (7).

Several gene therapies have also been approved, mainly in the treatment of rare disease (8). Many companies are evaluating novel gene therapy vectors to increase levels of gene expression/protein productions, reduce immunogenicity and improve durability including Astellas Gene Therapies, Bayer, ArrowHead Pharmaceuticals, Bayer, Bluebird Bio, Intellia Therapeutics, Kystal Biotech, MeiraGTx, Regenxbio, Roche, Rocket Pharmaceuticals, Sangamo Therapeutics, Vertex Pharmaceuticals, Verve Therapeutics, and Voyager Therapeutics (8).

While many biopharma companies have established their own in-house CGT good manufacturing practice (GMP) operation capabilities, others are looking to decentralize manufacturing and improve distribution by relying on external contracts with CDMOs and CMOs such as CELLforCURE, CCRM, Cell Therapies Pty Ltd (CTPL), Cellular Therapeutics Ltd (CTL), Eufets GmbH, Gravitas Biomanufacturing, Hitachi Chemical Advances Therapeutic Solutions, Lonza, MasTHerCell, MEDINET Co., Takara Bio, and XuXi PharmaTech (6, 9, 10).

The top 50 gene therapy start-up companies have attracted more than $11.6 billion in funds in recent years, with the top 10 companies generating US$5.3 billion in series A to D funding rounds (10). US-based Sana Biotechnology leads the field garnering US$700 million to develop scalable manufacturing for genetically engineered cells and its pipeline program, which include CAR-T cell-based therapies in oncology and CNS (Central Nervous System) disorders (11). In second place, Editas Medicine attracted $656.6 million to develop CRISPR nuclease gene editing technologies to develop gene therapies for rare disorders (12).

Overall, CGTs have attracted the pharma industrys attention as they provide an alternative route to target diseases that are poorly served by pharmaceutical and/or medical interventions, such as rare and orphan diseases. Private investors continue to pour money into this sector because a single shot has the potential to bring long-lasting clinical benefits to patients (13). In addition, regulators have approved several products and put in place fast track designation to speed up patient access to these life-saving medicines. Furthermore, healthcare providers have established reimbursement policies and manufacturers have negotiated value- and outcome-based contracts to reduce barriers to access to these premium priced products

On the downside, the manufacture of CGTs is labor intensive and expensive with manufacturing accounting for approximately 25% of operating expenses, plus there is still significant variation in the amount of product produced. On the medical side, many patients may not be suitable candidates for CGTs or not produce durable response due to pre-exposure to the viral vector, poor gene expression, and/or the development of immunogenicity due to pre-exposure to viral vectors. Those that can receive these therapies may suffer infusion site reactions, and unique adverse events such as cytokine release syndrome and neurological problems both of which can be fatal if not treated promptly (14).

Despite the considerable advances that have been made in the CGT field to date, there is still much work needed to enhance the durability of responses, increase biomanufacturing efficiencies and consistency and to implement a seamless supply chain that can ensure these agents are accessible, cost-effective, and a sustainable option to those in need.

Cleo Bern Hartley is a pharma consultant, former pharma analyst, and research scientist.

BioPharm InternationalVol. 35, No. 10October 2022Pages: 4951

When referring to this article, please cite it as C.B. Hartley, "Growth in Cell and Gene Therapy Market," BioPharm International 35 (10) 4951 (2022).

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Growth in Cell and Gene Therapy Market - BioPharm International

Allogene Therapeutics has begun the first pivotal test of an off-the-shelf cell therapy for cancer.

The biotechnology company, which has been at the forefront of a push in recent years to develop such treatments, known as allogeneic therapies and derived from donor cells, announced the start of two trials on Thursday. One will test a blood cancer drug known as ALLO-501A, while the other will evaluate a regimen Allogene uses to prepare patients for treatment. Assuming positive results, Allogene expects the studies will support approval applications for both of them.

The studies represent milestones for allogeneic cell therapies, which are meant to be more convenient alternatives to the personalized CAR-T treatments that have come to market for a handful of blood cancers. Allogene, spun out of Pfizers cell therapy work in 2018, is the largest and most advanced among the companies advancing them. Its run by former executives of Kite Pharma, which successfully developed the cell therapies now sold by Gilead Sciences.

Allogene raised more than a half a billion dollars in private financing and an initial public offering to fund its work. The company has had a bumpy ride since then, however. Its cell therapies, including ALLO-501A, have shown promise, but also face lingering questions about their durability and effectiveness compared to CAR-T treatments. The field has also gotten more competitive, with an emerging group of companies advancing therapies using different types of cells and CAR-T moving into earlier lines of care.

Additionally, Allogenes research was delayed by the unexpected finding of a chromosomal abnormality in a treated patient. The companys treatment was exonerated, but the FDA froze Allogenes programs for months during the investigation. Longtime partner Servier cut ties with the company last month as well.

The company now has the chance to prove how its technology stacks up. ALLO-501A is being tested in a Phase 2 study in patients with relapsed or refractory large B cell lymphoma, a setting for which multiple CAR-T treatments are already available.

In a research note, RBC Capital Markets analyst Luca Issi noted that its trial, ALPHA2, mimics the design of the studies underlying approvals of those treatments for lymphoma. Its a single-arm study of about 100 patients whove previously received at least two prior treatments, but not a CAR-T therapy. The study will be judged by ALLO-501As ability to induce a response. Allogene didnt disclose a bar for success, but Issi, after speaking with management, said executives believe efficacy needs to be in the range of approved CAR-T therapies.

Notably, the company is testing a single dose of the treatment, not a repeat-dosing regimen Allogene has been experimenting with to strengthen the effects of ALLO-501A. Issi indicated the decision was made for ease and convenience.

Allogenes other study, EXPAND, is a registrational trial for ALLO-647, an antibody drug the company is using to prepare patients for treatment. That study will enroll about 70 patients and have a control arm that doesnt receive Allogenes drug. Updates are expected by the end of the year, Allogene said.

Allogene shares climbed 12% in pre-market trading Friday, though at about $11, shares still trade well below their highs of about $54 apiece in 2020.

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Allogene starts first pivotal trials of an 'off-the-shelf' cell therapy for cancer - BioPharma Dive

Click to Access Audio Press Release

CHICAGO, Oct. 1, 2022 - The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the primary results from the Phase 1/2 study evaluating the investigational gene therapy botaretigene sparoparvovec (formerly AAV-RPGR) in patients with the inherited retinal disease X-linked retinitis pigmentosa (XLRP) associated with the retinitis pigmentosa GTPase regulator (RPGR) gene. Treatment with botaretigene sparoparvovec was found to have an acceptable safety profile, and efficacy assessments in this proof-of-concept study demonstrated encouraging improvements in retinal sensitivity, visual function and functional vision.1 These findings and additional updates, including data from a Phase 1 trial of investigational gene therapy JNJ-81201887 (JNJ-1887) for patients with geographic atrophy (GA), a late-stage and severe form of age-related macular degeneration (AMD), were presented in late-breaking oral presentations today at the Retina Subspecialty Day program of the American Academy of Ophthalmology (AAO) 2022 Annual Meeting (Abstracts #30071754 and #30071749).

XLRP is a rare condition estimated to impact one in 40,000 people globally.2,3 People with XLRP have progressive vision loss, starting in childhood with night blindness.4 Over time, they lose their peripheral vision leading to legal blindness by middle age.4 Botaretigene sparoparvovec is being investigated in collaboration with MeiraGTx Holdings plc to treat patients with XLRP caused by disease-causing variants in the eye-specific form of the RPGR (RPGR ORF15) gene. Through a one-time administration, botaretigene sparoparvovec is designed to deliver functional copies of the RPGR gene to counteract the loss of retinal cells with the goal of preserving and potentially restoring vision for those living with XLRP. Currently, there are no approved treatments for XLRP.4

"Individuals living with XLRP often begin to experience symptoms in childhood, and as retinal degeneration progresses toward blindness, they can start to feel a sense of hopelessness as there are no treatments to turn to," said Michel Michaelides, B.Sc., M.B., B.S., M.D. (Res), FRCOphth, FACS, Consultant Ophthalmologist, Moorfields Eye Hospital, Professor of Ophthalmology, University College London and lead investigator. "These results from the MGT009 study are promising, as they represent the potential for botaretigene sparoparvovec to preserve vision and ultimately restore hope for these patients."

The primary endpoint of the MGT009 study (NCT03252847) was safety, with secondary endpoints measuring changes in assessments of three domains of visionretinal sensitivity, visual function and functional visionat specified time points post-treatment.1 In the study's dose escalation and expansion phases, significant sustained or increased functional improvement in each visual domain was observed in participants treated with botaretigene sparoparvovec compared to the randomized untreated control arm of the study at six months post-treatment.1

Analyses of the pooled low and intermediate dose cohorts demonstrated improvement in retinal sensitivity in the treated eyes compared to untreated eyes in the randomized concurrent control arm as measured by both full-field static perimetry and microperimetry.1 An improvement in mean retinal sensitivity as measured by static perimetry in the central 10-degree area of the retina was observed at six months in the treated eyes compared to untreated eyes in the randomized concurrent control arm [in the full analysis of pooled low and intermediate doses across adults: 1.96 decibel (dB); (95% CI: 0.59, 3.34); and in the sensitivity analysis when applying the Phase 3 criteria: 2.42 (0.91, 3.93)].1

As part of the study, patients performed a functional vision assessment using a visual mobility maze to assess their ability to navigate through simulated real-life obstacles across a broad range of controlled light. At week 26, improvement in walk time was observed between the treated eyes in the low and intermediate dose cohorts and the untreated eyes in the randomized concurrent control arm at low illumination levels (full analysis nominal p-value < 0.05 at lux 1 and lux 16; in the sensitivity analysis when applying the Phase 3 criteria nominal p-value < 0.01 at lux 1, lux 4 and lux 16).1

The safety profile of botaretigene sparoparvovec observed in MGT009 was consistent with previous reports.1 Botaretigene sparoparvovec demonstrated an adverse event (AE) profile that was anticipated and manageable.1 Most AEs were related to the surgical delivery procedure, were transient and resolved without intervention.1 There were no dose-limiting events.1 A total of three serious adverse events (SAEs) were observed in the overall Phase 1/2 MGT009 clinical study; two SAEs, which were previously reported, were observed in the dose-escalation phase of the study (n=10; one retinal detachment and one panuveitis in the low dose cohort), and a single additional SAE of increased intraocular pressure was observed in the dose escalation phase and resolved with treatment.1

"Without an approved treatment option available, people with XLRP are faced with the inevitable fate of going blind in the prime of life," said James List, M.D., Ph.D., Global Therapeutic Area Head, Cardiovascular, Metabolism, Retina & Pulmonary Hypertension, Janssen Research & Development, LLC. "We're in a race to save sight for these patients and are encouraged by the strength of the data that we've shared so far. We look forward to advancing the clinical development of botaretigene sparoparvovec as part of our mission to preserve and potentially restore vision for these patients."

Further sensitivity analysis was conducted on study participants by applying the Phase 3 LUMEOS (NCT04671433) study eligibility criteria that corroborated the endpoints selected for the Phase 3 study.1 Currently, the LUMEOS study of botaretigene sparoparvovec for the treatment of patients with XLRP with disease-causing variants in the RPGR gene is actively dosing patients.

Phase 1 Data Evaluating JNJ-1887 in Geographic AtrophyJanssen also presented late-breaking data from a Phase 1, open-label, multicenter, dose-escalation, safety and tolerability study (NCT03144999) of a single intravitreal injection of JNJ-1887 in patients with advanced non-exudative (dry) age-related macular degeneration (AMD) with GA. GA is an irreversible condition that affects more than five million individuals worldwide.5 It has a devastating impact on GA patients' health-related quality of life, including their ability to read, drive and perform other day-to-day activities.5 In this study, patients (n=17) were sequentially enrolled at a low, intermediate and high dose without steroid prophylaxis, and all three doses of JNJ-1887 met the primary endpoint of safety over the two-year follow-up period.6 In addition, the supportive efficacy measures, including evaluation of GA lesion growth rates, showed a continual decline in lesion growth over six-month increments.6 These results are the first shared from the Company's common eye disease portfolio and indicate further evaluation of this investigational gene therapy is warranted.6

About the Phase 1/2 MGT009 Trial and Botaretigene SparoparvovecThe Phase 1/2 MGT009 trial (NCT03252847) was an open-label, multicenter dose escalation study that enrolled patients aged five years and older with X-linked retinitis pigmentosa (XLRP) caused by disease causing variants in the retinitis pigmentosa GTPase regulator (RPGR) gene at multiple sites in the United States and the United Kingdom. The primary endpoint was safety and tolerability; secondary endpoints assessed retinal sensitivity, visual function and functional vision.

The clinical study was composed of three parts: dose-escalation, pediatric dose-confirmation and an expansion phase. In the dose escalation phase, adult patients were treated at three escalating doses of botaretigene sparoparvovec; a low (2x1011 vg/mL), an intermediate (4x1011 vg/mL), and a high (8x1011 vg/mL) dose. In the expansion phase, 42 adult male patients were randomized to either immediate treatment with one of two low or intermediate doses or an untreated concurrent control arm with deferred treatment. At six months, the untreated control arm was randomized to receive either the low or intermediate treatment doses. Botaretigene sparoparvovec was administered through subretinal delivery in only one eye. The adult patients received treatment at three doses. The pediatric cohort (n=3) was only treated with an intermediate dose of botaretigene sparoparvovec.

Botaretigene sparoparvovec has been granted Fast Track and Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and PRIority MEdicines (PRIME), Advanced Therapy Medicinal Product (ATMP) and Orphan designations by the European Medicines Agency (EMA).

About the Janssen and MeiraGTx Strategic CollaborationIn January 2019, Janssen Research & Development, LLC entered into a worldwide collaboration and license agreementwith MeiraGTx Holdings plc, a clinical-stage gene therapy company, to develop, manufacture and commercialize its clinical-stage inherited retinal disease portfolio. Botaretigene sparoparvovec is being developed as part of a collaboration and license agreement. In addition to forming a research collaboration to explore new targets for other inherited retinal diseases, Janssen is working with MeiraGTx to build core capabilities in viral vector design, optimization and manufacturing.

About the Phase 1 JNJ-1887 Trial and JNJ-1887JNJ-81201887 (JNJ-1887), formerly referred to as AAVCAGsCD59, is an investigational gene therapy for the treatment of people with geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). JNJ-1887 is designed to increase the expression of a soluble form of CD59 (sCD59) intended to protect retinal cells to slow and prevent disease progression. JNJ-1887 was evaluated in a Phase 1 clinical trial (NCT03144999), an open-label, single-center dose escalation study to determine the safety of JNJ-1887 in adults 50 or older with advanced dry AMD with GA. The patients were treated at three escalating doses of JNJ-1887 without steroid prophylaxis through a single intravitreal injection in one eye.

This Phase 1 study met its primary endpoint of safety in all doses of JNJ-1887 (n=17), with supportive efficacy measures including evaluation of GA lesion growth rates, which showed a continual decline in lesion growth over six-month increments.

JNJ-1887 has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) and Advanced Therapy Medicinal Product (ATMP) designation by the European Medicines Agency (EMA).

About the Janssen Pharmaceutical Companies of Johnson & JohnsonAt Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism, & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.

Learn more at http://www.janssen.com. Follow us at@JanssenGlobal. Janssen Research & Development, LLC is part of the Janssen Pharmaceutical Companies of Johnson & Johnson.

Dr. Michaelides is a scientific founder of, and consultant to, and has a financial relationship with MeiraGTx.

Cautions Concerning Forward-Looking StatementsThis press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding botaretigene sparoparvovec and JNJ-81201887. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, any of the other Janssen Pharmaceutical Companies and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year endedJanuary 2, 2022, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online atwww.sec.gov,www.jnj.comor on request from Johnson & Johnson. None of the Janssen Pharmaceutical Companies nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

References1Michaelides, M et al. Ph1/2 AAV5-RPGR (Botaretigene Sparoparvovec) Gene Therapy Trial in RPGR-associated X-linked Retinitis Pigmentosa (XLRP). Abstract #30071754. Presented at the 2022 American Academy of Ophthalmology Annual Meeting.

2Boughman JA, Conneally PM, Nance WE. Population genetic studies of retinitis pigmentosa. Am J Hum Genet. 1980;32(2):223235.

3Fishman GA. Retinitis pigmentosa. Genetic percentages. Arch Ophthalmol. 1978;96(5):822826. doi:10.1001/archopht.1978.03910050428005.

4 Wang DY, Chan WM, Tam PO, et al. Gene mutations in retinitis pigmentosa and their clinical implications. Clin Chim Acta. 2005;351(1-2):5-16.

5 Cohen, MN et al. Phase 1 Study of JNJ-81201887 Gene Therapy in Geographic Atrophy (GA) Due to Age-related Macular Degeneration (AMD). Abstract #30071749. Presented at the 2022 American Academy of Ophthalmology Annual Meeting.

6 Singh RP, Patel SS, Neilsen JS, et al. Patient-, caregiver- and eye care professional-reported burden of geographic atrophy secondary to age-related macular degeneration. Am J Ophthalmic Clin Trials. 2019;2(1):1-6.

Investor Contact:Raychel KruperOffice +1 732-524-6164rkruper@its.jnj.com

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Janssen Announces Late-Breaking Data from Two Gene Therapy Programs at the American Academy of Ophthalmology 2022 Annual Meeting - Johnson &...

Essential that patients have the information they need to make informed choices in the future

Australians with inherited retinal disease (IRD) have a strong interest in undergoing gene therapy to prevent and treat blindness but theres a critical need for education programmes to help them make informed choices about future treatments, new research shows.

IRD is the umbrella term for broad group of genetic eye conditions, including retinitis pigmentosa and Stargardts disease, that cause progressive vison loss and blindness. They are the most common cause of blindness in working-age Australians, affecting more than 13,000 people nationally.

The study, led by the Centre for Eye Research Australia and University of Melbourne,reveals the results of the first national survey asking Australians living with IRD and their carers about their knowledge and views on gene therapy.

It provides new insight into patients knowledge of emerging gene therapies, the methods used, their willingness undergo future treatments and their views on the potential costs and logistics.

The findings demonstrated the need for continuing, targeted education about the outcomes and risks of gene therapy, and the difference between clinical research and approved treatments.

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Education vital to help patients explore gene therapies for blindness: Australian study - BSA bureau

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]

SOURCE AmplifyBio

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

Demand for viral-vector-based gene therapies has risen to unprecedented levels, thanks to their potential to help treat previously incurable diseases. The two vectors most in the spotlight? Lentiviral (LV) vectors and adeno-associated viral (AAV) vectors due to the increased research and positive clinical results they are seeing across a wide range of applications, including cancer, heart disease, and hematologic and genetic disorders. The more drug developers look to expand this range of therapeutic areas, the greater the demand for commercial-scale development. So its important to understand not only how these two vectors can be applied to drug development, but also the capabilities required for scale-up that allows us to bring these innovative therapies to patients.

LV vectors are derived from the single-stranded RNA retrovirus HIV-1, and have been used extensively because of their ability to infect non-dividing cells, efficiently integrate into the host genome, carry large transgene loads, and allow for long-term transgene expression. They are predominantly used as delivery vehicles for introducing genetic modifications into cell therapies, such as CAR-T, and HSC gene therapies. Importantly, recent regulatory approvals and clinical successes with LV vectors are spurring even more interest among drug developers.

Lets look at the benefits of LV vectors in more detail:

However, LV vectors also present two major risks to safety.

The first is a risk of accidental exposure because HIV can self-replicate during manufacturing thanks to the lentiviruss high mutation and recombination rate.Though research shows that the risk is low, it remains a major safety concern for lab engineers and workers during development. Before using a lentiviral vector system, a risk assessment must be completed and documented. Typically, lentiviral vectors may be safely handled using either BSL-2 or BSL-2 enhanced controls, depending upon the risk assessment.

The second risk is the potential for oncogenes to occur in cells through insertional mutagenesis. For this reason, lentiviral vectors are predominantly used for cell therapy applications with genetic modification of cells ex-vivo. Only limited use is seen for direct in vivo therapies.

Unlike their LV cousins, AAV vectors are single-stranded DNA parvoviruses that can replicate only in the presence of helper viruses, such as the adenovirus, herpes virus, human papillomavirus, and vaccinia virus. Following several landmark approvals, AAV vectors are currently being used for in vitro, ex vivo, and in vivo research. AAV therapies predominantly target rare genetic disorders for which the patient population tends to be highly limited. As the market is so small, drug developers feel immense pressure to be first to market to commercialize their therapies.

The biological elements of AAV vectors make them a very attractive candidate for gene therapy for several reasons:

As with LV vectors, AAV vectors come with several drawbacks that affect their applications and efficiency.

Firstly, AAV vectors are limited by their restricted capacity for insertion of transgene DNA; because of their relatively small transgene size, they are unable to deliver genes larger than 4.8 kilobytes. Secondly, the generation of neutralizing antibodies against AAV in non-human primates (NHP) and humans may attenuate the curative effects of AAV-mediated gene therapies and limit the size of patient populations suitable for these therapies. Thirdly, there are several different serotypes and capsids for AAVs, all of which have different production and purification requirements and vary greatly with respect to function and efficacy. Fourthly, AAV drug products have varying degrees of empty and partially filled capsids, and these have implications for safety and efficacy. Generally, the highest possible percentage of AAV particles with the full transgene DNA is desired, and this varies significantly depending on the production method, AAV serotype, and the transgene itself. The latter two factors introduce significant manufacturing challenges for AAV therapies.

Overall, the industrys collective ability to successfully scale up LVV and AAV vectors faces two challenges:

i) Manufacturing each viral vector currently requires different processes, so companies cannot apply a one-size-fits-all approach to their upstream and downstream processes. Therefore, manufacturing requires immense scientific and market expertise to make the informed decisions necessary for developing a robust plan.

ii) Given the industrys limited experience with commercial-scale viral vector supply, companies need to work closely with regulatory agencies. This can be especially challenging during the transition from preclinical to commercial, where complexities arise that can cause potential delays resulting in increased costs.

As demand continues to rise, pharma companies must understand how to navigate these challenges to continue delivering their life-saving medications.

Head of Commercial Development for Viral Vector, Cell and Gene Technologies (CGT) at Lonza

She works closely with the innovation, operations, engineering, strategic marketing, and business teams to enable prioritization, strategic development and commercialization of viral vector production services for CGT. Suparnas background is in Neuroscience, and she earned her PhD in Neuropharmacology from the University of Toronto. She has over 15 years of broad pharmaceutical and CDMO experience driving innovation, drug discovery, product and service development for CNS, oncology, and cell and gene therapy.

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The Pros and Cons of Lentiviral and Adeno-Associated Viral Vectors - The Medicine Maker

Gene and cell-based treatment is promising solutions for the control and cure of some chronic and life-threatening diseases such as sickle-cell disease (SCD), haemophilia, blood cancers, and HIV. Most of the current gene therapy clinical trials on SCD and HIV are conducted in North America.The treatment is either by using someone elses cells or those of the patient. Gene therapy, also called genetic engineering, involves getting ones cells (a patient), improving them either by enhancing them to fight disease or as a replacement for the diseased cells and using them to treat the disease.

Unlike in agriculture where a lot of the genetic engineering is on seed, Dr Cissy Kityo, the executive director at Joint Clinical Research Centre (JCRC) says in medicine, the human seed (ova or sperm) or the embryo is not touched.Its not about engineering custom humans as this has no current ethical basis. Therefore, it presents a new treatment paradigm, Dr Kityo says.

Gene therapy is administered once in a lifetime. Therefore, for someone with HIV, that eliminates the burden of taking ARTs. It also has the potential to save the overall healthcare cost and increase the individuals productivity.Research is ongoing to ensure this treatment is effective, safe, and free from off-target effects and any contamination.

The processDr Francis Ssali, the deputy executive director in charge of clinical care and research at JCRC, says genetic modification involves a series of processes, the first of which is to collect specialised white blood cells called T-cells and blood-forming stem cells from the patients blood.These cells are then taken to a clean medical laboratory where they are counted, checked for viability, and purified. Thereafter, the gene to correct the disease is inserted into these cells and this is done by either using special enzymes called CRISPR or by the use of self-inactivating partial viruses called Lentiviral vectors. The lentiviral vector delivers the required gene into the cells without resulting in viral infection in the patients cells, he says.

The process of introducing the corrective gene into the patients cell is called transduction and it can take between four to seven days to perform in the laboratory. Once the cells have received this gene modification, they are checked for quality and safety before they are ready for reinfusion back into the patient.In some instances, the patient is given medical treatment to enable them to receive the gene therapy cells, he adds.However, Dr Ssali says the current approaches to gene-therapy cell manufacturing are labour intensive and take a relatively long time to prepare, and require a large clean laboratory space.

Thankfully, there are newer laboratory instruments that can automate this genetic engineering work in a single closed instrument, with efficiency, he says.Uganda has 1.4 million people living with HIV and 400,000 people living with sickle cells yet adherence to medicine is inconsistent for some.Some HIV-resistant viral variants have emerged which threaten the efficacy of the treatment programme. As such, genetic engineering will be a blessing.Globally, the first-generation cure trials for HIV were done, second-generation trials are coming up and there is hope that soon a short-term cure will be got.

Ugandan perspectiveIn Uganda, Dr Ssali says the hope is that by 2030, Uganda will have controlled HIV/Aids greatly and also contributed to finding a functional cure.Dr Kityo says JCRC hopes to start HIV gene therapy trials in Uganda in 2024.The other focus is technology transfer where these gene therapy products are produced where they are needed, more efficiently, and more cost-effectively. That is why there will be more compact systems rather than the large labs, she adds.

In Africa, Uganda ranks fifth among countries with sickle cell disease and whereas bone-marrow transplants can cure SCD, only 10 percent of the eligible patients can get a matched donor. Nonetheless, with gene therapy, this will not be an issue since the patients own cells are used.Thankfully, the current gene therapy treatment technologies for HIV are the same used in sickle cell cure research. That is why preparing to address HIV also works to tackle the sickle cell disease, Dr Kityo says.

The joint Clinical Research Centre is working towards building the research teams and creating the necessary infrastructure for this novel research and clinical care. Arthur Makara, the coordinator of Uganda Biotechnology and Biosafety Consortium, calls for several partnerships because even when JCRC creates these technologies, they need help to mass produce them for a bigger population. Gene therapy only works on an individual, not on the sperm or ovary. Therefore, Dr Kityo says even after treatment, a sickle cell patient will still have sickle cell gene but normal cells in their marrow and live a normal life.

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Gene therapy brings hope to people with sickle cell, HIV - Monitor

(Precision Vaccinations)

Encouraging news from researchers at Fred Hutchinson Cancer Center in Washingtonrecently showed that an experimental gene therapy for herpes simplex virus infectionsmaysuppressthe amount of transmissible virus.

Drs.Keith JeromeandMartine Aubert, the virologists leading the research effort, reported the innovative treatment dramatically reduced or even eliminated viral shedding in treated mice compared to controls.

During multiple experiments, the researchers found substantial reductions in oral and genital viral shedding in the treated mice, with many of those treated showing no detectable virus shed.

This is an important milestone since the U.S. FDA has not approved any herpes prevention vaccine as of October 4, 2022.

"If you ask people living with Herpes simplex virus (HSV)what they care about, what they care most about is whether they have to worry about giving this virus to someone else, and shedding is how that happens," Dr. Jerome said in a media release issued on September 26, 2022.

Previously,Jerome and Aubert reportedthat the experimental drug could eliminate more than 90% of the latent herpesvirus in nerve clusters near the faces of the mice injected with the enzyme-carrying adeno-associated viruses (AAVs).

In this new study, they describe how they have tested the therapy for the first time to treat infections in a cluster of nerves called dorsal root ganglia near the genital tract of mice.

They found the experimental therapy reduced the latent herpes virusby about97%.

These results reinforce the curative potential of gene editing for latent orofacial and genital HSV disease, and provide a framework for additional safety studies before human trials can begin, wrote these researchers in a non-peer-reviewed study published on September 26, 2022.

HSV establishes latency in ganglionic neurons of the peripheral nervous system, from which it can reactivate, causing recurrent disease and possible transmission to a new host.

Current anti-HSV therapy does not eliminate latent HSV and thus is only suppressive rather than curative.

These researchers developed a potentially curative approach to latent HSV infection and pathogenesis based on gene editing using HSV-specific meganucleases delivered by AAVvectors.

This non-human study's results demonstrated that a dual meganuclease therapy, composed of two anti-HSV-1 meganucleases delivered by a triple AAV serotype combination (AAV9, AAV-Dj/8, AAV-Rh10), can eliminate up to 97% of latent HSV DNA from ganglia in both ocular and vaginal mouse models of latent HSV infection.

Using a novel pharmacological approach to reactivate latent HSV-1 in mice with the bromodomain inhibitor JQ-1, theydemonstrated that this reduction in ganglionic viral load leads to a significant reduction of viral shedding from treatedvs. control mice, with many treated mice showing no detectable virus shedding.

In general, the novel therapy was found well tolerated, although dose-ranging studies showed hepatotoxicity at high AAV doses, consistent with previous observations in animals and humans.

Also, in agreement with previous literature, theyobserved subtle histological evidence of neuronal injury in some experimental mice, although none of the mice demonstrated observable neurological signs or deficits.

The Hutch scientists noted that this study'spositivenews is tempered by recentconcerns within the gene therapy fieldabout the potential for therapies using AAVs to cause liver and nerve damage.

Jerome and Aubert are still hopeful that they can get FDA approval to test the therapy in humans in an early-stage clinical trialbefore the end of 2023.

The work was funded by the U.S. NIH, the Caladan Foundation, and more than 1,600 individual donors. Cellectis developed the meganucleases used in this research.

Disclosure note:Fred Hutch and certain of its scientists may benefit financially from this work in the future.

PrecisionVaccinations publishes fact-checked, research-based vaccine news manually curated for mobile readers.

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Herpes Gene Therapy Further Refined in Mice - Precision Vaccinations

New Jersey, United States,- Mr Accuracy Reports published new research on Global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy covering micro level of analysis by competitors and key business segments (2022-2029). The Global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy explores comprehensive study on various segments like opportunities, size, development, innovation, sales and overall growth of major players. The research is carried out on primary and secondary statistics sources and it consists both qualitative and quantitative detailing."The recession is going to come very badly . Please get to know your market RIGHT NOW with an extremely important information."

Some of the Major Key players profiled in the study are BioMarin Pharmaceutical, Sangamo Therapeutics, Amicus Therapeutics, Roche, Pfizer, NightstaRx, MeiraGTx, Sarepta Therapeutics, Neurocrine Biosciences, Voyager Therapeutics, Asklepios Biopharmaceutical

Get PDF Sample Report + All Related Table and Graphs @: https://www.mraccuracyreports.com/report-sample/588285

Various factors are responsible for the market's growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy market. This report is a consolidation of primary and secondary research, which provides market size, share, dynamics, and forecast for various segments and sub-segments considering the macro and micro environmental factors. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market.

Global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Segmentation:

Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Segmentation by Type:

Single-stranded AAV (ssAAV), Self-complementary AAV (scAAV).

Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Segmentation by Application:

Hemophilia, Ophthalmology, Lysosomal Storage Disorders, Neurological Disorders, Others

Key market aspects are illuminated in the report:

Executive Summary: It covers a summary of the most vital studies, the Global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy market increasing rate, modest circumstances, market trends, drivers and problems as well as macroscopic pointers.

Study Analysis: Covers major companies, vital market segments, the scope of the products offered in the Global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy market, the years measured and the study points.

Company Profile: Each Firm well-defined in this segment is screened based on a products, value, SWOT analysis, their ability and other significant features.

Manufacture by region: This Global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy report offers data on imports and exports, sales, production and key companies in all studied regional markets

Market Segmentation: By Geographical Analysis

The Middle East and Africa (GCC Countries and Egypt)North America (the United States, Mexico, and Canada)South America (Brazil etc.)Europe (Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

The cost analysis of the Global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market has been performed while keeping in view manufacturing expenses, labor cost, and raw materials and their market concentration rate, suppliers, and price trend. Other factors such as Supply chain, downstream buyers, and sourcing strategy have been assessed to provide a complete and in-depth view of the market. Buyers of the report will also be exposed to a study on market positioning with factors such as target client, brand strategy, and price strategy taken into consideration.

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Key questions answered in the report include:

who are the key market players in the Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market?Which are the major regions for dissimilar trades that are expected to eyewitness astonishing growth for the Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market?What are the regional growth trends and the leading revenue-generating regions for the Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market?What will be the market size and the growth rate by the end of the forecast period?What are the key Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market trends impacting the growth of the market?What are the major Product Types of Adeno-Associated Virus (AAV) Vector-Based Gene Therapy?What are the major applications of Adeno-Associated Virus (AAV) Vector-Based Gene Therapy?Which Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Services technologies will top the market in next 7 years?Table of Contents

Global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Research Report 2022 - 2029

Chapter 1 Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Forecast

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Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market will reach at $ 543.23 mn by 2032 demand and future scope with Russia-Ukraine Crisis...

Bloomsbury Genetic Therapies launches with Seed financing of 5 million to develop potentially curative gene therapy treatments for rare neurological and metabolic diseases

London, UK, 7 October 2022 Bloomsbury Genetic Therapies Limited (Bloomsbury), a biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases, based on clinically proven gene therapy technologies, today announced its launch with 5 million Seed financing led by UCL Technology Fund.

Bloomsburys innovative approach is designed to optimise therapeutic efficacy and safety, enable high manufacturability, accelerate development timelines and maximise regulatory success to create a pipeline of highly differentiated first- or best-in-class programs. The Companys lead programs are liver and CNS targeted gene therapies:

Liver targeted therapies: BGT-OTCD: Ornithine Transcarbamylase Deficiency (OTCD)

CNS targeted therapies: BGT-DTDS: Dopamine Transporter Deficiency Syndrome (DTDS)

BGT-NPC: Niemann-Pick Disease Type C (NPC)

BGT-INAD: Infantile Neuroaxonal Dystrophy (INAD)

These programs are underpinned by the unique medical insights derived from the internationally recognised clinics run by the companys academic founders, a deep understanding of disease biology and world-class translational science resulting from the close collaboration between Bloomsburys academic founders. Additionally, the viral vectors in the Companys programs have been designed with well-characterised adeno-associated virus (AAV) capsids that have demonstrated success in clinical and/or commercial settings. They have also been combined with innovative cassettes and routes of administration designed for optimal therapeutic effect.

Bloomsbury is developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases, said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. Our approach has been designed with a single goal in mind - to deliver the promise of gene therapy to rare disease patients by leveraging our industrys existing state-of-the art technology and we have already attracted a highly talented team of founders and experienced scientific and business professionals to the Company to that effect as we progress our first liver targeted gene therapy program, BGT-OTCD for the treatment of Ornithine Transcarbamylase Deficiency, into the clinic.

Our commitment to Bloomsbury is a great example of our strategy to seek to build truly innovative companies anchored by exceptional science and experienced teams, added Leigh Brody, Investment Manager of UCL Technology Fund. We are excited by the potential to develop best-in-class products in the gene therapy space.

Bloomsbury was founded as a spinout from UCL, and was supported by UCL Business, who helped foster the progression of these translational projects, supporting the team whilst managing the intellectual property, and making this deal possible. Bloomsbury is underpinned by world-leading gene therapy and rare disease expertise from the Companys academic founders:

After years of working relentlessly to identify and develop potential treatments for OTCD, NPC, DTDS and INAD, we are excited to have partnered with the team at Bloomsbury and are confident that these programs are in safe hands to be progressed through the development stage and towards regulatory approvals, with an ultimate goal of providing curative therapies to patients, added Professors Gissen, Kurian, Rahim and Waddington.

Senior leadership

Bloomsbury is led by Co-Founder & Chief Executive Officer, Adrien Lemoine, who brings 20 years of experience in pharma and biotech, including senior commercial, strategy and operation roles at AstraZeneca and GSK and joined the Company from Orchard Therapeutics where he served as Chief Business Officer. In this role he played a leading part in the companys fundraising efforts (Series B, Series C, NASDAQ IPO and follow-on financing) which raised over $750M and built the companys pipeline as well as setting-up Orchards discovery research efforts, alongside managing the program leadership & management function.

Alongside him on the senior leadership team are:

Board of Directors

Bloomsbury has also assembled an impressive trio of industry leaders as Independent Directors to assist management in driving the business forward; namely:

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Bloomsbury Genetic Therapies launches with Seed financing of 5 million to develop potentially curative gene therapy treatments for rare neurological -...

The prespecified exploratory analyses of the secondary survival endpoint demonstrated a >90% reduction in risk of death alone or in risk of death/permanently assisted ventilation at 24 weeks, when adjusted for baseline imbalances in risk (p=0.028 to p=0.075, unadjusted for multiple comparisons) with the CNM-Au8 30 mg dose. These survival results were statistically consistent for the 30 mg dose between the regimen only and full analysis sets, which included shared placebo from other regimens participating in the Healey ALS Platform trial (Regimens A, B, and D). This survival signal is consistent with results previously reported by Clene in the Phase 2 RESCUE-ALS trial with CNM-Au8.

The full analyses, including data on biomarkers of neurodegeneration and exploratory efficacy results, are expected later in 2022. The open-label extension will continue to follow participants and provide data updates in the future. Clene is in discussions with the Healey & AMG ALS Center to offer a broader EAP of CNM-Au8 30 mg for eligible participants of closed regimens and others.

Based on these topline findings, Clene has selected the CNM-Au8 30 mg dose for continued development in ALS. The CNM-Au8 60 mg dose did not demonstrate a survival benefit. CNM-Au8 was well-tolerated, and there were no drug-related serious adverse events or significant safety findings reported.

"There remains a high unmet medical need for treatments for people living with ALS. The potential survival benefit with CNM-Au8 at 30 mg is encouraging. Additional pre-specified exploratory analyses of both the RCT and open-label extension part of the study will be shared once available," said Merit Cudkowicz, M.D., MSc, principal investigator and sponsor of the Healey ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, chief of the Department of Neurology at Massachusetts General Hospital, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. "We are thankful to the many people who participated in this study. We will learn from these results and continue to use these data to inform future advances in ALS trial design," she concluded.

Robert Glanzman, M.D., FAAN, Clene's Chief Medical Officer, said, "We are very pleased to see a survival benefit in a broad population of people who had already been living with ALS for up to three years. Importantly, this is the second Phase 2 study demonstrating a survival benefit following CNM-Au8 treatment. CNM-Au8's mechanism of enabling energy metabolism and efficiency may not be reflected in the slope of ALSFRS-R change after only 24 weeks of treatment. These Healey ALS Platform Trial results support advancement of the CNM-Au8 30 mg dose. We look forward to discussions with U.S. regulatory authorities at an End of Phase 2 meeting for our CNM-Au8 development program in ALS."

Rob Etherington, Clene's President and CEO, added, "The survival results from this trial together with the consistent benefit seen in the open-label extension of the Phase 2 RESCUE-ALS trial, based on up to 31.5 months of long-term follow-up, support the rationale for treating neuronal and glial energetic failure with CNM-Au8. We have now completed multiple Phase 2 studies in ALS and MS, building a body of evidence demonstrating that CNM-Au8 supports cellular energy production, improving myelination and neuronal viability. We believe supporting brain energetic capacity translates to patient benefit, including survival. We will work closely with regulatory health authorities, ALS experts, and patient representatives to determine the proper path for FDA and EMA approval. Clene remains committed to advancing CNM-Au8 clinical programs to the ultimate goal of FDA approval. To support this effort, Clene is pursuing paths, including strategic partnerships, and is in dialogue with various potential partners."

Michael Hotchkin, Clene's Chief Development Officer, concluded, "We thank the ALS community for its support of the Healey ALS Platform trial. Furthermore, we thank the site investigators for their research excellence and dedication to patients, and we thank Dr. Cudkowicz and the team at the Healey & AMG ALS center for their leadership and for the development of the platform trial. Most importantly, we thank people living with ALS who participated in the study and their families for their effort and willingness to engage in clinical research."

Conference Call and Webcast Information

Clene will host a conference call and webcast at 8:30 am EDT to discuss the Healey ALS Platform trial topline results for CNM-Au8. Members of Clene's executive team will lead the discussion.

Time and Date: 8:30 a.m. EDT on Oct. 3, 2022 Investors: 1 (888) 660-6179 (toll-free) or 1 (929) 203-1946 (toll) Conference ID: 5318408 Press *1 to ask or withdraw a question, or *0 for operator assistance .

To access the live webcast, please register online at this link . Participants are requested to register at a minimum 15 minutes before the start of the call. A replay of the call will be available two hours after the call and archived on the same web page for six months. A live audio webcast of the call will be available on the Investors section of the Company's website Events page. An archived webcast will be available on the Company's website approximately two hours after the event.

About the Healey ALS Platform Trial The Healey ALS Platform Trial is a perpetual multi-center, randomized, double-blind, placebo-controlled program designed to evaluate the efficacy and safety of multiple investigational products utilizing a shared placebo group in people living with amyotrophic lateral sclerosis (ALS). In the CNM-Au8 regimen, 161 participants were randomized to 30 mg CNM-Au8, 60 mg CNM-Au8, or placebo as adjunct to standard of care for a 24-week treatment period. Active drug was offered to all participants who were eligible and elected to continue into the open-label extension. The primary outcome of the trial was the change in disease severity over time as measured by ALSFRS-R through 24 weeks accounting for mortality (analyzed using a Bayesian shared parameter model). Prespecified secondary efficacy endpoints included the Combined Assessment of Function and Survival joint rank test (CAFS), change in respiratory function as measured by slow vital capacity (SVC), and overall survival. For more information, please see ClinicalTrials.gov Identifier: NCT04297683 .

About CNM-Au8 CNM-Au8 is Clene's lead asset in mid- and late-stage clinical development for the treatment of multiple sclerosis and amyotrophic lateral sclerosis. An oral suspension of gold nanocrystals, CNM-Au8 was developed to protect neuronal health and function by increasing energy production and utilization. The catalytically active nanocrystals of CNM-Au8 drive critical cellular energy producing reactions that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors. CNM-Au8 is a federally registered trademark of Clene Nanomedicine, Inc.

About Clene Clene is a clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative disease by targeting energetic failure, an underlying cause of many neurological diseases. The company is based in Salt Lake City, Utah, with R&D and manufacturing operations in Maryland. For more information, please visit http://www.clene.com or follow us on Twitter , LinkedIn and Facebook.

Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended, which are intended to be covered by the "safe harbor" provisions created by those laws. Clene's forward-looking statements include, but are not limited to, statements regarding our or our management team's expectations, hopes, beliefs, intentions or strategies regarding our future operations. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words "anticipate," "believe," "contemplate," "continue," "estimate," "expect," "intends," "may," "might," "plan," "possible," "potential," "predict," "project," "should," "will," "would," and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. These forward-looking statements represent our views as of the date of this press release and involve a number of judgments, risks and uncertainties. We anticipate that subsequent events and developments will cause our views to change. We undertake no obligation to update forward-looking statements to reflect events or circumstances after the date they were made, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws. Accordingly, forward-looking statements should not be relied upon as representing our views as of any subsequent date. As a result of a number of known and unknown risks and uncertainties, our actual results or performance may be materially different from those expressed or implied by these forward-looking statements. Some factors that could cause actual results to differ include our ability to demonstrate the efficacy and safety of our drug candidates; the clinical results for our drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; our ability to achieve commercial success for our drug candidates, if approved; uncertainty regarding whether potential strategic partnerships will result in any agreements or transactions, or, if completed, any agreements or transactions will be successful or on attractive terms; our limited operating history and our ability to obtain additional funding for operations and to complete the development and commercialization of our drug candidates; and other risks and uncertainties set forth in "Risk Factors" in our most recent Annual Report on Form 10-K and any subsequent Quarterly Reports on Form 10-Q. In addition, statements that "we believe" and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date of this press release, and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain and you are cautioned not to rely unduly upon these statements. All information in this press release is as of the date of this press release. The information contained in any website referenced herein is not, and shall not be deemed to be, part of or incorporated into this press release.

Source: Clene Inc.

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Ginkgo Bioworks Acquires Circularis to Strengthen Capabilities in Cell and Gene Therapy - Investing News Network

DetailsCategory: DNA RNA and CellsPublished on Friday, 07 October 2022 11:34Hits: 395

Enrollment continues in clinical trial of MB-106 under Mustangs IND; next data disclosure anticipated 4Q 2022

Ongoing clinical trial of MB-106 at Fred Hutch continues to demonstrate high efficacy, durable responses, and favorable safety profile across wide range of hematologic malignancies

WORCESTER, MA, USA I October 06, 2022 I 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 that the first patient has been treated in its multicenter, open-label, non-randomized Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106, Mustangs first-in-class CD20-targeted, autologous CAR T cell therapy for the treatment of relapsed or refractory B-cell non-Hodgkin lymphomas (B-NHL) and chronic lymphocytic leukemia (CLL). The patient did not experience cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Center (Fred Hutch). The multicenter trial under Mustangs Investigational New Drug Application (IND) builds upon the initial, ongoing Phase 1/2 clinical trial taking place at Fred Hutch in a single-center study under Fred Hutchs IND.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang said, The first clinical trial under Mustangs IND is an important milestone in the ongoing development and evaluation of MB-106. Data presented at several prestigious medical meetings earlier this year from the initial, ongoing Phase 1/2 clinical trial at Fred Hutch show that MB-106 continues to demonstrate high efficacy and a favorable safety profile across patients with a wide range of hematologic malignancies. We look forward to providing updates on our multicenter MB-106 clinical trial as it progresses and anticipate reporting efficacy data in the fourth quarter of this year.

Interim data from 28 patients treated in the initial, ongoing Phase 1/2 investigator-sponsored clinical trial at Fred Hutch continue to support MB-106 as a viable CAR T cell therapy for B-NHLs and CLL. As of September 9, 2022, the interim data show:

We are excited to broaden the evaluation of MB-106 with this multicenter clinical trial under Mustangs IND. To date, the data from the initial, ongoing clinical trial at Fred Hutch continue to demonstrate a high rate of complete and durable responses, said Mazyar Shadman, M.D., M.P.H., Study Chair, Associate Professor and physician at Fred Hutch and University of Washington. In addition, MB-106 has shown potential to treat patients in an outpatient setting and provide another immunotherapy option for patients treated previously with CD19-directed CAR T cell therapy.

About Mustangs Multicenter MB-106 Phase 1/2 clinical trialThe six-center Phase 1/2 clinical trial is a three-arm study targeting CLL and B-NHL including FL, diffuse large B-cell lymphoma and mantle cell lymphoma. Included in the eligibility criteria are patients who have relapsed after treatment with CD19 CAR-T cell therapy. Additionally, the FL arm will evaluate other indolent histologies including Waldenstrom macroglobulinemia, a rare type of B-NHL for which the U.S. Food and Drug Administration recently granted MB-106 Orphan Drug Designation. Since the Mustang-sponsored multicenter clinical trial is using the same lentiviral vector as the Fred Hutch-sponsored single-center trial, the FDA has allowed dose escalation to begin at a higher dose than what was originally conducted at Fred Hutch.

An estimated 287 patients are anticipated to be enrolled in the trial. All patients must have evidence of CD20 expression in both phases of the clinical trial. In Phase 1, escalating MB-106 dose levels will be tested independently in each arm using a 3+3 design. Patients will be enrolled in one of three arms, based on their primary diagnosis.

A total of up to 18 patients are anticipated to be treated in each Phase 1 arm, including six patients at the maximum tolerated dose, prior to proceeding to the Phase 2 portion of the study for each respective arm, where a total of up to 71 patients will participate in each independent arm. Safety of each dose level will be reviewed for each arm until the maximum tolerated dose has been reached and the recommended Phase 2 dose (RP2D) has been established for each arm. An assessment of the safety and tolerability of the dose will be made by the Safety Review Committee based on the data from the 28-day dose-limiting toxicity observation period.

In Phase 2, specific arms of relapsed or refractory CD20-positive B-cell NHL or CLL patients will be treated with MB-106 at the respective RP2D for each arm. Each arm will initially include up to 20 patients. Based on the results of the interim analysis, up to an additional 51 patients may be added to each of the arms.

Additional information about the trial can be found on clinicaltrials.gov using the identifier NCT05360238.

About MB-106 (CD20-targeted autologous CAR T Cell Therapy)CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustangs research collaborator, Fred Hutch, in the laboratories of the late Oliver Press, M.D., Ph.D., and Brian Till, M.D., Associate Professor in the Clinical Research Division at Fred Hutch, and was exclusively licensed to Mustang in 2017. The lentiviral vector drug substance used to transduce patients cells to create the MB-106 drug product produced at Fred Hutch has been optimized as a third-generation CAR derived from a fully human antibody. MB-106 is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in patients with B-NHLs and CLL. The same lentiviral vector drug substance produced at Fred Hutch will be used to transduce patients cells to create the MB-106 drug product produced at Mustang Bios Worcester, MA, cell processing facility for administration in the multicenter Phase 1/2 clinical trial under Mustang Bios IND. It should be noted that Mustang Bio has introduced minor improvements to its cell processing to facilitate eventual commercial launch of the product. In addition, prior to commercial launch, Mustang Bio will replace the Fred Hutch lentiviral vector drug substance with vector produced at a commercial manufacturer. Additional information on these trials can be found athttp://www.clinicaltrials.govusing the identifierNCT05360238for the Mustang multicenter trial andNCT03277729for the ongoing trial at Fred Hutch.

About Mustang BioMustang Bio, Inc. 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 lentiviral gene therapies for severe combined immunodeficiency. 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.

SOURE: Mustang Bio

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Mustang Bio Announces First Patient Treated in Its Multicenter Phase 1/2 Clinical Trial of MB-106, a First-in-Class CD20-targeted, Autologous CAR T...

For medical geneticist Dr. Xiao-Ru Yang, BSc 13, MD16, few things are more rewarding than helping a patient understand what caused their mysterious, unexplained illness answers those patients often seek for many years.

Thanks to a new University of Calgary fellowship generously supported by Albertas only neurogeneticist, Dr. Oksana Suchowersky, MD 78, and her partner, the board chair of the Alberta Cancer Foundation, Dr. Chris Eagle, BSc 73, MD 77, Yang is poised to help sleuth out new diseases and improve health while adding Canadian capacity in the rapidly advancing field of neurogenetics.

There are so many diagnostic odyssey families, who get tests and investigations done for many years, but still have no diagnosis. To be able to tell these families, I think we actually have the answer now and see their reaction, is really incredible, she says.

Every case is different. It's really like solving a puzzle and you're motivated to continue to work and study these things until you do.

Yang is the first recipient of Canadas first clinical fellowship in neurogenetics and medical genetics, the 12-month Dr. Oksana Suchowersky and Dr. Chris Eagle Clinical Fellowships, offered alternating years at UCalgary and the University of Alberta.

The fellowship provides up to $100,000 to support training for exceptional clinical fellows who have completed residency in neurology or medical genetics. It allows trainees to conduct clinical or translational research or advanced subspecialty training in the area of neurogenetics (the study of the role of genetics in the development and function of the nervous system) or genetics (the diagnosis and management of genetic disorders).

Cumming School of Medicine alumni and UCalgary philanthropists, Oksana Suchowersky and Chris Eagle.

Courtesy Oksana Suchowersky and Chris Eagle

Over the past number of years, genetics has mushroomed its becoming an important point in how we diagnose and even treat patients. We're starting to use gene therapy in a lot of different specialties, says Suchowersky.

And yet in medical school, students receive only about seven hours of general training in genetics, and funding for clinical fellowships is very limited. That started the discussions between Chris and myself about the importance in developing expertise in genetics.

The donors have worn many hats in Albertas health care and education communities. Suchowersky is an adjunct professor and former department head of medical genetics at UCalgary. She is a professor in the departments of Medicine (Neurology), Medical Genetics and Paediatrics at the University of Alberta and director of the Neurogenetics and Huntington disease program.

Eagle is a past president and CEO of Alberta Health Services and past CEO of Calgary Health Trust. He is also a professor emeritus, former department head of anesthesiology and assistant dean of medical education at UCalgarys Cumming School of Medicine (CSM). He says:

We've watched generations of talented young physicians leave Alberta to get training elsewhere sometimes they return, sometimes they don't. This fellowship is an opportunity to keep or bring people here. Its also about making the care thats given in Alberta the best in the world.

Yang started her training this summer under the supervision of Dr. Francois Bernier, MD, Dr. Billie Au, MD, PhD and Dr. Mike Innes, MD, at the Alberta Childrens Hospital (ACH) all international leaders in their field. Shell learn how to analyze genomic data and how to ultimately apply emerging genetic sequencing technologies in the clinic.

The funding of this fellowship is transformational, supporting advanced training of the some of the best physicians in one of most rapidly advancing fields of medicine. Canada desperately needs academic and physician leaders to maximize the impact of genomic medicine and neurogenetics and UCalgary have consistently been at the forefront of these fields, says Bernier, professor and head of Medical Genetics.

Dr. Yang will be among the new generation of leaders in genomics thanks to this fellowship.

Rapid advances in genomics the study of genes and their functions are allowing researchers and physicians to customize health care and treat individuals according to their genetic makeup. This precision medicine approach is giving physicians more tools to understand what their patients need and to provide highly personalized, precise care.

There are more than 4,800 known, rare syndromes, affecting between one and two million Canadians. More accessible gene sequencing has allowed UCalgary geneticists to diagnose many previously undiagnosed, complex chronic diseases, which has a positive impact on families as well as the health-care system.

The cost to sequence the full genome in one single person used to be $100 million, but now costs about $1,000.

Yang moved from Halifax to Calgary with her family as a teenager and completed her five-year residency in medical genetics at the CSM earlier this year. During the COVID-19 pandemic, she became a mom to a now two-year-old boy. She previously had the opportunity to spend time during her residency in clinic with Suchowersky, who teaches at both UCalgary and UAlberta, and Yang looks forward to learning from the neurogeneticist again as part of the fellowship.

"I'm so grateful for this investment that Dr. Suchowersky and Dr. Eagle are making into training the next generation of physicians in the fields of genetics and neurology. I get to train with some really fantastic physicians and scientists and it wouldn't happen without their generosity, Yang says.

I hope through the course of my career that I can pay it forward by furthering the education of other trainees, like they're doing for me right now.

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Clinical fellowship offers opportunity to diagnose previously undiagnosable diseases through genetic 'detective' work - University of Calgary

LONDON, Oct. 04, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that the first patient has been dosed in the second dose cohort of the Phase 1/2 MARVEL-1 clinical trial of FLT190 in Fabry disease, a debilitating inherited disorder that leads to progressive organ damage and can result in early death as a result of a harmful build-up of fat in cells due to an enzyme deficiency.

We believe FLT190 has the potential to be a life-changing therapy for people with Fabry disease by providing durable enzyme activity above the normal range with a one-time treatment, and we are excited to have initiated this cohort in MARVEL-1, said Pamela Foulds, MD, Chief Medical Officer at Freeline. We have multiple trial sites now open across five countries with more expected to open by year-end. We look forward to reporting updated safety and efficacy data in the first half of next year.

Freeline recently reached alignment with the U.S. Food and Drug Administration to dose patients in MARVEL-1 in the United States. Efficacy and safety data from the first dose cohort of MARVEL-1 (in which patients received 7.5e11 vg/kg of FLT190) supported progression to the second, mid-dose cohort (1.5e12 vg/kg).

MARVEL-1 is an international, multicenter, adaptive dose-escalation and dose-expansion Phase1/2 clinical trial in adult men ( 18 years) with classic Fabry disease. The trial is evaluating the safety and efficacy of one-time treatment with FLT190 in up to four dose cohorts. Efficacy measures include-GalA activity levels and clearance of globotriaosylceramide (Gb3) and globotriaosylsphingosine (lyso-Gb3) as measured in plasma and urine. In the dose-escalation part, patient dosing is staggered to allow for adequate monitoring of both safety and efficacy. Patients will be monitored for nine months after dosing and will be eligible to participate in a long-term follow-up study for at least five years (MARVEL-2).

About Fabry Disease

Fabry disease is caused by a mutation in the GLA gene, which encodes for the enzyme -galactosidase A (-Gal A) that is required to metabolize a certain type of fat called globotriaosylceramide 3 (Gb3). As a result of deficient -Gal A production or function, Gb3 accumulates in the lysosome of various cell types and causes damage, commonly leading to a variety of painful and debilitating renal, cardiac, skin, neurological, and gastrointestinal conditions. A Gb3 metabolite, called lyso-Gb3, is considered a good biomarker for Fabry disease and may contribute to disease pathogenesis. Fabry disease can lead to premature death with renal failure and cardiac disease being the most common causes of death. Fabry disease affects approximately 16,000 people in the United States, European Union and Japan.

About FLT190 for Fabry Disease

FLT190 is a gene therapy candidate designed to generate durable plasma -Gal A levels above the normal range to enable sufficient enzyme activity in relevant tissues with a one-time treatment. FLT190 uses Freelines proprietary AAVS3 capsid to introduce a functional GLA gene into liver cells to produce functional -Gal A. AAVS3 has been rationally designed to enable effective liver cell transduction and strong and durable enzyme production at low doses and with a good safety profile. From the liver, -Gal A can circulate in the blood and be taken up by various tissues in the body, where it is transported to the cells and lysosomes and can break down Gb3.

About Freeline Therapeutics

Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies. The company is dedicated to improving patient lives through innovative, one-time treatments that may provide functional cures for inherited, systemic debilitating diseases. Freeline uses its proprietary, rationally designed AAV vector and capsid (AAVS3), along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing or dysfunctional protein into the patients bloodstream. The company has clinical programs in hemophilia B, Fabry disease, and Gaucher disease Type 1. Freeline is headquartered in the UK and has operations in Germany and the U.S.

Forward-Looking Statements

This press release contains statements that constitute forward-looking statements as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the opinions, expectations, beliefs, plans, objectives, assumptions or projections of Freeline Therapeutics Holdings plc (the Company) regarding future events or future results, in contrast with statements that reflect historical facts. All statements, other than historical facts, including statements regarding FLT190s potential to be a life-changing therapy for people with Fabry disease by providing durable enzyme activity above the normal range with a one-time treatment and that the Company expects to open more sites in its MARVEL-1 clinical trial by year end and plans to report updated safety and efficacy data from such trial in the first half of next year, are forward-looking statements. In some cases, you can identify such forward-looking statements by terminology such as anticipate, intend, believe, estimate, plan, seek, project, expect, may, will, would, could or should, the negative of these terms or similar expressions. Forward-looking statements are based on managements current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks and uncertainties, including the Companys recurring losses from operations; the uncertainties inherent in research and development of the Companys product candidates, including statements regarding the timing of initiation, enrollment, continuation, completion and the outcome of clinical studies or trials and related preparatory work and regulatory review, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preclinical and clinical data, including the possibility of unfavorable new preclinical, clinical or safety data and further analyses of existing preclinical, clinical or safety data; the Companys ability to design and implement successful clinical trials for its product candidates; whether the Companys cash resources will be sufficient to fund the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements for the Companys expected timeline; the potential for a pandemic, epidemic or outbreak of infectious diseases in the United States, United Kingdom or European Union, including the COVID-19 pandemic, to disrupt and delay the Companys clinical trial pipeline; the Companys failure to demonstrate the safety and efficacy of its product candidates; business interruptions resulting from geopolitical actions, including global hostilities, war and terrorism, global pandemics or natural disasters, including earthquakes, typhoons, floods and fires; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Companys ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Companys product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys limited manufacturing history, which could result in delays in the development, regulatory approval or commercialization of its product candidates; and the Companys ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. The Company cannot guarantee that any forward-looking statement will be realized. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated, or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties, and other matters can be found in the Companys Annual Report on Form 20-F for the fiscal year ended December 31, 2021, and in subsequent reports on Form 6-K, in each case including in the sections thereof captioned Cautionary Statement Regarding Forward-Looking Statements and Item 3.D. Risk factors. Many of these risks are outside of the Companys control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law. For further information, please reference the Companys reports and documents filed with the U.S. Securities and Exchange Commission (the SEC). You may review these documents by visiting EDGAR on the SEC website at http://www.sec.gov.

Media Contact:

Arne Naeveke, PhDVice President, Head of Corporate Communicationsarne.naeveke@freeline.life+1 617 312 2521

IR Contact:

Naomi Aokinaomi.aoki@freeline.lifeSenior Vice President, Head of Investor Relations & Communications+ 1 617 283 4298

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Freeline Initiates Dosing of Second Cohort in MARVEL-1 Trial of FLT190 Gene Therapy Candidate for People with Fabry Disease - GlobeNewswire

EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular diseases, today announced the dosing with ATA-100 of a first patient in a phase 1/2 clinical study in FRKP-related limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).

This is an exciting milestone for our company but most importantly, if this clinical trial is successful, it could have a life-changing impact for patients affected by LGMD-R9, said Stephane Degove, Chief Executive Officer and Co-Founder of Atamyo Therapeutics.

This clinical trial (EudraCT 2021-004276-33, NCT05224505) is a multicenter, Phase 1/2 study evaluating safety, pharmacodynamic, efficacy, and immunogenicity of intravenous ATA-100, a single-dose Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.

This study will consist of 2 phases: an open-label dose escalation phase (Stage 1) and a double-blind placebo controlled, randomized phase (Stage 2).

LGMD-R9 is a severe progressive and debilitating disease with no approved treatment, said Pr. John Vissing, Director of the Copenhagen Neuromuscular Center at the National Hospital, Rigshospitalet, in Copenhagen, where the first patient was dosed, and principal investigator of this trial. This experimental treatment represents a new hope for the patients. It is a great motivation to know that the work we are doing here has the potential to make a life-changing difference.

After the first patient dosed in Copenhagen, we are now expecting recruitments at the two other approved clinical sites (Paris, FR, and Newcastle, UK) to complete enrollment of the dose escalation phase (Stage 1) of the study. For Stage 2 (after dose selection), we plan to open additional clinical sites in Europe and in the United States, said Dr. Sophie Olivier, Chief Medical Officer of Atamyo.

About the LGMD-R9 program ATA-100

ATA-100 is a one-time gene replacement therapy for LGMD-R9/2I based on the research of Dr. Isabelle Richard, who heads the Progressive Muscular Dystrophies Laboratory at Genethon (UMR 951 INSERM/Genethon/UEVE). ATA-100 has been awarded Orphan Drug Designation status by the U.S. Food and Drug Administration and the European Medicines Agency.

LGMD2I/R9 is a rare genetic disease caused by mutations in the gene that produces fukutin-related protein (FKRP). It affects an estimated 5,000 people in the US and Europe. Symptoms appear around late childhood or early adulthood. Patients suffer from progressive muscular weakness leading to loss of ambulation. They also are prone to respiratory impairment and myocardial dysfunction. There are currently no curative treatments for LGMDR9.

About Atamyo Therapeutics

Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyos most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD). The name of the company is derived from two words: Celtic Atao which means Always or Forever and Myo which is the Greek root for muscle. Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments. For more information visit http://www.atamyo.com

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Atamyo Therapeutics Announces First Patient Dosed with ATA-100 Gene Therapy in LGMD-R9 Clinical Trial - Business Wire

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

Gene Therapy Cell Culture Media Market Research ReportThe global Gene Therapy Cell Culture Media industry research report provides an in-depth and methodical assessment of regional and global markets, as well as the most current service and product innovations and the global markets predicted size. The Gene Therapy Cell Culture Media research does a complete market analysis to find the major suppliers by integrating all relevant products and services in order to understand the roles of the top industry players in the Gene Therapy Cell Culture Media segment. The global Gene Therapy Cell Culture Media market also provides a thorough analysis of cutting-edge competitor research and new industry advancements, as well as market dynamics, challenges, restrictions, and opportunities, in order to give precise insights and the latest scenarios for appropriate judgments.

The gene therapy cell culture media market was valued at 152.67 million in 2019 and is expected to record a CAGR of 10.87% during the forecast period, 20202029.

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This research study contains a SWOT analysis, significant trends, and a financial evaluation of the Gene Therapy Cell Culture Media and the global markets major competitors. Additionally, the Gene Therapy Cell Culture Media study provides a complete perspective of the Gene Therapy Cell Culture Media market and assists organizations in generating sales by providing a better knowledge of the leading competitors growth plans and competitive environment. This report includes a deep investigation of PEST and the industrys overall dynamics during the anticipated term. The research includes essential results as well as highlights of guidance and significant industry changes in the Gene Therapy Cell Culture Media industry, supporting market leaders in developing new tactics to increase income.

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About Us:StraitsResearch is a leading research and intelligence organization, specializing in research, analytics, and advisory services along with providing business insights & research reports.Contact Us:Email: sales@straitsresearch.comTel: +1 6464807505, +44 203 318 2846

Other Reports:https://www.globenewswire.com/en/news-release/2022/07/26/2486248/0/en/Battery-Recycling-Market-Size-is-projected-to-reach-USD-18-96-Billion-by-2030-growing-at-a-CAGR-of-7-12-Straits-Research.htmlhttps://www.digitaljournal.com/pr/airbag-control-unit-market-share-to-witness-significant-revenue-growth-during-the-forecast-period-2026https://www.digitaljournal.com/pr/light-fidelity-li-fi-devices-market-study-by-latest-research-trends-and-revenue-till-2026-top-key-players-signify-holding-philips-n-v-netherlands-purelifi-uk-oledcomm-francehttps://www.digitaljournal.com/pr/application-modernization-services-industry-report-global-market-manufacturers-outlook-growth-and-forecast-2029

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Gene Therapy Cell Culture Media Market Research Report is Likely to grow at a higher CAGR during the Forecast Period The Colby Echo News - The Colby...

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

BLA Includes Substantial Body of Data from Pivotal Phase 3 and Ongoing Phase 1/2 Studies

If Approved, Would Be 1st Gene Therapy in U.S. for Treatment of Severe Hemophilia A

SAN RAFAEL, Calif., Sept. 29, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Company resubmitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The resubmission incorporates the Company's response to the FDA Complete Response (CR) Letter for valoctocogene roxaparvovec gene therapy issued on August 18, 2020, and subsequent feedback, including two-year outcomes from the global GENEr8-1 Phase 3 study and supportive data from five years of follow-up from the ongoing Phase 1/2 dose escalation study.

BioMarin anticipates an FDA response by the end of October on whether the BLA resubmission is complete and acceptable for review. Typically, BLA resubmissions are followed by a six-month review procedure. However, the Company anticipates three additional months of review may be necessary based on the number of data read-outs that will emerge during the procedure. If approved, valoctocogene roxaparvovec would be the first commercially-available gene therapy in the U.S. for the treatment of severe hemophilia A.

The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received for valoctocogene roxaparvovec in 2017.

In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A. Orphan drug designation is reserved for medicines treating rare, life-threatening or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN on August 24, 2022 and endorsed the recommendation from the European Medicines Agency (EMA) to maintain orphan drug designation, thereby granting a 10-year period of market exclusivity in the European Union.

"We are pleased to reach this point in the development program for valoctocogene roxaparvovec and look forward to working with the FDA with the goal of bringing a potentially transformative therapy to people with severe hemophilia A in the United States," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "This large and robust data set provided in this BLA resubmission shows an encouraging efficacy profile. We remain committed to sharing these data with the public, along with even longer-term data generated through our ongoing clinical trials and any post-approval studies, to further our understanding of AAV gene therapy in severe hemophilia A and of gene therapies more broadly."

The resubmission includes a substantial body of data from the valoctocogene roxaparvovec clinical development program, the most extensively studied gene therapy for severe hemophilia A, including two-year outcomes from the global GENEr8-1 Phase 3 study. The GENEr8-1 Phase 3 study demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate (ABR) and the mean annualized Factor VIII infusion rate. In addition, the data package included supportive evidence from five years of follow-up from the 6e13 vg/kg dose cohort in the ongoing Phase 1/2 dose escalation study. The resubmission alsoincludesaproposedlong-term extension studyfollowingall clinicaltrialparticipantsfor up to 15years, as well astwo post-approval registry studies.

Robust Clinical Program

BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A (Study 270-303). Also ongoing are a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors (Study 270-205).

Safety Summary

Overall, to date, a single 6e13 vg/kg dose of valoctocogene roxaparvovec has been well tolerated with no delayed-onset treatment related adverse events. The most common adverse events (AE) associated with valoctocogene roxaparvovec have occurred early and included transient infusion associated reactions and mild to moderate rise in liver enzymes with no long-lasting clinical sequelae. Alanine aminotransferase (ALT) elevation, a laboratory test of liver function, has remained the most common adverse drug reaction. Other adverse reactions have included aspartate aminotransferase (AST) elevation (101 participants, 63%), nausea (55 participants, 34%), headache (54 participants, 34%), and fatigue (44 participants, 28%). No participants have developed inhibitors to Factor VIII, thromboembolic events or malignancy associated with valoctocogene roxaparvovec.

About Hemophilia A

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (Factor VIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate (Factor VIII 1-5%) or mild (Factor VIII 5-40%) disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week (100-150 infusions per year) or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month (12-48 injections or shots per year). Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visitwww.biomarin.com.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: BioMarin anticipating an FDA response by the end of October on whether the BLA resubmission is complete and acceptable for review, BioMarin's expectations regarding the duration of the review procedure, valoctocogene roxaparvovec being the first commercially-available gene therapy in the U.S. for the treatment of severe hemophilia A, if approved, BioMarin's commitment to sharing longer-term data generated through its ongoing clinical trials and any post-approval studies. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec; additional data from the continuation of the clinical trials of valoctocogene roxaparvovec, any potential adverse events observed in the continuing monitoring of the participants in the clinical trials; the content and timing of decisions by the FDA and other regulatory authorities, including decisions to grant additional marketing registrations based on an EMA license; the content and timing of decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture valoctocogene roxaparvovec for the clinical trials and commercially; and those and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc and ROCTAVIAN is a trademark of BioMarin Pharmaceutical Inc.

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BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

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BioMarin Resubmits Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Severe Hemophilia A to the FDA - PR...

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INTRODUCTION With the increasing number of cell and gene therapies being developed and launched for a wide range of therapeutic areas, these modalities are on their way to become one of the highest valued markets in the biopharmaceutical domain.

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

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

Ryan Cawood, chief scientific officer, WuXi Advanced Therapies.

Cawood began by noting that WuXi Advanced Therapies supports clients throughout their journeys toward developing cell and gene therapies. With the acquisition of the UK-based contract research organization (CRO) Oxgene and its adenoassociated virus (AAV) and lentivirus platforms (known as the TESSA and XLenti platforms/technologies, respectively), WuXi Advanced Therapies now can scale processes up to good manufacturing practice (GMP) manufacturing through to commercial supply.

After describing the work that takes place in different company locations, Cawood focused on the TESSA technology, which provides a plasmid-free alternative for large-scale clinical manufacturing. Most AAV manufacturing is based on plasmid transfection. Not only are such processes expensive, but transfection can occur only at a certain cell density. Adenovirus can be used to manufacture AAV, and such processes are easy to scale up. However, they yield as much adenovirus as AAV, creating major downstream purification issues and raising product-contamination concerns. The goal of the TESSA technology is to use an adenoviral vector to manufacture AAV without contaminating the preparation. In the early phase of the adenoviral life cycle, genes in the AAV helper plasmid are expressed by cells to manufacture both AAV and adenovirus. But in late phases of the cycle, those genes induce cells to make unwanted adenovirus. The TESSA technology is designed to close down all of those late genes.

Cawood described how TESSA technology regulates the promoter that drives expression of structural proteins. The adenovirus titer is determined by the promoter that it represses, so the more the virus tries to make itself, the more it cripples itself. WuXi Advanced Therapies is working on different models of the technology and now has made TESSA rep-cap genes for all of the main serotypes that people work with. Production titers for one particular construct yielded 1 1012 gc/mL. TESSA rep-cap 1, 2, 4, and 5 showed significant improvements in productivity per cell in a suspension-based process for all the serotypes that the company has worked on generally 10-fold more than what is produced using a plasmid system. He also described how data from a cell line developed at WuXi AppTec showed improvement in packaging efficiencies.

Cawood noted that the US Food and Drug Administration (FDA) is increasing pressure on manufacturers to ensure absence of residual contaminants and confirm efficacy. WuXi Advanced Therapies has tested a number of different serotypes and compared the ability of those AAV particles to infect cells with AAVs made from the plasmid process. He illustrated work showing that in some cases, the particles were >10-fold more infectious when produced by TESSA technology than when induced by the plasmid-based process. In an example of scaling up the TESSA technology for AAV6 to 50 L, every cell in the population contained the adenoviral DNA after three days. Around 3% of the particles were able to infect a cell compared with 0.5%0.7% with the plasmid system. Before any purification, 66% of full particles were obtained in the bioreactor. Following purification, the number came to about 102%, and using analytical ultracentrifugation yielded 94.4% full capsids of pure AAV. In an example of scaling up the technology for AAV2, the yields were lower than in the bioreactor but still 20 higher than what was produced by the plasmid-based equivalent. In an alternative model, the company introduced the AAV genome into the chromosome of an HEK293 cell line. The cells were infected with just one TESSA vector, after which AAV was removed with purification.

Other examples illustrated how the technology increased AAV particle yields for all serotypes tested. It increased particle infectivity for a number of the serotypes, is safe and efficient, and removes dependency on transfection. It allows for a number of different operations in bioreactors that simply cant be done in a transfection-based process.

Cawood concluded by noting that the company provides materials that clients can access through evaluation in their own laboratories. WuXi Advanced Therapies also can construct specific TESSA vectors for clients.

Find More OnlineWatch the complete presentation online at https://bioprocessintl.com/sponsored-content/the-future-of-aav-gene-therapy-is-scalable.

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The Future of AAV Gene Therapy Is Scalable - BioProcess Insider