Archive for the ‘Gene Therapy Research’ Category

CHICAGO, Feb. 2, 2021 /PRNewswire/ -- In-depth analysis and data-driven insights on the impact of COVID-19 included in this Europe cell and gene therapy market report.

The Europe cell and gene therapy market is expected to grow at a CAGR of over 23% during the period 20202026.

Key Highlights Offered in the Report:

Key Offerings:

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Europe Cell and Gene Therapy Market Segmentation

Europe Cell and Gene Therapy Market by Product

Europe Cell and Gene Therapy Market by End-user

Europe Cell and Gene Therapy Market by Application

Europe Cell and Gene Therapy Market Dynamics

Cell and gene therapy is revolutionizing the global healthcare segment. Although various new cell and gene therapies are approved, there are various hurdles that limit the penetration of new therapies, such as high cost, multiple regulatory hurdles, and other manufacturing challenges. These cell and gene therapy developers need reliable, efficient, and cost-effective manufacturing services with the flexibility to scale up production as the demand increases. Cell and gene therapy products are very complex, and their manufacturing requires skilled labor, developed infrastructure for limited patients. Such huge investments will affect vendors and contract manufacturing organizations (CMOs) work with companies to overcome these challenges.

Key Drivers and Trends fueling Market Growth:

Europe Cell and Gene Therapy Market Geography

European countries such as Germany, France, the UK, Italy, and Spain play a significant role in the cell and gene therapy market. However, clinical trials and the number of manufacturing facilities are increasing slowly in Europe. Europe has become a major R&D destination for many vendors as the funding for cell and gene therapies is increasing across many European countries. Europe stands next to North America in the global cell and gene therapy market. Initially, Europe led the cell and gene therapy market due to first product approvals. France, Germany, and Italy had a greater contribution globally and in Europe. However, from the past decade, the US has competed and increased its market share globally. Europe stands second in the market, with the increasing prevalence of cancer and rare genetic disorders that are not effectively solved by the conventional therapies are increasing in the region. This increased target population is driving the demand for cell and gene therapy in the region.

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Europe Cell and Gene Therapy Market by Geography

Major Vendors

Other Prominent Vendors

Emerging Investigational Vendors In Europe

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Europe Cell and Gene Therapy Market Size to Reach Revenues of USD 2.9 Billion by 2026 - Arizton - PRNewswire

Biopharma stocks advanced in the week ended Feb. 5, driven higher by a slew of catalysts. Large-cap pharma earnings, vaccine updates, M&A activity, IPO news flow and follow-on offerings all served to lift stocks higher.

In an unexpected move, Jazz Pharmaceuticals PLC (NASDAQ: JAZZ) agreed to buy plant-derived cannabinoid drug maker GW Pharmaceuticals PLC- ADR (NASDAQ: GWPH) for $7.2 billion.

Novavax, Inc. (NASDAQ: NVAX) shares found follow-up buying interest amid vaccine updates. Earnings from large-cap biopharma companies can at best be qualified as mixed. The IPO calendar of the week was heavy, with 10 healthcare stocks making their Wall Street debuts this week.

Conferences

Transplantation and Cellular Therapy, or TCT, Meeting of the American Society of Transplantation and Cellular Therapy and Center for International Blood & Marrow Transplant Research: Feb. 8-12

17th Annual WORLDSymposium: Feb. 8-12

Guggenheim Healthcare Talks: 2021 Oncology Days: Feb. 11-12

2021 ASCO Genitourinary Cancers Symposium: Feb. 11-13

Angiogenesis, Exudation, and Degeneration 2021 conference: Feb. 12-13

PDUFA Dates

The Food and Drug Administration is scheduled to rule on Regeneron Pharmaceuticals Inc's (NASDAQ: REGN) biologic license application for evinacumab, which is being evaluated for the treatment of homozygous familial hypercholesterolemia, an ultra-rare, inherited form of high cholesterol. The decision is expected by Thursday, Feb. 11.

Clinical Readouts

17th Annual WORLDSymposium Presentations

Regenxbio Inc (NASDAQ: RGNX): interim results from the Phase 1/2 clinical trial of RGX-121 for the treatment of mucopolysaccharidosis type II (Monday, Feb. 8, at 10:06 a.m.)

Orchard Therapeutics PLC ADR (NASDAQ: ORTX): updated Phase 1/2 data for ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA; Phase 1/2 data for ex vivo hematopoietic stem cell gene therapy for mucopolysaccharidosis type I (Tuesday, Feb. 9)

Protalix Biotherapeutics Inc (NYSE: PLX): one year of treatment data from BRIDGE, a phase 3 open-label study of pegunigalsidase alfa to treat patients with Fabry disease (Wednesday, Feb. 10, at 2:30-3:30 p.m.)

Story continues

Avrobio Inc (NASDAQ: AVRO): new phase 1/2 data for AVR-RD-02 ex-vivo lentiviral vector, autologous gene therapy for Gaucher disease (Thursday, Feb. 11, 2:30-3:30 p.m.); updated results from a phase 1/2 study of hematopoietic stem cell gene therapy for cystinosis (Friday, Feb. 12, 11 a.m.); updated Phase 2 data for AVR-RD-01 in Fabry disease (Friday, Feb. 12)

Abeona Therapeutics Inc (NASDAQ: ABEO): updated results from Phase 1/2 study of ABO-101 gene therapy for mucopolysaccharidosis IIIB and Phase 1/2 study of ABO-102 gene therapy for mucopolysaccharidosis IIIA (Friday, Feb. 12)

Denali Therapeutics Inc (NASDAQ: DNLI): data from Phase 1/2 study of DNL310 in mucopolysaccharidosis II patients

Related Link: Attention Biotech Investors: Mark Your Calendar For February PDUFA Dates

TCT Meeting Presentations

Gamida Cell Ltd (NASDAQ: GMDA): results of the Phase 1 study of omidubicel versus standard myeloblative umbilical cord blood transplantation in patients with hematologic malignancies undergoing allogeneic bone marrow transplant (Tuesday, Feb. 9 at 4:45-5 p.m.)

Equillium Inc (NASDAQ: EQ): interim data from the Phase 1b/2 EQUATE study of itolizumab in acute graft-versus-host disease (Friday, Feb. 12, 3 p.m.)

Takeda Pharmaceutical Co Ltd (NYSE: TAK): Phase 3 data for TAK-620 in the treatment of transplant recipients with refractory/resistant cytomegalovirus infections (Friday, Feb. 12)

ASCO Genitourinary Cancers Symposium Presentations

Clovis Oncology Inc (NASDAQ: CLVS): Pharmacokinetics and safety data from the phase 1b RAMP study evaluating rucaparib plus enzalutamide in patients with metastatic castration-resistant prostate cancer (Thursday, Feb. 11, 8 a.m.)

Infinity Pharmaceuticals Inc. (NASDAQ: INFI): Preliminary analysis of a phase 2 data for eganelisib in combination with and Bristol-Myers Squibb Co's (NYSE: BMY) Opdivo compared to Opdivo monotherapy in patients with advanced urothelial carcinoma (Thursday, Feb. 11)

View more earnings on IBB

BioXcel Therapeutics Inc (NASDAQ: BTAI): updated Phase 1b/2 data for BXCL701 in combination with Merck & Co., Inc.'s (NYSE: MRK) Keytruda in men with metastatic castration-resistant prostate cancer (Thursday, Feb. 11, 8 a.m.)

Angiogenesis, Exudation, and Degeneration 2021 Conference Presentation

Kodiak Sciences Inc (NASDAQ: KOD): Clinical data on its investigational therapy KSI-301 in wet age-related macular degeneration and retinal vascular diseases (Saturday, Feb. 13 at 8:30 a.m.)

Earnings

Monday

Corcept Therapeutics Incorporated (NASDAQ: CORT) (after the market close)Enanta Pharmaceuticals Inc (NASDAQ: ENTA) (after the market close)

Tuesday

Incyte Corporation (NASDAQ: INCY) (before the market open)Deciphera Pharmaceuticals Inc (NASDAQ: DCPH) (before the market open)SurModics, Inc. (NASDAQ: SRDX) (before the market open)

Wednesday

Polypid Ltd (NASDAQ: PYPD) (before the market open)Veru Inc (NASDAQ: VERU) (before the market open)Vanda Pharmaceuticals Inc. (NASDAQ: VNDA) (after the market close)Pacific Biosciences of California Inc (NASDAQ: PACB) (after the market close)Fluidigm Corporation (NASDAQ: FLDM) (after the market close)Exelixis, Inc. (NASDAQ: EXEL) (after the market close)Aethlon Medical, Inc. (NASDAQ: AEMD) (after the market close)

Thursday

Karyopharm Therapeutics Inc (NASDAQ: KPTI) (before the market open)Myovant Sciences Ltd (NYSE: MYOV) (before the market open)Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) (before the market open)Applied Genetic Technologies Corp (NASDAQ: AGTC) (before the market open)Alkermes Plc (NASDAQ: ALKS) (before the market open)Laboratory Corp. of America Holdings (NYSE: LH) (before the market open)AVITA Medical Inc (NASDAQ: RCEL) (after the market close)Aytu Bioscience Inc (NASDAQ: AYTU) (after the market close)Otonomy Inc (NASDAQ: OTIC) (after the market close)Illumina, Inc. (NASDAQ: ILMN) (after the market close)Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) (after the market close)

Friday

ImmunoGen, Inc. (NASDAQ: IMGN) (before the market open)

IPOs

Adagene, Inc., a China-based, clinical-stage biopharmaceutical company developing antibody-based cancer immunotherapies, has filed to offer 7.354 million ADSs, representing 9,1925 million ordinary shares, in an initial public offering. The company expects to price the offering in the $17-$19 range, and has applied for listing its ADSs on the Nasdaq under the ticker symbol ADAG.

French clinical-stage biotech Biophytis S.A., which focuses on development of therapeutics that slow the degenerative processes associated with aging, is proposing to offer 1.2 million ADSs in an IPO. Each ADS represents the right to receive 10 ordinary shares. The offering price is expected to be between $15 and $18 per ADS. The company has applied to list its ADSs on the Nasdaq under the ticker symbol BPTS.

Longeveron LLC, a clinical stage biotech developing cellular therapies for specific aging-related and life-threatening conditions, is offering 2.73 million shares at an estimated price range of $10-$12. The Miami, Florida-based company has applied for listing the shares on the Nasdaq under the ticker symbol LGVN.

Durham, North Carolina-based Bioventus Inc. is planning a 7.53-million-share IPO, with the offering estimated to be priced between $16 and 18 apiece. The medical device company has applied for listing its shares on the Nasdaq under the ticker symbol BVS.

Related: Pfizer Expects COVID-19 Vaccine To Bring $15B In Revenue This Year

Photo by National Cancer Institute on Unsplash

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The Week Ahead In Biotech (Feb. 7-13): Regeneron FDA Decision, Earnings, IPOs and Conference Presentations In The Spotlight - Yahoo Finance

New York, Feb. 05, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030" - https://www.reportlinker.com/p06020737/?utm_source=GNW Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods (such as genome sequencing), and genome editing and manipulation technologies (such as molecular switches), have enabled the development of novel therapy development tools / platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

SCOPE OF THE REPORT The Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy (Ex vivo and In vivo), Type of Vectors used (Adeno Associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Plasmid DNA, Retrovirus and Others), Target Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Dermatological Disorders, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebellar, Intradermal, Intramuscular, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal and Others), and Key Geographical Regions (US, EU5 and rest of the world): Industry Trends and Global Forecasts, 2020-2030 report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

Amongst other elements, the report features: - A detailed review of the overall market landscape of gene therapies and gene editing therapies, including information on phase of development (marketed, clinical, preclinical and discovery) of pipeline candidates, key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene, therapeutic approach (gene augmentation, oncolytic viral therapy and others), type of gene therapy (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any). - A detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. - An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors. - A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies. - Detailed profiles of marketed and late stage (phase II/III and above) gene therapies, including development timeline of the therapy, information on the current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, as well as details related to the developer company. - An elaborate discussion on the various commercialization strategies that can be adopted by drug developers across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing. - A review of the various emerging technologies and therapy development platforms that are being used to design and manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates. - An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2016. The analysis assesses several relevant parameters associated with the patents, including type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation. - A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area. - An analysis of the investments made at various stages of development in companies that are focused in this area, between 2015-2020, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings. - A detailed geographical clinical trial analysis of completed, ongoing and planned studies of numerous gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population. - An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies. - An analysis of the big biopharma players engaged in this domain, featuring a heat map based on parameters, such as number of gene therapies under development, funding information, partnership activity and strength of patent portfolio. - An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. - A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors. - A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2020-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vectors used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals: - Adam Rogers (CEO, Hemera Biosciences) - Al Hawkins (CEO, Milo Biotechnology) - Buel Dan Rodgers (Founder & CEO, AAVogen) - Christopher Reinhard (CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)) - Michael Triplett (CEO, Myonexus Therapeutics) - Robert Jan Lamers (CEO, Arthrogen) - Ryo Kubota (CEO, Chairman & President, Acucela) - Tom Wilton (CBO, LogicBio Therapeutics) - Jeffrey Hung (CCO, Vigene Biosciences) - Cedric Szpirer (Executive & Scientific Director, Delphi Genetics) - Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) - Molly Cameron (Corporate Communications Manager, Orchard Therapeutics)

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 METHODOLOGY The 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 will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include - Annual reports - Investor presentations - SEC filings - Industry databases - News releases from company websites - Government policy documents - Industry analysts views

While the focus has been on forecasting the market over the coming decade, the report also provides our independent view on various emerging trends 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 ANSWERED - Who are the leading industry players engaged in the development of gene therapies? - How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products? - Which types of vectors are most commonly used for effective delivery of gene therapies? - What are the key regulatory requirements for gene therapy approval, across various geographies? - Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development? - What are the different pricing models and reimbursement strategies currently being adopted for gene therapies? - What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies? - Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development? - What are the key value drivers of the merger and acquisition activity in the gene therapy industry? - Who are the key stakeholders that have actively made investments in the gene therapy domain? - Which are the most active trial sites (in terms of number of clinical studies being conducted) related to this domain? - How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 800 gene therapies and gene editing therapies that are currently approved or are in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene, type of gene therapy (ex vivo and in vivo), therapeutic approach (gene augmentation, oncolytic viral therapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and therapeutic approach.

Chapter 7 provides a detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe and the Asia-Pacific region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the companys patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that can be adopted by therapy developers across key commercialization stages, including prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2020.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each drug profile provides information on the current developmental status of the drug, its route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, technology, patent portfolio, clinical trials and collaborations (if any).

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic disorders, metabolic disorders, nervous system disorders, oncological disorders and ophthalmic diseases.

Chapter 13 provides an overview of the various patents that have been filed / granted in relation to gene therapy and gene editing technologies. It also features a detailed analysis, highlighting the prevalent trends related to type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation. For the purpose of this analysis, we have taken into consideration patents that have been filed / granted since 2016.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2015 and 2020, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents a detailed, geographical clinical trial analysis of completed, ongoing and planned studies focused on gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 highlights top big biopharma players engaged in the field of gene therapy, featuring a heat map analysis based on several parameters, including therapeutic area, type of vector used, therapeutic approach and type of gene therapy.

Chapter 19 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 20 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2030. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vector used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world).

Chapter 21 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 22 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.

Chapter 23 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 24 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Christopher Reinhard (CEO & Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)), Michael Triplett (CEO, Myonexus Therapeutics), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (CEO, Chairman & President, Acucela), Tom Wilton (CBO, LogicBio Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) and Molly Cameron (Corporate Communications Manager, Orchard Therapeutics). In addition, a brief profile of each company has been provided.

Chapter 25 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 26 is an appendix, which contains a list of companies and organizations mentioned in this report.Read the full report: https://www.reportlinker.com/p06020737/?utm_source=GNW

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Originally posted here:
Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key...

First participant dosed in the RESOLUTESM trial, a Phase 1/2 dose-escalation study of SPK-3006

Enrollment of approximately 20 total study participants is ongoing

PHILADELPHIA, Feb. 01, 2021 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the dosing of the first participant in the Phase 1/2 RESOLUTESM trial of SPK-3006, an investigational liver-directed adeno-associated viral (AAV) vector gene therapy for late-onset Pompe disease (LOPD), a rare, inherited lysosomal storage disorder.

Dosing the first participant in the Phase 1/2 RESOLUTE trial of investigational SPK-3006 for late-onset Pompe disease is an important milestone and first step to what we hope will ultimately allow us to bring an innovative gene therapy to these patients, said Gallia G. Levy, M.D., Ph.D., chief medical officer of Spark Therapeutics. We are deeply appreciative of the ongoing collaboration of the Pompe disease community as we continue to enroll participants in this Phase 1/2 study.

The RESOLUTE trial is an open-label Phase 1/2, dose-escalation gene transfer study designed to evaluate the safety, tolerability and efficacy of a single intravenous infusion of investigational SPK-3006, an AAV vector-based gene therapy, developed in collaboration with Genethon, in adults with clinically moderate LOPD currently receiving enzyme replacement therapy. The study is expected to enroll approximately 20 participants receiving the investigational gene therapy in sequential, dose-level cohorts. Additional details are available on ClinicalTrials.gov (NCT04093349).

We are honored to have the first participant dosed in this clinical trial, which we hope will lead us to introduce a novel therapeutic option for patients living with late-onset Pompe disease, said Principal Investigator Tahseen Mozaffar, M.D., University of California Irvine Health.

The International Pompe Association has been proud to collaborate with Spark Therapeutics to enhance the Pompe disease communitys understanding of gene therapy research, said Tiffany House, International Pompe Association Board Chairman. We look forward to the progress in the Phase 1/2 RESOLUTE trial, as well as the ongoing work aimed at developing gene therapies that have the potential to help individuals living with genetic diseases.

Pompe disease is a rare, inherited lysosomal storage disorder. It is a progressive, often life-limiting disease caused by the buildup of a complex sugar, glycogen, in the bodys cells. Mutations in the gene encoding acid alpha-glucosidase (GAA) result in deficiencies of the GAA enzyme and limit the breakdown of glycogen. For patients living with LOPD, the respiratory system, locomotion and maintenance of gait are the most critically impacted. These symptoms commonly result in patients becoming wheelchair bound and requiring respiratory support, which may result in reduced life-expectancy.

About SPK-3006 for Pompe diseaseSPK-3006is an investigational liver-directed AAV gene therapy for the potential treatment of late-onset Pompe disease (LOPD).SPK-3006has been engineered to produce a modified enzyme (secretable GAA) that is produced by the liver, which may result in sustained GAA plasma levels and could potentially provide greater uptake in muscle tissue. The transgene integrates technologies designed at and licensed from Genethon, where the in-vivo proof of concept in pre-clinical models was demonstrated. Spark Therapeutics retains global commercialization rights toSPK-3006.

About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.

Media Contact:Kevin Giordanocommunications@sparktx.com(215) 294-9942

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Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease - BioSpace

- First potentially disease-modifying gene therapy for GM2 gangliosidosis to enter clinical studies

- Expect to continue patient identification, screening, and enrollment in Stage 1 of the study throughout 2021

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 03, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the first patient with infantile Tay-Sachs disease has been dosed in a Phase 1/2 trial evaluating AXO-AAV-GM2,an investigational gene therapy for the treatment of GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff disease.

We are proud to bring the first potentially disease-modifying treatment for GM2 gangliosidosis to the clinic, which is a milestone for Sio, for patients, and for the field of gene therapy, said Gavin Corcoran, M.D., Chief R&D Officer of Sio. By restoring lysosomal enzyme activity where it is essential, AXO-AAV-GM2 has the potential to change the course of this disease and help affected children attain and retain important neuro-developmental milestones. The prior expanded access study of AXO-AAV-GM2 provided important proof-of-concept data and we look forward to the results of the first stage of our study as we strive to develop a treatment for children suffering from this rapidly progressive and fatal disease.

Florian Eichler, M.D., Director of the Leukodystrophy Service of the Center for Rare Neurological Diseases at Massachusetts General Hospital, and principal investigator, added, To date, the current GM2 treatment landscape is limited to supportive care, underscoring the significant need for new treatment options to address this devastating pediatric neurodegenerative disease. AXO-AAV-GM2 has significant potential to address the clinical manifestations of both Tay Sachs and Sandhoff diseases, and as a result, the dosing of this patient represents a major step forward for this therapy. We look forward to evaluating the results of this study and advancing the first potentially disease-modifying treatment option for patients with GM2.

The Phase 1/2 study (NCT04669535) is an open-label, two-stage clinical trial designed to evaluate safety and dose-escalation (Stage 1) and safety and efficacy (Stage 2) of surgical delivery of AXO-AAV-GM2 directly to the brain and spinal cord of pediatric participants with both infantile and juvenile GM2 gangliosidosis. AXO-AAV-GM2 has been granted Orphan Drug and Rare Pediatric Disease Designation by the FDA and is the first investigational gene therapy to enter clinical trials for GM2 gangliosidosis. In 2019, clinical evidence from two patients under an expanded access IND found that treatment with AXO-AAV-GM2 was generally well-tolerated and associated with improved bioactivity outcomes.

The families of children with Sandhoff and Tay-Sachs diseases show incredible bravery in choosing to participate in investigational studies of novel therapeutics like AXO-AAV-GM2. We share their hope that this treatment can halt or reverse the otherwise inexorable course of these tragic diseases, said Terence R. Flotte, MD, Professor of Pediatrics and Dean at the University of Massachusetts Medical School and principal investigator of the trial.

GM2 gangliosidosis is a set of rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes that encode the enzyme -Hexosaminidase A. It can be categorized into two distinct diseases, Tay-Sachs disease, which results from a mutation in the gene encoding the alpha subunit of the -Hexosaminidase A enzyme (HEXA), and Sandhoff disease, which results from a mutation in the gene encoding the beta subunit of the -Hexosaminidase A enzyme (HEXB). Children affected by GM2 gangliosidosis suffer from a progressively debilitating disease course and reduced life expectancy.

Sue Kahn, Executive Director of National Tay-Sachs & Allied Diseases Association(NTSAD), added, This news represents the culmination of many years of work to advance this research and immense support from the GM2 community, and it underscores the dire need for new treatment options capable of providing meaningful benefits to patients and families. We are extremely excited by the progress Sio has made and the hope it brings to our community.

Sio aims to advance the program through strategic partnerships with leading research organizations. The Company has a partnership with Viralgen, an AskBio subsidiary, to support AAV-based vector manufacturing of clinical trial material for the registrational study. Additionally, through an existing genetic testing collaboration with Invitae, ongoing partnership with GM2 gangliosidosis patient groups, and collaboration with leading academic researchers at the University of Massachusetts Medical School and Massachusetts General Hospital, Sio has begun patient identification and screening activities for the ongoing clinical study.

About AXO-AAV-GM2

AXO-AAV-GM2 is an investigational gene therapy for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA (leading to Tay-Sachs disease) or HEXB (leading to Sandhoff disease) genes that encode the two subunits of the -hexosaminidase A (HexA) enzyme. These genetic defects lead to progressive neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims to restore HexA function by introducing a functional copy of the HEXA and HEXB genes via delivery of two co-administered AAVrh8 vectors.

About Sio Gene TherapiesSio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry, and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

In 2018, Sio licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as expect potentially, and potential, and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief Commercial Officerinvestors@siogtx.com

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Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)...

Vancouver, British Columbia, Feb. 04, 2021 (GLOBE NEWSWIRE) -- The Global Regenerative Medicine Market is predicted to attain a market valuation of USD 6.49 billion by 2027, growing at a CAGR of 9.3% throughout the estimated period, according to a recent analysis by Emergen Research. Targeted therapy of specific disease indication and chronic illnesses are anticipated to alter the dynamics of the healthcare field. The escalating prevalence of chronic health conditions and increasing patient pool of geriatric populace coupled with neurodegenerative disorders, cancers, orthopedic, and other age-related conditions are further bolstering the industrys expansion.

The numerous applications and subsequent advancements in tissue engineering, gene therapy, nanotechnology, and stem cells research are foreseen to boost the scope of regenerative medicine. 3D printing is playing a pivotal role in stem cells research as it allows for the easy restoration of structural and functional properties.

North America is predicted to occupy a significant share of the market in the projected timeframe and the growth can be attributed to the increasing number of academic institutions and universities extensively exploring regenerative medicine approaches based on stem cells.

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For the purpose of this report, Emergen Research has segregated the Global Regenerative Medicine Market on the basis of product, therapeutic category, application, and region:

Product Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

Therapeutic Category Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

Application Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

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Regional Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

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Regenerative Medicine Market to be Valued at USD 6.49 Billion by 2027 | The Escalating Burden of Chronic Diseases and Genetic Aberrations will be the...

January was a busy month for the US Food and Drug Administrations precision medicine efforts, as the agency produced guidance on ASO drugs for patients with debilitating or life-threatening genetic disorders and guidance on manufacturing considerations for certain cellular and gene therapy products during the COVID-19 pandemic.

The agency first issued a draft guidance to facilitate the development of individualized antisense oligonucleotide (ASO) drugs for patients with severely debilitating or life-threatening genetic disorders (ASO Guidance). The Food and Drug Administration (FDA) also issued a guidance, with immediate effect, on manufacturing considerations for licensed and investigational cellular and gene therapy products during the COVID-19 public health emergency (Manufacturing Guidance). Sponsors investigating or marketing these products should pay special attention to the discussion in these documents, as FDA outlines its approach to COVID-19 and development considerations with respect to these personalized therapies.

The Manufacturing Guidance supplements FDAs June 2020 guidance on Good Manufacturing Practice Considerations for Responding to COVID-19 Infection in Employees in Drug and Biological Products Manufacturing. However, because cell and gene therapy (CGT) manufacturers may face special challenges, FDA recommends that CGT manufacturers perform risk assessments to identify, evaluate, and mitigate factors that may allow for the transmission of SARS-CoV-2 through CGT products. Any plans should take into account FDAs view that allogeneic products may be associated with a higher risk of infection compared to autologous products.

FDA specifically recommends the following:

As always, any adopted risk assessment and mitigation strategies must be documented and approved by the manufacturers quality unit, should include scientific justification and literature references, and should be submitted to FDA.

Turning away from the current COVID-19 crisis, FDA indicated that it is also looking ahead to the continued advancement of personalized therapies, issuing the ASO Guidance to assist sponsor investigators in the development of individualized ASO products for severely debilitating or life-threatening genetic diseases that are tailored to a patients specific genetic variant. As noted by FDA, the ASO Guidance is targeted to academic investigators, who may be less familiar with FDAs requirements and less experienced in interacting with FDA.

While the specific impetus for this guidance is unclear, assumedly FDA is receiving more inquiries regarding individualized ASO drugs from investigators, patients, or those acting on their behalf. Regardless of the reason, healthcare institutions where ASO products are used should familiarize themselves with FDAs requirements and processes to ensure that any use of an investigational ASO product accords with FDAs regulations. It will also be important that manufacturers supporting the use of ASO products or that later intend to work with ASO product investigators ensure that programs comply with FDAs regulations via contractual agreements and, as appropriate, due diligence.

For these programs, FDA recommends the following:

The ASO Guidance is likely a first step in the development of individualized therapies. As stated by FDA, the agency is optimistic that development of [ASO] individualized drug products may spur gene sequencing that leads to the development of additional individualized drug products. Accordingly, through the ASO Guidance, FDA aims to determine the most effective and efficient way to bring personalized drugs to patients, while ensuring the right risk-benefit balance.

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FDA Issues More Guidance on Gene and Cell Therapy Products - JD Supra

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically improve the lives of patients with neurodegenerative diseases, announced today that it has received $11.6 million from the closing of the sale of Arvelle Therapeutics to Angelini Pharma. Per the terms of the sale, additional payments to Sio Gene Therapies Inc. are expected over time, including a payment of approximately $4.8 million by mid-2021 upon marketing approval of cenobamate by the European Medicines Agency (EMA).

About Sio Gene Therapies

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as expect and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding amounts to be received and income tax liabilities associated with the sale of its stake in Arvelle Therapeutics, as well as the duration of its ability to support its development programs, are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

David NassifSio Gene Therapies Inc.Chief Financial Officer and General Counsel(646) 677-6770investors@siogtx.com

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Sio Gene Therapies Announces Receipt of $11.6 Million from Closing of the Sale of Arvelle Therapeutics - BioSpace

REDWOOD CITY, Calif., Feb. 02, 2021 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc.(Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the publication of preclinical data on ADVM-022 intravitreal (IVT) gene therapy in Translational Vision Science & Technology (TVST), an official journal of the Association for Research in Vision and Ophthalmology (ARVO). ADVM-022 is in clinical trials for wet AMD and DME, and this preclinical study in NHPs is the longest safety and expression study to date, with measurements out 30 months following a single IVT injection.

There is a growing body of both clinical and preclinical data demonstrating durable efficacy and favorable safety profile following a single IVT injection of ADVM-022, said Laurent Fischer, M.D., chief executive officer at Adverum Biotechnologies. In this preclinical study, we saw long-term, sustained aflibercept expression out to 30 months following ADVM-022. The levels of aflibercept were sustained at therapeutic levels, with no measurable adverse effects on normal retinal structure and function. We are excited to work on developing ADVM-022 as a potential one and done IVT injection therapy that may dramatically reduce the treatment burden for patients living with wet AMD and DME.

Szilrd Kiss, M.D., academic retina specialist, added, Currently, patients with wet AMD are treated with frequent anti-VEGF intravitreal injections to maintain their vision. One of the highest priorities in research today is to develop therapies that extend the duration of efficacy following treatment, enabling patients to preserve sight for months or years following treatment. The preclinical data on ADVM-022 demonstrate long-term safety and aflibercept expression following a single intravitreal injection of this novel IVT injection gene therapy. We are excited to continue to assess ADVM-022 as it demonstrates the potential to improve real-world visual outcomes over intermittent anti-VEGF injections for patients living with wet AMD.

The publication, titled Long-Term Safety Evaluation of Continuous Intraocular Delivery of Aflibercept by the Intravitreal Gene Therapy Candidate ADVM-022 in Nonhuman Primates, reported the following:

The full online publication can be accessed from the TVST website.

About ADVM-022 Gene TherapyADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection (IVT), designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve vision outcomes for patients with wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME).

In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration granted Fast Track designation for ADVM-022 for the treatment of wet AMD.

Adverum is currently evaluating ADVM-022 in the OPTIC Phase 1 clinical trial in patients with wet AMD and the INFINITY Phase 2 trial in patients with DME at 2 x 10^11 vg/eye and 6 x 10^11 vg/eye doses. The Company plans to begin a pivotal trial in mid-2021 for ADVM-022 in wet AMD.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is advancing the clinical development of its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with wet age-related macular degeneration and diabetic macular edema. For more information, please visit http://www.adverum.com.

Forward-looking StatementsStatements contained in this press release regarding the events or results that may occur in the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include but are not limited to statements regarding: the potential for ADVM-022 in treating patients with wet AMD and DME; the potential efficacy and safety of ADVM-022 in wet AMD and DME; Adverums expectations as to its plans to advance ADVM-022 in wet AMD by initiating a pivotal trial mid-2021. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include risks inherent to, without limitation: Adverums novel technology, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; the results of early clinical trials not always being predictive of future results; the potential for future complications or side effects in connection with use of ADVM-022. Risks and uncertainties facing Adverum are described more fully in Adverums Form 10-Q filed with theSEConNovember 5, 2020under the heading Risk Factors. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Adverum Biotechnologies Announces Publication of Preclinical Long-Term Safety Data on ADVM-022 IVT Gene Therapy - BioSpace

NEW YORK, Feb. 3, 2021 /PRNewswire/ -- Aruvant Sciences, a private company focused on developing gene therapies for rare diseases,today announced that the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to ARU-1801, a one-time investigational gene therapy for sickle cell disease (SCD).

"PRIME designation from EMAhighlightsthe importance of ARU-1801, administeredwith only reduced intensity conditioning,for the treatment ofindividuals with severe sickle cell disease,"said Will Chou, M.D., Aruvant chief executiveofficer."With PRIME,we will be able to work closely with EMAon the development of ARU-1801, with the goal of rapidlybringingthispotential cure toSCD patients in Europe."

PRIME was created by the European Medicines Agency (EMA) to enhance support for the development of innovative medicinesthat target an unmet medical needand demonstratethe potential to achieve relevant clinical outcomes on morbidity, mortality or underlying disease progression. The PRIME designation offersenhanced early interaction with companiesdeveloping promising medicines, to optimize development plans and speed up evaluation. PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments, or that benefit patients without treatment options.

ARU-1801 was designated PRIME status based on clinical data from the MOMENTUMstudy, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, that demonstrate meaningful,durable reductions in disease burden.

About ARU-1801ARU-1801 is designed to address the limitations of current curative treatment options, such as low donor availability and the risk of graft-versus-host disease (GvHD) seen with allogeneic stem cell transplants. Unlike investigational gene therapies and gene editing approaches which require fully myeloablative conditioning, the unique characteristics of ARU-1801 allow it to be given with reduced intensity conditioning ("RIC"). Compared to myeloablative approaches, the lower dose chemotherapy regimen underlying RIC has the potential to reduce not only hospital length of stay, but also the risk of short- and long-term adverse events such as infection and infertility. Preliminary clinical data from the MOMENTUMstudy, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, demonstrate continuing durable reductions in disease burden.

The MOMENTUM StudyAruvant is conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time potentially curative investigational gene therapy for patients with SCD. This Phase 1/2 study is currently enrolling participants, and information may be found at http://www.momentumtrials.comwhich includes a patient brochure, an eligibility questionnaireand information for healthcare providers.

About Aruvant SciencesAruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talentedteamwith extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an activeresearchprogram with a lead product candidate, ARU-1801, in development for individuals suffering fromsickle cell disease(SCD). ARU-1801, an investigational lentiviral gene therapy, is being studied in aPhase 1/2 clinical trial,the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. For more information on the clinical study, please visit http://www.momentumtrials.comand for more on the company, please visitwww.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciencesand on Instagram @Aruvant_Sciences.

About RoivantRoivant's mission is to improve the delivery of healthcare to patients by treating every inefficiency as an opportunity. Roivant develops transformative medicines faster by building technologies and developing talent in creative ways, leveraging the Roivant platform to launch Vants nimble and focused biopharmaceutical and health technology companies. For more information, please visit http://www.roivant.com.

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Aruvant Announces the European Medicines Agency (EMA) Granted Priority Medicines (PRIME) Designation to ARU-1801 for the Treatment of Sickle Cell...

Nottingham, UK 2nd February 2021 Life Science Newswire Albumedix Ltd. (Albumedix), an enabler of advanced therapies and the world leader in recombinant human albumin (rAlb), announced today that they have entered into a collaboration agreement with the Cell and Gene Therapy Catapult (CGT Catapult) to investigate the use of Albumedix proprietary albumin-based solutions for advanced therapy applications, including viral vectors manufacturing.

This agreement reflects the continued efforts of Albumedix to engage with the industry and expand upon its knowledge in the field, and the CGT Catapults mission to drive the growth of the UK cell and gene therapy industry by helping cell and gene therapy organisations across the world translate early-stage research into commercially viable and investable therapies.

Albumedix Chief Executive Officer; Jonas Skjdt Mller commented on the collaboration:

With a mission to empower excellence in advanced therapies, we are committed to continuously playing an integral part in enabling our customers to advance in a fast-moving industry. For us to do so, we continuously look at other industry leaders to establish collaborations. Continuing to learn from each other allows innovation in the market to advance, and Albumedix to support our customers with in-depth knowledge of how rAlb can uniquely benefit their therapies. Cell and Gene Therapy Catapult is the ideal partner; located in our own backyard here in the UK and with incredible skills, knowledge and drive to advance the cell and gene therapy industry we are excited about this collaboration.

Matthew Durdy, Chief Executive Officer at Cell and Gene Therapy Catapult commented:

The opportunity to collaborate with a leading company like Albumedix Ltd in order to assess and drive the potential of their latest technology is something that we embrace. The prospect of improving manufacturing of viral vectors such as Adeno-associated virus (AAV) through this exciting technology is something which could significantly benefit and advance the wider cell and gene therapy field.

Activities under this agreement will be carried out both at the CGT Catapult facility in Braintree and at Albumedix new R&D center, with state-of-the-art laboratories specifically designed for the process optimization, characterization and formulation development of advanced therapies.

Get in touch with Albumedix today by emailing communications@albumedix.com to learn more about their Recombumin range of world leading recombinant human albumin products. Reach out to Cell and Gene Therapy Catapult by emailing communications@ct.catapult.org.uk to learn more about how they can help your organisation to translate early stage research into commercially viable and investable therapies.

About Albumedix Dedicated to Better Health

Albumedix is a science-driven, life-science company focused on enabling the creation of superior biopharmaceuticals utilizing our recombinant human albumin products. We believe in empowering excellence to enable advanced therapies and facilitate otherwise unstable drug candidates reach patients worldwide. We are proud to be recognized as the world leader in recombinant human albumin with products and technologies used in clinical and marketed drugs by pharmaceutical and medical device companies worldwide. Headquartered in Nottingham, England with more than 100 people all committed to improving patient quality of life. We are just as passionate about albumin and albumin-enabled therapies today as we were when we started more than 35 years ago. For more information, please reach out to Albumedix at communications@albumedix.com or visit http://www.albumedix.com

About Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

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Albumedix enters into collaboration agreement with Cell and Gene Therapy Catapult - PharmiWeb.com

London, Feb. 02, 2021 (GLOBE NEWSWIRE) -- Roots Analysis has announced the addition of Gene Therapy Market (4th Edition), 2020-2030 report to its list of offerings.

Success of approved gene therapies has resulted in a surge in interest of biopharmaceutical developers in this rapidly evolving domain. Presently, the ability of gene therapies to treat diverse disease indications is considered among the most prominent drivers of this market. In addition, promising clinical results of pipeline candidates are anticipated to draw in more investments to support product development initiatives.

To order this 720+ page report, which features 220+ figures and 375+ tables, please visit this link

Key Market Insights

Around 800 gene therapies are currently being developed across different stages Apart from 10 approved products, most of the aforementioned therapies (65%) are in the early stages of development (discovery / preclinical), while the rest are being evaluated in clinical trials. It is worth mentioning that more than 40% of clinical stage candidates are intended for the treatment of oncological disorders.

Over 65% of innovator companies focused on gene therapy development, are based in North AmericaInterestingly, more than 75 players based in the same region, are start-ups, while over 35 are mid-sized players, and 10 are large and very large firms. Since the majority of gene therapy developers are headquartered in the US, it is considered a key R&D hub for such advanced therapy medicinal products.

There are 400+ registered gene therapy focused clinical trials, worldwideClinical research activity, in terms of number of trials registered, is reported to have increased at a CAGR of 12% during the period 2015-2020. Of the total number of trials, close to 25% have already been completed, and 35% claim to be actively recruiting.

USD 25.4 billion has been invested by both private and public investors, since 2015So far, a significant proportion of the capital raised has been through secondary offerings (USD 12.9 billion). On the other hand, around USD 5 billion was invested by venture capital investors, representing 20% of the total amount.

Close to 20,000 patents have been filed / published related to gene therapies, since 2016Around 30% of the total number of applications were related to gene editing-based therapies, while the remaining were associated with gene therapies. Further, majority of the patent assignees were industry players, however, the contribution of non-industry players in the overall patent filing activity has increased considerably (CAGR of 16%), over the past few years.

There have been several mergers and acquisitions in this market during the period 2015-2019 In fact, M&A activity is reported to have increased at a CAGR of more than 40%. Key drivers of the acquisitions mentioned in the report include, therapeutic area expansion, access to a novel technology / platform, drug class consolidation and drug class expansion.

North America and Europe are anticipated to capture over 90% of the market share, in terms of sales revenues, in 2030In vivo gene therapies currently represent a significant share of the market, and this trend is unlikely to change in the foreseen future, as several such candidates are being evaluated in late stages. In addition, more than 130,000+ patients are projected to use gene therapies in 2030 and the demand for gene therapies is expected to grow at an annualized rate of 29% and 31% during the periods 2020-2025 and 2025-2030, respectively.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 14.6 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom, gene therapies exhibit the potential to become a promising alternative for the treatment of genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research includes brief profiles of key players (listed below) engaged in the development of gene therapies; each profile features an overview of the therapy, current development status, clinical trials and its results (if available), target indication, route of administration, and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapies-market/268.html or email sales@rootsanalysis.com

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The gene therapy market is projected to be worth USD 14.6 billion in 2030, growing at a CAGR of 30%, over the next decade, claims Roots Analysis -...

Global Retinal Gene Therapy Market: Overview

The retinal gene therapy market is estimated to expand at an exponential growth rate. For the use of gene therapy, retina is considered a highly desirable target as it an irreplaceable part of a body. The global retinal gene therapy market is likely to be influenced by the promise its holds for the treatment of various forms of inherited and non-inherited blindness. Furthermore, this therapy can also be used in the treatment of rare genetic retinal diseases, such as Leber's congenital amaurosis, which is likely to augur well for the development of the global retinal gene therapy market during the forecast period, from 2020 to 2030. It is expected that the global retinal gene therapy market is anticipated to witness the entry of new players, with the presence of promising candidates in the phases of drug approval process.

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Type, application, and region are the three important parameters based on which the global retinal gene therapy market has been classified. Such detailed analysis of the market comes with the sole purpose to provide stakeholders with a detailed and clear analysis of the global retinal gene therapy market.

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Global Retinal Gene Therapy Market: Notable Developments

One of the important market developments that give a quick view of the dynamics pertaining to the global retinal gene therapy market is mentioned as below:

There is only one player in this global retinal gene therapy market, which is mentioned as below:

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Global Retinal Gene Therapy Market: Key Trends

The global retinal gene therapy market is characterized by the presence of the following restraints, drivers, and opportunities.

Advent of High-end Technologies to Support Development of the Market

Mostly in the cases of inherited retinal diseases, retinal gene therapy is performed. Gene therapy is capable of bettering vision impairment through mutation in RPE65 gene. Luxturna, a recently introduced gene therapy is utilized for the treatment of patients suffering from type 2 Leber's congenital amaurosis. This disease is a form of inherited disease that causes impairment in vision at the time of birth, which leads to a highly progressive degeneration. At present, there are many retinal gene therapy at the clinical trial phase and those are utilizing recombinant viruses. This factor is likely to increase the scope of growth for the global retinal gene therapy market over the period of assessment, from 2020 to 2030.

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In addition to that, the emergence of new market players together with the advent of high-end technological developments is likely to encourage growth of the global retinal gene therapy market during the forecast period. It is estimated that retinal gene therapy is likely to come up as a standard form of treatment for such retina-related diseases.

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Global Retinal Gene Therapy Market: Geographical Analysis

North America is clearly at the forefront of the growth of the global retinal gene therapy market at the very moment. It is estimated that the region will continue to retain its dominance over the period of forecast, from 2020 to 2030. So far, the product that has been approved for use is from a manufacturer from this region. Europe is likely to emerge as another lucrative region in the global retinal gene therapy market over the period of forecast.

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Retinal Gene Therapy Market: Advent of High-end Technologies to Support Development of the Market - BioSpace

SAN DIEGO, Feb. 5, 2021 /PRNewswire/ -- Angiocrine Bioscience, Inc., a clinical-stage biopharmaceutical company today announced that the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR) has selected Angiocrine's AB-205 Phase 1b/2 study results for an oral presentation.Intravenous AB-205 is being developed to treat diffuse damage of vascular niches of multiple organs caused by off-target cytotoxicity from high-dose chemotherapy (HDT) in the course of conditioning patients undergoing autologous hematopoietic cell transplantation to effect a cure of aggressive lymphomas.Treating the damaged vascular niches enable prompt repair of multiple organs.In case of HDT, the most severely and frequently affected organ systems are oral-gastrointestinal and hematopoietic.By enabling multi-organ repair, AB-205 has the potential of substantially reducing the incidence of severe transplant-related complications that can be life-threatening and prolong hospitalization.

"Our investigators and Angiocrine are honored to be selected by ASTCT & CIBMTR to present at its annual meeting this February," commented Paul Finnegan, MD, Angiocrine's CEO. "We look forward to Dr. Lihua Budde's presentation of AB-205's efficacy and safety results from our Phase 1b/2 study as well as preparing for the upcoming Phase 3 registration study for this indication."

Session Name: Oral Abstract - Session D - Acute Regimen-Related Toxicity and Supportive Care

Title of Abstract: Results of an Open Label Dose Escalation Trial of AB-205 (E-CELcells) in Adults with Lymphoma Undergoing High-Dose Therapy and Autologous Hematopoietic Cell Transplantation (HDT-AHCT)Session Date: Monday, February 8, 2021Session Time: 2:30 PM - 4:00 PM CSTPresentation Time: 3:00 PM CST

About Severe Toxicities and Complications during High-Dose Chemotherapy (HDT) and Autologous Hematopoietic Cell Transplant (AHCT)HDT followed by AHCT is considered a standard-of-care consolidative treatment to cure patients with aggressive systemic lymphoma who have failed 1st-line chemotherapy and respond to chemotherapy induction. Although highly effective in eradicating aggressive cancer cells, HDT also causes collateral damage to healthy tissue, which can lead to severe toxicities and serious, costly complications.The most affected organ system is the lining of the oral-gastrointestinal (GI) tract. The oral GI tract renews its mucosal lining every 3 to 7 days. Because of the collateral damage from HDT, the oral GI tract loses its ability to renew its lining, leading to inflammation (mucositis) and breakdown. The patient suffers from debilitating nausea, vomiting and diarrhea, refractory to medications and prolonging hospitalization.Severe oral GI complications can occur as frequently as 50% and cause profound misery.The rates and severity increase with age and, thus, many older patients are turned away from this potentially curative therapy due to concerns over complication risks.

About AB-205AB-205 represents a new and unique approach to repairing damaged tissues through advanced cell-and-gene therapy. AB-205 consists of allogeneic (off-the shelf) 'universal' E-CEL (human engineered cord endothelial) cells. Intravenous AB-205 is given after completion of HDT and on the same day as AHCT.AB-205's immediate action repairs damaged tissue and thereby prevents (reduces) the extent of breakdown of tissues, which is the root cause of severe toxicities experienced by patients. Reducing or preventing severe toxicities can lead to better quality of life and shorter stay in the hospitali.e., savings to the healthcare system. AB-205 was recently granted both the Regenerative Medicine Advanced Therapy (RMAT) Designation and Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). Angiocrine is currently advancing intravenous AB-205 into a multi-center single registration Phase 3 trial.

About Angiocrine Bioscience, Inc.Angiocrine Bioscience is a clinical-stage biotechnology company developing a radically new way to biologically repair damaged and diseased tissues and organs. Based on its novel and proprietary E-CEL platform, Angiocrine is developing multiple E-CEL therapies designed to repair damaged tissue from age-related degenerative disease of the musculoskeletal system; immune diseases that attack vessels and tissues; and ischemic diseases involving soft tissue, central nervous system and the heart.

For additional information, please contact:

Angiocrine Bioscience, Inc.John R. Jaskowiak(877) 784-8496IR@angiocrinebio.com

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Angiocrine Bioscience Announces Oral Presentation of Intravenous AB-205 Data during the Annual Transplantation & Cellular Therapy Meetings of ASCT...

Feb. 3, 2021 12:30 UTC

ST. LOUIS--(BUSINESS WIRE)-- M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced its scientific advisory board (SAB) that will support the Companys mission of translating its innovative bicistronic-S1S3 technology platform into best-in-class therapies that address unmet needs within the LSD community. The Companys platform enables improved biodistribution of recombinant enzymes to target tissues and efficient cross-correction for gene therapies.

As we work to advance our robust pipeline, we seek the input and support of a world-class team of scientific advisors with deep expertise in genetics, rare diseases, and lysosomal storage and metabolic disorders in particular, said Pawel Krysiak, president and chief executive officer of M6P Therapeutics. The collective insights, knowledge, commitment, and expertise of our scientific advisory board will help us translate this high science into potential medical benefit for the individuals affected by these serious conditions.

By combining the substantial expertise of the SAB with the expertise of the Companys internal R&D team in recombinant enzyme and gene therapies, M6P Therapeutics is well positioned to rapidly advance its deep pipeline of LSD programs. The members of the SAB are:

M6P Therapeutics bicistronic-S1S3 technology platform enhances mannose 6-phosphate content on lysosomal enzymes for both recombinant enzyme and gene therapies, which improves enzyme uptake across target tissues, said Stuart Kornfeld, MD, M6P Therapeutics co-founder and chairman of its SAB. With promising pre-clinical data across numerous LSD programs, this innovation can potentially translate into new and more efficacious treatments, reduced immunogenicity, and more efficient dosing regimens.

About M6P Therapeutics

M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics proprietary bicistronic-S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both enzyme replacement and gene therapies leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of recombinant enzyme and gene therapies by harnessing the power of protein phosphorylation using its bicistronic-S1S3 platform. M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: http://www.m6ptherapeutics.com.

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M6P Therapeutics Announces Formation of Distinguished, Experienced Scientific Advisory Board - BioSpace

The University of Sheffield has been given 5.85 million - the largest single gift from an individual alumnus in its history - to launch a new student support programme and search for new therapies for a range of incurable and debilitating diseases.

The University of Sheffield has been given 5.85 million - the largest single gift from an individual alumnus in its history - to launch a new student support programme and search for new therapies for a range of incurable and debilitating diseases.

The record donation has been made by The Law Family Charitable Foundation (LFCF), which was established by Andrew and Zo Law to support charitable initiatives with an emphasis on education and health.

Andrew Law is Chairman and CEO of Caxton Associates - a global macro hedge fund. He studied Economics at the University of Sheffield and graduated with a First Class honours degree in 1987.

The new student support initiative, named The Law Family Ambition Programme, will run over a five-year period to help disadvantaged students from low participation backgrounds access university, support their success in higher education and help them develop the skills, confidence and social abilities to graduate into successful careers.

It will fund new scholarships, academic mentoring, residential summer schools, career mentoring from successful Sheffield alumni, networking coaching, work placements and employability and skills workshops with businesses.

The landmark donation will also enable the University of Sheffield to expand and enhance its Discover outreach programme, which widens access to professions for pupils from disadvantaged backgrounds throughout the north of England.

Andrew Law said: The Law Family Charitable Foundation is delighted to support the University of Sheffield with the grant award. Since being established in 2011, LFCF has focused upon education and health, together with social mobility and the environment.

The country will only prosper if all of society has the access should it wish to participate fully in leading education opportunities, and supporting the disadvantaged is central to this. I owe a large part of my success to the University of Sheffield and we would like others to have equal opportunities. We are providing 2.85 million to launch a range of activities to help students from disadvantaged backgrounds help gain access and also be supported at the University.

The medical research donation will fund innovative research that could lead to the development of medical therapies for a range of rare genetic diseases which have a devastating effect on people's lives.

The University is already part of a new consortium which aims to accelerate the development of advanced therapies allowing potentially transformative treatments to reach patients sooner. However, the donation will enable the University to develop further partnerships with biotech and pharma companies to help accelerate gene therapy programmes and clinical trials for rare diseases at the same time as supporting regional economic growth and job creation.

The donation presents a real opportunity to drive innovation and excellence in the area of gene therapy and to catalyse the creation of new start-up companies to facilitate commercialisation in the North.

Andrew Law said: It is imperative that more leading UK universities expand their research, exploit their immense expertise for the greater good, and commercialise their success thus creating further growth. The University of Sheffield is developing a national and global reputation in gene therapy. Investing in new medical technology is very capital intensive but is critical to combat rare genetic diseases and cancers. With this in mind we are contributing 3 million to fund the next phase of the gene centres growth.

Professor Koen Lamberts, President and Vice-Chancellor of the University of Sheffield, said: We would like to thank Andrew, Zo and The Law Family Charitable Foundation for making such a generous gift to the University. This donation will make a huge difference to the work we do here at Sheffield. It will drive the next crucial step in research to tackle a range of diseases for which there is currently no cure, as well as provide vital support to students and young people who are considering applying to university. The gift will have a real impact and we are extremely grateful.

For more information on the Law Family Charitable Foundation please visit:

https://www.lawfamilycharitablefoundation.org/

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University of Sheffield receives record donation to support disadvantaged students and pioneering medical research - University of Sheffield News

Over the course of 2020, Sanofisaw significant growth in its specialty care sales primarily driven by its blue-chip asset, Dupixent, as well as an increase in vaccine sales that were driven by demand for differentiated influenza vaccines, the company announced in its year-end financial report.

For the year, Dupixent generated 3.5 billion (about $4.2 billion) for Sanofi. Dupixent, which was co-developed with Regeneron, continues to be a significant driver for Sanofi. In its year-end financial report, Sanofi said sales of Dupixent were driven by continued strong demand in atopic dermatitis (AD) in adult and adolescent patients, rapid adoption in children aged 6 to 11 years, and continued uptake in asthma and chronic rhinosinusitis with nasal polyposis. By the end of 2020, Dupixent has become available in 47 different countries with approximately 230,000 patients on therapy, Sanofi said.

Earlier this year, during a call ahead of the J.P. Morgan Healthcare conference, Frank Nestle, Sanofis chief scientific officer and Global Head of Research, and Dietmar Berger, Sanofis chief medical officer and Global Head of Development, predicted 2021 will be a transformative year for the company. Dupixent plays a significant role in that future, they said.

Sanofi Chief Executive Officer Paul Hudson touted the companys progress in 2020, despite some limitations imposed by the COVID-19 pandemic. He noted the continued uptake in Dupixent use across the globe and also pointed to potential approvals in new indications for the monoclonal antibody that inhibits the signaling of the interleukin-4 and interleukin-13 proteins. The company anticipates approval of Dupixent in pediatric asthma this year.

Other highlights for Sanofi include multiple acquisitions that are expected to provide the company a strong standing in areas like, immune-oncology, synthetic biology and cell and gene therapy. The company is also aiming for innovations with protein degraders, antibody conjugates, nanobodies and multi-specific antibodies, specifically bi- and tri-specific.

Hudson also pointed to its ongoing COVID-19 vaccine development programs. He said clinical trials are expected to begin on its vaccine candidates. Hudson also said the company is making a more immediate contribution to the pandemic fight by providing manufacturing support to BioNTech and Pfizer.

The year-end report wasnt all positives. Sanofi reported a few setbacks, including a Phase II Parkinsons disease study with venglustat. Sanofi said the Phase II study of Parkinsons disease patients with GBA mutations did not meet the primary endpoint at the end of January and the study for this indication was halted. The disclosure is a setback for the companys plans for venglustat. Ahead of the J.P. Morgan conference, company officials said venglustat had the potential to be a pipeline in a pill. The venglustat safety profile continues to be favorable and the development moves forward as planned in other Rare Disease indications, the company said. Venglustat is being assessed in other studies, including Phase III programs for GM2 Gangliosidosis and Autosomal Dominant Polycystic Kidney Disease, as well as Phase II studies in Fabry disease and Gaucher disease Type 3.

Other programs that will not continue include the anti-IL4/IL13 bispecific mAb Romilkimab, which was in a Phase II study for systemic scleroderma, as well as asthma treatment Itepekimab and lymphoma treatment Isatuximab. Both of those were also in Phase II.

Sanofi said it is working to resolve a Complete Response Letter issued by the U.S. Food and Drug Administration during the fourth quarter for sutimlimab, an investigational monoclonal antibody for the treatment of hemolysis in adults with cold agglutinin disease. The CRL was issued due to manufacturing concerns. There were no clinical concerns. Sanofi said it is working with both the FDA and the third-party manufacturer to reach a resolution in a timely manner.

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Sanofi Succeeding with Blue-Chip Asset, Dupixent, While Other Programs Fall Away - BioSpace

publication date: Jan. 29, 2021

Craig Lockhart was named chief of the Division of Hematology and Oncology in the Department of Medicine at the Medical University of South Carolina and associate director for clinical science at MUSC Hollings Cancer Center, effective April 1.

Lockhart will be replacing interim chief Michael Lilly, and will be working both on campus and remotely until he transitions to campus full time in September.

Lockhart holds several roles at the University of Miami Sylvester Comprehensive Cancer Center, including chief of the Division of Oncology and associate director for regional and strategic research affiliations. His research specialty is gastrointestinal cancers, and he has been a principal investigator on more than 100 phase I/II and III trials.

Lockhart is chief of service for oncology as part of the University of Miami Medical Group.

Prior to joining the University of Miami, Lockhart served on faculty and in leadership roles at Washington University and Siteman Cancer Center in St. Louis and at Vanderbilt University and Vanderbilt-Ingram Cancer Center in Nashville.

Lockhart has been conducting early-phase clinical trials for more than 20 years. His specific research interests are developing and conducting Phase I/II clinical trials of novel therapeutics applied to gastrointestinal cancers.

Terence M. Williams named radiation oncology chair at City of Hope

Terence M. Williams was named professor and chair of City of Hopes Department of Radiation Oncology.

Williams is tasked with expanding clinical and basic science research in the department. He will also integrate and expand novel treatment therapies, provide professional development and advancement opportunities for radiation oncology physicians, expand the Radiation Oncology Residency Program and build on the departments financial performance.

Previously, Williams held several leadership roles at The James Cancer Hospital and Comprehensive Cancer Center at The Ohio State University. Most recently, he served as vice chair of translational research, associate professor of radiation oncology and division director of the Thoracic and Hepatopancreaticobiliary clinical programs.

Williams specializes in treating patients with thoracic and gastrointestinal cancers, with a particular emphasis on non-small cell lung cancer, pancreatic cancer and hepatobiliary malignancies.

His laboratory-based, NIH-funded research focuses on DNA damage response pathways, DNA repair and novel mechanisms of sensitization to radiation and other genotoxic therapies and nutrient scavenging through caveolae-mediated endocytosis.

Coalition of cancer organizations urges resumption of cancer screening and treatment during pandemic

The National Comprehensive Cancer Network and the American Cancer Society are teaming up with cancer organizations across the country to endorse the resumption of cancer screening and treatment during the ongoing COVID-19 pandemic.

The coalition of 76 organizations released an open letter reminding the public that cancer still poses a major threat to peoples health, but acting as soon as is safely possible can lead to much better outcomes in the future.

The letter strongly recommends that hospitals, medical systems and patients:

Ensure people in our communities are not delaying care for important medical issues.

Encourage people in our communities to resume recommended cancer screening.

Facilitate and encourage people with cancer to resume evidence-based treatment.

Contact your doctor right away if any concerning medical symptoms arise.

Resume all preventive and prescribed care, including regular cancer screening, as recommended by your doctor.

The letter examines distressing trends showing a significant drop-off in recommended cancer screening and treatment compared to prior years. This concerning side-effect of the pandemic could lead to an increase of preventable cancer deaths over the next ten years and beyond.

Experts agree that people should not delay any necessary prevention or care.

When the pandemic first hit the United States, a short delay in care was an appropriate choice for many cancer types. However, the balance of risk has shifted significantly, Robert W. Carlson, chief executive officer of NCCN, said in a statement. Cancer centers are taking multiple measures to protect patients and staff from COVID-19 and transmission within cancer centers is quite unusual. Meanwhile, far too many cancers are being left to grow unchecked. Postponing cancer care will add tragedy on top of tragedy.

Over the past decade we have seen overall cancer mortality rates drop dramatically. This decline is in large part due to screenings ability to catch cancers before they spreadwhen the chances of good outcomes are most likely, William G. Cance, chief medical and scientific officer of ACS, said in a statement. We have come too far in our fight against cancer to allow long breaks in vital screening to slow down our progress in saving lives.

Hospitals and medical systems have begun vaccinating health care providers among other measures to ensure a safe environment for people receiving cancer screening and treatment. The confirmed use of evidence-based precautions against COVID-19 should provide reassurance against fears of infection during necessary medical care.

Experts are now asking everyone, in coordination with their health care provider, to resume preventive and prescribed care and contact their doctor right away about any new symptoms or concerns.

Visit NCCN.org/resume-screening or acs4ccc.org/ReengageLetter to read the entire letter.

Coalition of cancer scientists create global initiative to evaluate genetic mutations

Cancer scientists from the Wellcome Sanger Institute, the Human Technopole in Milan, and the Broad Institute of MIT and Harvard are calling on cancer researchers to join a global initiative to systematically evaluate the effect of every genetic mutation and every drug on every cancer.

Researchers from the organizations published a perspective on the subject in Nature.

These collaborators plan to create the Cancer Dependency Map, an approach that has shown great promise in pilot studies to help develop new cancer treatments. The goal is to make precision cancer medicine a reality for every patient.

A dependency is a gene, protein, or other molecular feature that a tumor depends on for growth. These dependencies are also vulnerabilities, which can be targeted to kill a cancer. Such vulnerabilities can inspire new drugs or ways to repurpose existing drugs, even ones that have not been considered for cancer treatment before.

To build this map, the authors think it will be necessary to perturb 20,000 genes and assess 10,000 drugs in 20,000 laboratory cancer models. Doing that will take a coordinated global effort similar in scale to the Human Cell Atlas, drawing on the expertise of specialists in genome editing, machine learning, cancer biology, cancer modeling, and high-throughput drug screening.

Authors Mathew Garnett and Jesse Boehm will be giving a news briefing at the American Association for the Advancement of Science annual meeting on Feb. 8.

NYU Dentistry receives $3.28M NIH grant for oral cancer pain research

NYU College of Dentistry clinician-scientists Seiichi Yamano and Brian Schmidt have received a five-year, $3.28 million grant (R01DE029694) from the National Institute of Dental and Craniofacial Research.

With the grant, the researchers will test whether nonviral co-delivery of DNA and RNA will safely alleviate oral cancer pain. Yamano and Schmidt have set out to develop a new class of medicines using gene therapy to effectively and safely treat oral cancer pain.

Patients with oral cancer often suffer from severe pain. The opioid medications used to treat oral cancer pain become less effective as patients develop drug tolerance, and cause numerous debilitating side effects.

Gene therapy offers an alternative to opioids for the treatment of cancer pain by reversing cancer-induced epigenetic changes. This approach selectively disrupts pain signaling without the side effects of opioids.

Complete elimination of cancer pain in a patient is exceptionally challenging because there are multiple and redundant pain-signaling mechanisms and pathways, Schmidt, professor in the Department of Oral and Maxillofacial Surgery at NYU College of Dentistry and director of NYUs Bluestone Center for Clinical Research and the NYU Oral Cancer Center, said in a statement.

As a strategy to obstruct these multiple and varied pathways, Yamano and Schmidt created two nonviral vectors that can efficiently deliver DNA and RNA to cells (transfection) with no toxicity: a cell-permeable peptide combined with a cationic lipid for DNA, and a lipopolymer for RNA. They hypothesize that the combination of OPRM1 (mu opioid receptor gene) re-expression and F2RL1 (gene for protease-activated receptor-2, or PAR2) downregulation in the cancer could eliminate cancer pain.

In preliminary studies, Yamano and Schmidt demonstrated that nonviral transfection with OPRM1 DNA led to re-expression of the mu opioid receptor and partial reduction of pain in preclinical cancer models. PAR2 was found to be elevated in certain neurons that supply the cancer with nerves and drive pain. Knockdown of the F2RL1 gene partially attenuated pain.

In their newly funded NIH grant, the researchers will test whether the combination of OPRM1 re-expression and F2RL1 downregulation in the cancer can go beyond reducing cancer pain to eliminate it.

Our approach is innovative because delivering DNA and RNA into a cancer with nonviral vectors for the management of pain has not been done before, Yamano, associate professor of prosthodontics at NYU College of Dentistry, said in a statement. If we are successful, the knowledge generated through this research could set the stage for a clinical trial and ultimately lead to the development of novel non-opioid medicines for cancer pain.

Yamano and Schmidt have collaborated on this work over the last decade; their efforts have been supported by three previous NIH-funded grants.

UAMS Winthrop P. Rockefeller Cancer Institute receives $1M to pursue NCI designation

The University of Arkansas for Medical Sciences has received a $1 million pledge from Larry Crain Sr. to support the Winthrop P. Rockefeller Cancer Institutes pursuit of NCI designation.

In appreciation of the gift, UAMS will rename the Seed of Hope Garden on the Cancer Institutes ground floor as the Janett Crain Seed of Hope Garden, after Crains late wife, who died of cancer in 2018.

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Craig Lockhart named chief of hematology and oncology at MUSC - The Cancer Letter

Over the next 10 years, gene therapies are expected come into their own as a treatment option for a variety of diseases. So far, two such therapies have snagged regulatory approval, Novartis Zolgensma for spinal muscular atrophy, and Sparks Luxturna for a rare form of genetic blindness. More are waiting their turn.

Multiple companies are delving into gene therapy research with hopes of developing a one-time treatment for devastating genetic diseases. Gene therapies offer great reward in the form of treating various devastating diseases, but there are also significant risks. Over the past year, several clinical studies have been halted or scrapped due to safety concerns.

Bay Area-based Audentes Therapeutics had a temporary hold placed on the gene therapy under development for X-linked myotubular myopathy following reports of several patient deaths. That hold has since been lifted by the U.S. Food and Drug Administration. Uniqure also saw a hold placed on its hemophilia B trial after a patient in the study developed liver cancer. The hold was placed weeks after the company announced promising Phase III results at a conference in December.

Despite those risks, hundreds of millions of dollars in research dollars are being invested in gene therapies because of the potential near-curative capabilities the technology could offer. In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies. Eli Lilly also dove into the field in December with the acquisition of Prevail Therapeutics. That deal was expected to extend Eli Lillys research efforts through the creation of a gene therapy program that will be anchored by Prevail's portfolio of clinical-stage and preclinical neuroscience assets.

This week, German scientists reported they were able to use gene therapy to help paralyzed mice run again. The researchers were able to genetically engineer a unique protein dubbed hyper-interleukin-6, which was then able to stimulate the regeneration of nerve cells in the visual system. A few weeks after the treatment, the injured animals were able to walk again.

Scientists in China announced the development of a gene therapy that could potentially reverse the effects of ageing. Initial research was conducted with mice, but if it is proven to be safe, human testing could begin. As Reuters reported, the method involved inactivating a gene called kat7 which the scientists found to be a key contributor to cellular ageing. Researchers used CRISPR/Cas9 to screen thousands of genes for those which were particularly strong drivers of cellular senescence, the term used to describe cellular ageing, Reuters said.

Earlier this month, a public-private partnership in Boston formed to open a new facility to boost advances in cell and gene therapies. This creation of this new facility is being helmed by Harvard University and the Massachusetts Institute of Technology. Those prestigious universities are partnering with industry members such as Fujifilm Diosynth Biotechnologies, Cytivia and Alexandria Real Estate Equities, as well as multiple research hospitals. Part of the goal of this new institute, which is still unnamed at this point, is to boost the supply of materials for research and early clinical studies, provide space for some research and also offer training in equipment used for gene therapies, The Harvard Gazette reported this week.

On Monday, Curadigm, a subsidiary of France-based Nanobiotix, forged a collaboration with Sanofi to assess if that companys Nanoprimer technology is a promising option to significantly improve gene therapy development. The goal of the project is to establish proof-of-concept for the Nanoprimer as a combination product that could improve treatment outcomes for gene therapy product candidates.

Many promising nucleic acid-based therapeutics administered intravenously are limited in their efficacy due to rapid clearance in the liver, which prevents these therapies from reaching the necessary accumulation in target tissues to generate their intended outcomes. Additionally, accumulation in the liver, rather than in the target tissues, can lead to dose-limiting hepatic toxicity, Nanobiotix said in its announcement. The Nanoprimer is designed to precisely and temporarily occupy therapeutic clearance pathways in the liver. Delivered intravenously, immediately prior to the recommended therapy, the technology acts to prevent rapid clearancethereby increasing bioavailability and subsequent accumulation of therapeutics in the targeted tissues.

The Nanoprimer is a combination product candidate that does not alter or modify the therapies it is paired with, which means if the research with Sanofi is successful, Curadigm could seek out other opportunities for its technology.

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Are Gene Therapies the Medicine of the Future? - BioSpace

News Release

Thursday, January 28, 2021

Approach could provide new path for difficult-to-treat forms of Leber congenital amaurosis.

Scientists at the National Eye Institute (NEI) have developed a promising gene therapy strategy for a rare disease that causes severe vision loss in childhood. A form of Leber congenital amaurosis, the disease is caused by autosomal-dominant mutations in the CRX gene, which are challenging to treat with gene therapy. The scientists tested their approach using lab-made retinal tissues built from patient cells, called retinal organoids. This approach, which involved adding copies of the normal gene under its native control mechanism, partially restored CRX function. The study report appears today in Stem Cell Reports. NEI is part of the National Institutes of Health.

Our treatment approach, which adds more copies of the normal gene, could potentially treat autosomal-dominant LCA caused by a variety of mutations, said Anand Swaroop, Ph.D., chief of the NEI Neurobiology, Neurodegeneration and Repair Laboratory and senior author of the report.

The U.S. Food and Drug Administration approved Luxturna in 2017 for the treatment of LCA patients with mutations in a gene called RPE65. Although hailed as a major advance in gene therapy, Luxturna is ineffective against other forms of LCA, including those caused by autosomal-dominant mutations in CRX.

The CRX gene encodes a protein (also called CRX) that binds to DNA and instructs the retinas photoreceptors to make light-sensitive pigments called opsins. Without functional CRX protein, photoreceptors lose their ability to detect light and eventually die.

Disorders like autosomal-dominant LCA are tricky to treat with gene therapy, because adding more of the normal gene does not always restore function. People with autosomal-dominant mutations still have one normal copy of the gene, but the mutant version of the protein interferes with the normal protein. Sometimes, instead of restoring normal function, simply adding more of the normal protein can enhance the disease in unpredictable ways.

To explore how gene augmentation adding copies of the normal gene would affect autosomal-dominant LCA, Swaroops team, developed retinal organoids from two volunteers with LCA and from their unaffected family members. Led by Kamil Kruczek, Ph.D., a postdoctoral fellow in Swaroops lab, they built the complex retina-like tissues in several stages, starting with skin cells, inducing the production of mature photoreceptors and other retinal cells with the genetic profile of each volunteer. As expected, patient organoids made far less light-sensing opsin than the organoids made from unaffected family members.

To carefully control how much CRX gene would be expressed by the recipient photoreceptors, the team re-engineered the CRX promoter so it could be delivered with the CRX gene as part of the gene therapy. A promoter is a neighboring sequence of DNA that controls when and how genes are expressed. The researchers packed the gene and their engineered promoter inside a virus that shuttled them into the organoid photoreceptors.

The teams gene augmentation strategy restored some CRX protein function for organoids from both patients, driving expression of opsins in both types of photoreceptors: rods and cones.

The fact that this strategy worked for both CRX mutations was pretty exciting, said Swaroop. Gene augmentation may be a viable therapy for LCA caused by other autosomal-dominant mutations.

This proof-of-concept gene therapy study is the first step toward a potential treatment for a rare form of LCA, said Brian Brooks, M.D., NEI clinical director and co-author on the study. Its a great example of bench-to-bedside science, when researchers in basic and clinical science collaborate.

The current study was funded through the intramural programs of the NEI and the National Institute of Allergy and Infectious Diseases, both part of NIH. Patient samples were collected at the NIH Clinical Center, clinical trial number NCT01432847.

NEI has protected intellectual property around this technology which is available for licensing and or co-development. Details can be found on the NIH OTT Licensing website: Gene Therapy for Treatment of CRX-Autosomal Dominant Retinopathies | Office of Technology Transfer, NIH or by contacting NEI Office of Translational Research mala.dutta@nih.gov

Additional authors include: Zepeng Qu, James Gentry, Benjamin Fadl, Linn Gieser, Suja Hiriyanna, Zacahry Batz, Mugdha Samant, Ananya Samanta, Colin Chu, Laura Campello, and Zhijian Wu.

NEI leads the federal governments research on the visual system and eye diseases. NEI supports basic and clinical science programs to develop sight-saving treatments and address special needs of people with vision loss. For more information, visit https://www.nei.nih.gov.

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

NIHTurning Discovery Into Health

Kruczek K. Qu Z, Gentry J, Fadl BR, Gieser L, Hiriyanna S, Batz Z, Samant M, Samanta A, Chu CJ, Campello L, Brooks BP, Wu Z, and Swaroop A. Gene therapy of dominantCRX-Leber congenital amaurosis using patient stem cell-derived retinal organoids.Stem Cell Reports, January 28, 2020.https://doi.org/10.1016/j.stemcr.2020.12.018

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Researchers use patients' cells to test gene therapy for rare eye disease - National Institutes of Health

NOIDA, India, Jan. 29, 2021 /PRNewswire/ -- A comprehensive overview of the gene therapy market is recently added by UnivDatos Market Insights to its humongous database. The gene therapy market report has been aggregated by collecting informative data of various dynamics such as market drivers, restraints, and opportunities. This innovative report makes use of several analyses to get a closer outlook on the gene therapy market. The gene therapy market report offers a detailed analysis of the latest industry developments and trending factors in the market that are influencing the market growth. Furthermore, this statistical market research repository examines and estimates the gene therapy market at the global and regional levels. The Global Gene therapy Market is expected to grow at a CAGR of 29.7% from 2021-2027 to reach US 20.9 billion by 2027.

Market Overview

Gene therapy is the next trend of curative transformation in the life sciences industry. Globally, around 2,600 clinical trials in gene therapy have been performed, are underway, or have been approved to date. More than ever, the field of gene therapy seeks to identify a route to the clinic and the market. Approximately 20 gene therapies have now been licensed and over two thousand clinical trials of human gene therapy have been published globally. Aging populations worldwide and socio-economic risk factors are among the primary influences driving this surge.

As per Alliance for Regenerative Medicine (ARM) Quarterly Regenerative Medicine Global Data Report Q12019, 372 gene therapy clinical trials were in progress as of the end of Q1. Remarkably, a margin (217 or 58%) were studies in Phase II, followed by Phase I (123 or 33%), and Phase III (32 or 9%). The number of gene therapy clinical trials edged up by 10 from the 362 recorded as of the end of 2018.

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Covid-19 Impact:

The COVID-19 pandemic has dislocated global management attempts across gene therapies. The manufacture and delivery of treatments, research and clinical development, and commercial operations are the three areas within the gene therapy sector that have been most interrupted amid the COVID-19 crisis. The development of gene therapies has been less affected. For instance, Peter Marks, Director of FDA's Center for Biologics Evaluation and Research (CBER) stated that with the arrival of therapies for cell and gene therapies over the last five years, it should have doubled in size while it is only modestly larger, 15-20% larger in size.

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Global Gene therapy Market report is studied thoroughly with several aspects that would help stakeholders in making their decisions more curated.

By Vector, the market is primarily bifurcated into

The viral vector segment dominated the gene therapy vector market in 2019 and will grow at 29.2% CAGR to reach US$ 17.9 billion by the year 2027.

By Viral Vector, the market is primarily sub-segmented into

Amongst viral vector types, adeno-associated virus accounted for the largest share and is expected to grow at 29.3% CAGR during the forecast period 2021-2027. In 2019, the adeno-associated virus segment accounted for a revenue share of almost 34%.

By Gene Type, the market is primarily studied into

In 2019, the antigen segment dominated the global gene therapy market with nearly 19.2% of the market share and it is anticipated by 2027, the segment will garner US$ 3.9 billion of the market.

By Indication, the market is primarily studied into

In 2019, the oncology segment dominated the global gene therapy market by indication with nearly 48.6% of the market share and it is anticipated to grow at 27.6% CAGR during the forecast period 2021-2027.

By Delivery Method, the market is primarily segmented into

Amongst delivery method, In vivo accounted for the largest share and is expected to grow at 28.6% CAGR during the forecast period 2021-2027. In 2019, the ex vivo segment accounted for a revenue share of 12.5%.

Gene therapy Market Geographical Segmentation Includes:

Based on the estimation, the North America region dominated the gene therapy market with almost US$ 1.7 billion revenue in 2019. At the same time, the Asia-Pacific region is expected to grow remarkably with a CAGR of 28.7% over the forecast period on account of owing increasing government initiative to improve healthcare infrastructure and rise in healthcare expenditure and surging cancer incidence rate in the region.

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The major players targeting the market includes

Competitive Landscape

The degree of competition among prominent global companies has been elaborated by analyzing several leading key players operating worldwide. The specialist team of research analysts sheds light on various traits such as global market competition, market share, most recent industry advancements, innovative product launches, partnerships, mergers, or acquisitions by leading companies in the gene therapy market. The leading players have been analyzed by using research methodologies for getting insight views on global competition.

Key questions resolved through this analytical market research report include:

We understand the requirement of different businesses, regions, and countries, we offer customized reports as per your requirements of business nature and geography. Please let us know If you have any custom needs.

For more informative information, please visit us @ https://univdatos.com/report/global-gene-therapy-market-current-analysis-and-forecast-2020-2027

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Gene Therapy Market to Reach US$ 20.9 Billion by 2027, Globally |CAGR: 29.7%|UnivDatos Market Insights - PR Newswire India

Newswise ROCHESTER, Minn. In a new study published in Circulation, Mayo Clinic researchers provide the first preclinical, proof-of-concept study for hybrid gene therapy in long QT syndrome, a potentially lethal heart rhythm condition.

Researchers demonstrated its potential therapeutic efficacy in two in vitro model systems using beating heart cells reengineered from the blood samples of patients with 1 long QT syndrome. They targeted the whole KCNQ1 gene rather than specific LQT1-causative mutations, making this study applicable to all patients with long QT syndrome 1, regardless of their specific disease-causing variant.

The team consisted of Mayo Clinic researchers from Mayo Clinic's:

The prevalence of long QT syndrome is about 1 in 2,000. When untreated, high-risk patients have an estimated 10-year mortality of 50%.

Long QT syndrome is a genetic heart rhythm condition that can potentially cause fast, chaotic heartbeats. These rapid heartbeats might trigger people to suddenly faint. Some people with the condition have seizures. In some severe cases, long QT syndrome can cause sudden cardiac death. The most common subtype, type 1 long QT syndrome, or LQT1, is caused by pathogenic variants in the KCNQ1 gene.

"Gene therapy is an emerging area of interest for treating a variety of genetic heart diseases in general and long QT syndrome in particular," says Michael Ackerman, M.D. Ph.D., a Mayo Clinic genetic cardiologist and director of Mayo Clinic's Windland Smith Rice Comprehensive Sudden Cardiac Death Program. "We designed and developed the first suppression and replacement KCNQ1gene therapy approach for the potential treatment of patients with type 1 long QT syndrome." Dr. Ackerman is senior author of this study.

According to Dr. Ackerman, over the past two decades, substantial improvements have been made to manage long QT syndrome, but current therapies, such as beta blockers and defibrillators, a more invasive therapy, still have limitations, risks and an array of unwanted side effects.

Gene therapy is a technique that treats diseases caused by defective genes by altering genes in a patient's cells rather than using drugs or surgery. Genes contain DNA the code that controls the body's form and function. Gene therapy replaces faulty genes or adds a new gene to try to treat a disease.

According to Dr. Ackerman in this case, this is the first time that hybrid gene therapy (simultaneous out with the old, in with the new) has been created for any form of genetic heart disease.

"If the therapeutic efficacy of this 'disease-in-the-dish' gene therapy trial with KCNQ1 can be replicated in a nonhuman, animal model of long QT syndrome, then suppression-replacement (hybrid) gene therapy may be a promising strategy for long QT syndrome in general and in theory almost any sudden death-predisposing autosomal dominant genetic heart disease," says Dr. Ackerman. "Of course, we still have a long way to go from nearly curing a patient's heart cells in the dish to effectively treating the whole person. Nevertheless, we are excited by this first critical milestone and look forward to the next step."

In addition to heart disease, researchers at Mayo Clinic's Center for Individualized Medicine are investigating an approach to replace and fix mutated genes in a wide range of genetic disorders.

Funding This work was supported by Mayo Clinic's Windland Smith Rice Comprehensive Sudden Cardiac Death Program (MJA), the Dr. Scholl Foundation (MJA), Mayo Clinic's Center for Individualized Medicine High-Definition Therapeutics grant (MJA), and Mayo Clinic's Department of Molecular Pharmacology and Experimental Therapeutics training grant under National Institutes of Health award T32GM072474 (SMD and MJA).

Disclosures Dr. Ackerman is a consultant for Abbott, Armgo Pharma Inc., Audentes Therapeutics, Boston Scientific Corporation, Daiichi Sankyo Co. Ltd., Invitae Corporation, LQT Therapeutics Inc., Medtronic, MyoKardia, and UpToDate Inc. Dr. Ackerman and Mayo Clinic are involved in an equity/royalty relationship with AliveCor Inc.

About Mayo Clinic Mayo Clinic is a nonprofit organization committed to innovation in clinical practice, education and research, and providing compassion, expertise and answers to everyone who needs healing. Visit the Mayo Clinic News Network for additional Mayo Clinic news and Mayo Clinic Facts for more information about Mayo.

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First hybrid gene therapy shows early promise in treating long QT syndrome - Newswise

With the rising demand for cell and gene therapies, the need for manufacturing innovation has never been higher. A surge of deals and expansions in the last year is fuelling the push to truly make these therapies widely available and affordable.

Cell and gene therapies offer huge potential to treat a wide range of diseases including cancer, neurological, and genetic diseases. They have even shown promise to treat the symptoms of Covid-19.

The amount of academic and early-stage biotech research in this area has skyrocketed over the last few years. According to the Alliance for Regenerative Medicine, there are currently 1,220 ongoing clinical trials in this space, 152 of which are at phase III. Despite the global pandemic, investment in this area is also at a record high around the world, with the equivalent of 15.7B invested in 2020, a figure double that of 2019.

But research alone cannot get these complex treatments to patients. The sharp discrepancy between the high number of products in early-stage development and the still very small number that have made it onto the market, as well as their cost, speaks to the impact and importance of cost-effective and scalable manufacturing, Ryan Cawood, CEO of Oxgene (previously Oxford Genetics), told me. Oxgene is a UK biotech aiming to improve manufacturing for cell and gene therapies.

To meet this challenge, cell and gene therapy producers are exploding into motion. With 2021 only just getting started, weve seen manufacturing deals between Vigeneron and Daiichi Sankyo, Sirion Biotech and Cellectis, and Cevec and Biogen. The giant Thermo Fisher Scientific absorbed the Belgian viral vector producer Henogen for 724M. And CDMO heavyweights like Cognate BioServices and Polyplus Transfection have announced expansions to their manufacturing capacity.

Thedifficulties with manufacturing the recently approved Covid-19 mRNA vaccines in high enough quantities has really highlighted the importance of having a solid manufacturing strategy in place. This lesson applies equally to companies trying to take cell and gene therapies to market.

Stuck in the first generation

Despite the huge increase in development of cell and gene therapies over the past couple of years, manufacturing technology for these therapies is largely still at the first-generation stage. This can make scaling up a challenge.

Often cell and gene therapy manufacturing processes are highly manual stemming from the early academic or process development stage and, without adequate technology solutions available currently, these processes often remain this way through clinical trials and then into commercial manufacturing, said Jason Foster, CEO of Ori Biotech, a London- and New Jersey-based company focusing on cell and gene therapy manufacturing.

These first-generation processes cause manufacturing to be expensive, highly variable and low-throughput, which reduces the ability of patients to access these potentially life-saving therapies.

Another problem common to all bio-based therapeutics is that any product sourced from a live cell or a component of one is subject to a lot more variation than a simpler pharmaceutical product.

Most gene therapies are built on viruses found in nature. They have not evolved for very high productivity in a large-scale, animal component-free bioreactor, said Cawood.

The more complicated the biologic becomes, the more parts of it require optimization, and the more analytics you require.

According to Kevin Alessandri, the cofounder and CEO/CTO of the French company TreeFrog Therapeutics, there is also a lot of waste in cell therapy manufacturing.

Yields are impaired by high cell death at every passage, and genetic alterations inevitably arise, said Alessandri. When it comes to producing commercial batches to treat thousands of patients, scaling out 2D cell culture processes is far too expensive and poses batch-to-batch reproducibility issues.

While many in the industry are now turning to bioreactors to produce cells on a bigger scale, this is also not without problems. Impeller-induced shear stress is damaging the cells, thus negatively impacting cell viability and triggering undesired genetic mutations, explained Alessandri.

Taking manufacturing up a gear

What are companies in this space doing to make scaling up cell and gene therapies easier, quicker, and cheaper?

Ori Biotech raised24.8M in Series A funding in October last year to develop an automated and robotic manufacturing system to minimize the number of manual steps needed to produce a given cell or gene therapy. This speeds up the process as well as making it more accurate. Another advantage of the technology is that it can tailor the production capacity according to demand.

This is impossible to do in most current processes, which involve manual tube welding and transfers from flask to bag to bigger bag to bioreactor, said Foster, adding that this increases cost and variability while constraining throughput. Oris technology, in contrast, could take years off the production timeline and cut costs by as much as 80%.

London-based Synthace is one of several companies trying to improve advanced therapeutic manufacturing by developing software and computer systems to optimize the process, rather than industrial machinery.

Peter Crane, Corporate Strategy Manager for the company, said that in-depth data analysis and planning before starting the manufacturing process can make a big difference to outcomes, and that connected software can help make this task easier.

The best way to remove some of the risk associated with biomanufacturing of these products is to solve as many problems as possible before manufacturing.

In addition to making the process quicker, cheaper, and more accurate, computing tools can also help with quality control and tracking. In cell therapy manufacturing, especially autologous products, line of sight around electronic batch records, as well as the vein-to-vein supply chain, is incredibly important, emphasized Crane.

Another company specifically focusing on logistics and quality control is the Cardiff- and San Francisco-based TrakCel, which nailed deals with Ori Biotech in February and the UKs National Health Service in November.

The company TreeFrog Therapeutics works with cell encapsulation technology to improve quality and reduce waste, albeit from a more mechanical viewpoint. The company launched an industrial demonstration plant in June last year, followed by two co-development deals with undisclosed big pharma partners.

Encapsulated stem cells spontaneously self-organize in an in vivo-like 3D conformation promoting fast and homogeneous growth, as well as genomic stability, said Alessandri. The resulting 3D stem cell colony can then be differentiated in the capsule into functional microtissues ready for transplantation.

With our technology, which is based on high-throughput microfluidics capable of generating over 1,000 capsules per second, it becomes possible to expand and differentiate stem cells at a large scale, in industrial bioreactors, with best-in-class cell quality and reduced operating costs.

Oxgene has a focus on scaling up production for manufacturers. In September, the company launched a technology to scale up manufacturing of viral vector production with less contamination and a 40-fold improvement in yield compared to current methods. Oxgenes expertise with viral vectors also prompted a collaboration deal in April with the CDMO Fujifilm Diosynth Biotechnologies.

Innovation in new manufacturing technologies just hasnt kept pace with the level of discovery around genetic disease and potential avenues open to treat them, or even development of the viral vectors themselves, said Cawood. This is definitely changing though.

Enter the second generation of manufacturing

Cell and gene therapy manufacturing is definitely hot right now, boosted by increased needs from biotech and pharma companies developing Covid-19 vaccines and therapies, and by notable increases in investment.

Huge advances in gene and cell therapies over the last few years, such as the approval of the eye gene therapy Luxturna and the first CAR T-cell therapies, mean the demand for new manufacturing technologies has also increased exponentially.

A lot of very promising programs are now in the pipeline, and patients are waiting for their approval, said Alessandri. Industry urgently needs robust manufacturing technology, capable of serving millions of patients.

European biotechs are busy developing second-generation technologies to allow easier and cheaper scale up, producing higher quality products with less waste. They could start to phase out first-generation methods very soon.

The realm of cell manufacturing in industrial and food biotech is also likely to see big breakthroughs in the coming years. Earlier this month, for instance, the nutrition and health giant Royal DSM set up a lab in the Netherlands dedicated to applying artificial intelligence (AI) to the challenge of growing microbial strains at a commercial scale.

Rapid improvements in advanced computing options such as AI and machine learning technology, as well as robotics, are already having an effect on the industry, but this will only get bigger as time goes on.

Cover image from Elena Resko. Body text image from Shutterstock

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Cell and Gene Therapy Firms Gear up to Revolutionize Manufacturing - Labiotech.eu

Vancouver, British Columbia, Jan. 26, 2021 (GLOBE NEWSWIRE) -- The global cell and gene therapy market is projected to reach a market size of USD 7,250.0 Million by 2028 at a rapid and steady CAGR of 16.3% over the forecast period, according to most recent analysis by Emergen Research. The growing demand for cell and gene therapy can be attributed to increasing investments in production capacity expansion for cell and gene therapy. Several contract development & manufacturing organizations and contract manufacturing organizations are making huge investments in the expansion of cell and gene therapy production capacity, anticipating a rise in demand for their services from biopharmaceutical companies that emphasize the development and production of emerging therapeutic technologies.

For instance, in May 2019, CDMO Catalent invested USD 1.20 billion in Paragon Bioservices, a contract development & manufacturing organization involved in developing and producing viral vector development for gene therapy. In April 2019, Paragon Biosciences had commenced its second good manufacturing practices (GMP) gene therapy production facility in Harmans, Maryland, the US, to provide customized manufacturing set-ups to manage the specific requirements for gene therapy products.

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Cell and Gene Therapy Market Size to Reach USD 7,250.0 Million by 2028 | Increasing Investments in Production Capacity Expansion for Cell and Gene...

RED BANK, N.J., Jan. 28, 2021 /PRNewswire/ --Provention Bio, Inc. (Nasdaq: PRVB), a biopharmaceutical company dedicated to intercepting and preventing immune-mediated diseases, today reported results from a pre-clinical proof-of-concept study for PRV-3279, a DART(bispecific antibody-based molecule) targeting the B cell surface proteins CD32B and CD79B, conducted in a murine model of gene therapy for Pompe disease. A PRV-3279 mouse surrogate was tested in mice transgenic for human CD32B, which received gene therapy with an adeno-associated virus (AAV) vector AAV9 encoding for the enzyme acid-alpha-glucosidase (GAA) gene. Errors in the GAA gene cause the serious human glycogen storage disease type II (Pompe disease).

In the study, the PRV-3279 surrogate reduced anti-AAV9 vector antibody levels in a dose-dependent fashion. Anti-AAV9 antibodies have been linked to reduced efficacy, safety concerns and the inability to re-dose patients, and thus, based on these and other study data, we believe PRV-3279 co-administration with gene therapy products has the potential to improve the safety and efficacy of this therapeutic modality. The PRV-3279 surrogate in combination with sirolimus increased skeletal muscle levels of GAA enzyme expression. Consistent with prior results from clinical trials in healthy human subjects, the PRV-3279 surrogate decreased IgM production and was well tolerated.

"As the field of gene therapy advances, patients' immune responses to the viral vectors and the transgene products remain a key challenge negatively impacting the safety, efficacy and ability to deliver additional courses systemically," stated Francisco Leon, M.D., Ph.D., chief scientific officer, Provention Bio. "One of the current mitigation strategies to overcome these immune responses is pharmacological modulation of the patients' antibody immune responses with the B cell depleting agent rituximab in combination with the immune-suppressive agent sirolimus. Prolonged use of rituximab has been associated with certain adverse events. The use of PRV-3279, a non-depleting B cell inhibitor, is a potential strategy to address this unmet need in serious genetic diseases."

"A critical challenge for the success of gene therapy is the host immune responses to both the vector capsid and transgene product, which pose ongoing concerns regarding the safety, longevity, extent of gene expression and ability to re-dose," stated Professor Barry Byrne, director of the Powell Gene Therapy Center at the University of Florida. "PRV-3279's mechanism of action, inhibiting B cell activation without depleting these important cells, has the potential to provide a unique opportunity to be used as an adjunctive therapy with gene therapy products. We look forward to collaborating with Provention Bio and other potential partners in forthcoming clinical studies."

"We believe PRV-3279 has the potential to intercept and prevent the immunogenicity of life-saving gene therapy products and other biotherapeutics," stated Ashleigh Palmer, CEO and co-founder, Provention Bio. "Administration of PRV-3279 has been well-tolerated and pharmacodynamically effective in Phase 1 studies, with linear PK and dose-dependent reduction in B cell activation in the absence of depletion. PRV-3279 has also been shown to reduce B cell responses to viral antigens using experimental vaccine challenge in Phase 1. Given these promising clinical data and the novel pre-clinical data in gene therapy, we look forward to opportunities to work with academic and industry experts to combine PRV-3279 with gene therapy products to further our mission of preventing and intercepting devastating immune-mediated conditions."

The company plans to submit the data from this study for presentation at an upcoming medical conference later in 2021.

About PRV-3279:

PRV-3279 is a humanized diabody (a bispecific DART molecule) targeting the B cell surface proteins, CD32B and CD79B.Simultaneous engagement of the CD32B and CD79B receptors triggers inhibition of B cell function and suppression of autoantibody production, thereby regulating B cells without causing depletion. Provention is initially developing PRV-3279 for the interception of systemic lupus erythematosus (SLE), a chronic autoimmune disorder characterized by an abnormal overactivation of B cells and subsequent pathologic production of auto-antibodies.PRV-3279 also has the potential to prevent or reduce the immunogenicity of biotherapeutics, including but not limited to gene therapy vectors and transgenes.

About Provention Bio, Inc.:

Provention Bio, Inc. (Nasdaq: PRVB) is a biopharmaceutical company focused on advancing the development of investigational therapies that may intercept and prevent debilitating and life-threatening immune-mediated diseases. The Biologics License Application (BLA) for teplizumab, its lead investigational drug candidate, for the delay or prevention of clinical type 1 diabetes in at-risk individuals has been filed by the U.S. Food and Drug Administration (FDA). The Company's pipeline includes additional clinical-stage product candidates that have demonstrated in pre-clinical or clinical studies proof-of-mechanism and/or proof-of-concept in other autoimmune diseases, including celiac disease and lupus. Visit http://www.ProventionBio.comfor more information and follow us on Twitter: @ProventionBio.

Internet Posting of Information:

Provention Bio, Inc. uses its website,www.proventionbio.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation F.D. Such disclosures will be included on the Company's website in the "News" section. Accordingly, investors should monitor this portion of the Company's website, in addition to following its press releases,SECfilings and public conference calls and webcasts.

Forward Looking Statements:

Certain statements in this press release are forward-looking, including but not limited to, statements relating to the Company's studies, the potential safety, health benefits of and planned research and development efforts for PRV-3279. These statements may be identified by the use of forward-looking words such as "anticipate," "believe," "forecast," "estimate," "expect," and "intend," among others. These forward-looking statements are based on Provention's current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to delays in, or failure to obtain FDA approvals or clearances and noncompliance with FDA regulations; the potential impacts of COVID-19 on our business and financial results; changes in law, regulations, or interpretations and enforcement of regulatory guidance; uncertainties of patent protection and litigation; dependence upon third parties; substantial competition; our need for additional financing and the risks listed under "Risk Factors" in our annual report on Form 10-K for the year endedDecember 31, 2019, our quarterly reports on form 10-Q,and any subsequent filings with the Securities and Exchange Commission. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Provention does not undertake an obligation to update or revise any forward-looking statement. The information set forth herein speaks only as of the date hereof.

Partnering:Alex Rabiee, SVP, Business Development & Program Management[emailprotected]908-698-4612 (EXT-118)

Investor Contacts:Robert Doody, VP of Investor Relations[emailprotected] 484-639-7235

Sam Martin, Argot Partners[emailprotected]212-600-1902

Media:Lori Rosen, LDR Communications[emailprotected]917-553-6808

SOURCE Provention Bio, Inc.

Link:
Provention Bio Announces Key Findings from Pre-clinical Proof-of-concept Study for PRV-3279 for the Prevention of Immunogenicity of Gene Therapy -...