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    FDA: Flexibility okay for gaps in gene therapy trials

    The US Food and Drug Administration (FDA) is not amenable to changing study endpoints or sample sizes for gene therapy clinical trials but is encouraging sponsors to fill in gaps in data collection with telemedicine and remote visits.   Wilson Bryan, director of the FDA’s Office of Tissues and Advanced Therapies (OTAT) in FDA’s Center for Biologics Evaluation and Research, told a 12 May meeting of the American Society of Gene and Cell Therapy that the pandemic is promp...
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    Euro Convergence: Academic clinics should establish risk management programs for ATMP development

    Academic centers should establish risk management programs to successfully commercialize cell and gene therapy products and to be a viable partner with contract research organizations (CROs) and manufacturers in developing these therapies.   So asserted Lutz Uharek, a professor of internal medicine and founder and CEO of Xencura, at RAPS Euro Convergence 2021 on 12 May in discussing some of the challenges and opportunities for academic centers in developing cell and ge...
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    Drugmakers suggest tweaks to FDA’s neurodegenerative disease gene therapy guidance

    Several drugmakers and Health Canada are suggesting changes to the US Food and Drug Administration’s (FDA) recently released draft guidance on developing gene therapies for neurodegenerative diseases.   Released in January, the draft guidance provides recommendations for the development, testing and clinical study of gene therapies for neurodegenerative diseases. The guidance also discusses considerations for chemistry, manufacturing and controls (CMC) and offers an ov...
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    EMA: Lentiviral thalassemia treatment on hold pending cancer investigation

    The European Medicines Agency (EMA) was notified by a gene therapy firm that the company has suspended marketing of its beta thalassemia treatment Zynteglo (betibeglogene autotemcel, bluebird bio) as a precautionary measure.   In a press release , the agency related that bluebird bio’s actions came after a recipient of the related bb1111 gene therapy for sickle cell disease developed acute myeloid leukemia (AML). Another bb1111 recipient has also developed myelodyspla...
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    FDA guides on COVID considerations in cell and gene therapy

    Manufacturers of cell and gene therapies have a new guidance from the US Food and Drug Administration (FDA) that provides pandemic-related manufacturing considerations.   The guidance specifically addresses both licensed and investigational cell and gene therapy (CGT) manufacture, and “is intended to supplement the recommendations to drug and biological product manufacturers provided in FDA’s ‘Good Manufacturing Practice Considerations for Responding to COVID-19 Infect...
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    FDA offers first thoughts on neurodegenerative disease gene therapies

    The US Food and Drug Administration (FDA) has issued draft guidance on the development, testing, and trial design for human gene therapies for neurodegenerative diseases. The document, released on 5 January 2021, also highlights approval pathways for these novel products.   The draft guidance, which applies to products for both adult and pediatric populations, emphasizes the importance of early communication with FDA before the submission of an investigational new drug...
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    Marks fields questions on cell and gene therapy development

    Expect new guidance from the US Food and Drug Administration (FDA) on the manufacture of CAR-T cells, said Peter Marks, director of the agency’s Center for Biologics Evaluation and Research (CBER).   Marks answered questions on a range of issues related to cell and gene therapy development during a webinar hosted by Genetic Engineering & Biotechnology News .   Saying that CBER is working on new guidelines for CAR T-cell therapy manufacturing, Marks said, “The agen...
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    September’s Regulatory Focus: Countdown to EU MDR and IVDR

    Feature articles during September focused on aspects of the EU Medical Device Regulation (MDR) and In Vitro Diagnostic Regulation (IVDR), with articles on compliance and manufacturer obligations, and perspectives examining the current status of the regulations. The issue also included the second part of the cell and gene therapy series, and an article on regulatory tools for generic drug companies.   MDR and IVDR, close up Full implementation of MDR is slated for ne...
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    Regulation of cell and gene therapy products in China

    This article provides an overview of the Chinese regulations and guidance documents for cell and gene therapy products (CGTPs).   Introduction As early as 1993, Chinese regulators provided guidance on clinical research for somatic cell and gene therapy. More regulations, laws, and guidances have since been published, either specific to, or including, CGTPs. The two most recent publications have been draft guidelines for clinical studies for immune cell therapy produc...
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    Gene therapies – The regulatory road to individualized medicine

    This article discusses the evolving US regulatory landscape for gene therapies. The author explores the recent history and developmental paradigm shifts for gene therapy products and concludes with advice for abbreviated development and thoughts on policies that will create a sustainable gene therapy ecosystem. The author examines the paradigm shift of clinical trial and lifecycle management for gene therapies and considers the changes to the US regulatory framework that ...
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    Regulatory Focus, July issue: Cell and gene therapy

    Feature articles during July focused on global regulatory strategy for cell and gene therapy, with articles on US and EU regulations and guidances and the development and manufacture of the therapies. Also included were articles on recasting the corrective and preventive action (CAPA) process as a continuous improvement process, a military-civilian perspective on real-world evidence (RWE) to support regulatory decision making, and regulatory reporting in multinational tria...
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    Gene therapies: Industry asks for clarification on FDA’s sameness guidance

    Biotech companies and industry groups are raising questions about the US Food and Drug Administration’s (FDA) recent draft guidance on interpreting sameness of gene therapy products under its orphan drug regulations.   The draft guidance, issued for comment in January, explains how FDA intends to determine the sameness of two gene therapies intended for the same use or indication for the purposes of awarding orphan drug designation and exclusivity. (RELATED: FDA fina...