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    EMA to Continue Adaptive Pathways Program as Pilot Ends

    The European Medicines Agency (EMA) on Wednesday published a final report on its experience from its adaptive pathways pilot project, noting that a few aspects of the program need to be further improved and refined moving forward.  Though the vast majority of applications for the pilot were rejected, EMA said that moving forward, the agency needs to further integrate the patient voice into the program and refine the definition of methodologically sound strategies for col...
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    European Commission to Overhaul Concept of ‘Similar’ Medicines Within Context of Orphan Legislation

    The European Commission on Friday proposed to review the concept of “similar medicinal products” in the context of its orphan legislation as part of a wider effort to adapt the text to technical progress. Fifteen years after the implementation of the orphan legislation, the commission says it is currently launching initiatives to improve the implementation of the regulatory framework with a view to ensuring timely access to medicinal products.  In this context, the Comm...
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    EC Report Analyzes Regulatory Frameworks for Advanced Therapies in US, Canada, Japan and Korea

    The European Commission report released Wednesday dissects the similarities in how gene-, cell- and tissue-based advanced therapies are regulated across the four different geographic regions, though ongoing research projects in such therapies are heavily concentrated in the US and Japan. The 327-page report highlights the “high degree of convergence” in the regulation of advanced therapies across the US, Canada, South Korea and Japan, noting that only Japan has enacted (...
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    Advanced Therapies: Stakeholders Call for EMA Changes on Regulations, Manufacturing

    The European Medicines Agency (EMA) on Friday released a report outlining the concerns and regulatory changes described by developers, manufacturers and investors in advanced therapy medicinal products (ATMPs), which include gene therapies, tissue-engineered products and somatic cell therapies, at a meeting last week. The report notes that recurring themes included the need for early interaction with regulators and guidance, more transparency, greater harmonization betwe...
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    FDA Looking for Two New Cellular and Gene Therapy Fellows

    • 28 March 2016
    The US Food and Drug Administration’s Division of Cellular and Gene Therapies is opening up two contract fellowship positions: one to study the potential of human pluripotent stem cells for developing gene and cell therapies, and one to further investigate mechanisms that RNA viruses use to inhibit host immune cell function. Candidates with research experience in virology, immunology, cell biology, molecular biology or gene therapy will receive the strongest consideratio...
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    FDA Sees Spike in Gene and Cell Therapy Applications

    The US Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) met Tuesday to discuss updates of research programs in the Office of Cellular, Tissue and Gene Therapy (CTGT), which is part of FDA’s Center for Biologics Evaluation and Research (CBER). The research updates came as Raj Puri, MD, PhD, Director, Division of Cellular, and Gene Therapies (DCGT) at CBER told the committee that although his division has only licensed 12 prod...
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    Senate Bill Seeks to Create Standards Body for Regenerative Medicine

    Sen. Tammy Baldwin (D-WI) introduced a bill on Tuesday to promote the development of regulatory science and standards for regenerative medicine and advanced therapies. The bill, known as the Advancing Standards in Regenerative Medicine Act , would require the US Food and Drug Administration (FDA) to oversee the creation of a "Standards Coordinating Body" for regenerative medicine. Regenerative medicine and advanced therapies belong to a growing field of medicine that i...
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    FDA Authorizes First Human Study to Use In Vivo Genome Editing Application

    The US Food and Drug Administration (FDA) broke new ground last week when it authorized the world’s first human clinical trial for an  in vivo  genome editing application. The investigational new drug (IND) application approval for California-based Sangamo, which is the first and only company to put a gene editing therapy into clinical trials, allows the company to begin conducting in 2016 a Phase 1/2 open-label, dose-escalation study in up to nine male adults with sever...
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    Human Gene Editing, CRISPR and FDA: How Will They Mix?

    A number of the world’s top scientists, policy experts and bio-ethicists met this week in Washington, DC to discuss the implications of human gene editing, and though the focus of the summit was centered more on ethical considerations than regulations, opinions flew on Wednesday over how some new technologies will likely be regulated. Background As Jennifer Doudna, a molecular and cell biologist at University of California, Berkeley, and a leader in the field notes in a...
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    Cancer Gene Therapies: FDA Officials Highlight Regulatory Approaches

    As gene therapies begin to get a foothold among other cancer treatments, the US Food and Drug Administration (FDA) is taking a more flexible, data-driven approach for the preclinical testing programs of these biologically complex products, according to a new review from five FDA officials in Cancer Gene Therapy . Although FDA has yet to approve a gene therapy to treat cancer, the authors note that about two-thirds of gene therapy clinical trials are for cancer treatment...
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    NGS Altering the Landscape of Antiviral Drug Resistance Analysis, FDA Officials Say

    As next-generation sequencing (NGS) costs gradually decline, the Food and Drug Administration’s (FDA) Division of Antiviral Products (DAVP) anticipates that more companies will make the switch to NGS for future antiviral drug resistance analyses and other additional uses. The push to more NGS use comes as DAVP independently assessed NGS resistance data for three new drug applications (NDAs), which taught the agency a few important lessons and will help it prepare for the...
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    EMA Issues New Guideline on Gene Therapy

    The European Medicines Agency (EMA) is seeking feedback on a new draft guideline aimed at clarifying the scientific evidence necessary to support the authorization of new gene therapies. Background Gene therapy works by modifying a patient's genes to correct for genetic issues. Researchers have been studying gene therapy for the past 30 years, but few products have reached advanced stages of development. Because so few gene therapies have been submitted for authorizatio...