• Regulatory NewsRegulatory News

    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...
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    Following Negative Comments by Reviewer, EU Committee OK's Gene Therapy

    Last month, German regulators postponed their review of UniQure's gene therapy Glybera after the European Medicines Agency (EMA) rapporteur assigned to the product claimed new data showed Glybera lacked efficacy and called for it to be reassessed . Now, EMA's Committee for Advanced Therapies (CAT) has completed its assessment, and is maintaining its positive opinion of Glybera, Reuters reports. Background Glybera is the first gene therapy approved for use in the E...
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    First EU-Authorized Gene Therapy Hits Snag, Benefit Assessment Delayed in Germany as EMA Reviews New Report

    Regulators at the German Federal Joint Commission (G-BA) have postponed their review of the gene therapy Glybera, Reuters reports . The decision was made after the European Medicines Agency (EMA) rapporteur assigned to Glybera found the product lacked efficacy and called for it to be reassessed. Glybera, which treats a rare condition called lipoprotein lipase deficiency (LPLD), made headlines both for being the first gene therapy approved in Europe and for its record-...
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    Are Biotechnology Products Safe for the Environment? FDA's New Policy Asks for Data

    While the US Food and Drug Administration (FDA) may be primarily concerned with what medicinal products do to the human body, a lesser-known secondary concern of regulators is what a product will do to the environment. Background Under 21 CFR 25.40 of the  Code of Federal Regulations , most pharmaceutical and biotechnology companies are required to submit an Environmental Assessment (EA) when submitting a new drug for approval. The assessments are meant to show how a...
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    FDA Wants Sponsors of Biotech Products to Assess Product's Impact on Environment

    The US Food and Drug Administration's (FDA) Center for Biologics Evaluation and Research (CBER) has released a new draft guidance document meant to clarify when companies need to conduct an Environmental Assessment (EA) for gene therapy and other recombinant products. Background Under 21 CFR 25 of the Code of Federal Regulations , most pharmaceutical and biotechnology companies are required to submit EAs as part of their approval package. The assessments are gene...
  • Cell and Gene Therapy Guidance Finalized by FDA, Calls for Early and Frequent Communication

    The US Food and Drug Administration (FDA) has announced the final release of a guidance intended to clarify the processes by which cellular and gene therapy (CGT) products should be assessed prior to human testing. Background CGTs are essentially used to make therapeutic changes to a person's cells, tissue or genes by injecting a patient with altered DNA, cells or entire organs. The products include cellular therapies, gene therapies, therapeutic vaccines, xenotransplan...
  • FDA Extends Comment Period on Cell and Gene Therapy Guidance by Six Months

    Manufacturers of cell therapy (CT) and gene therapy (GT) products will have six months of additional time to comment on a July 2013 draft guidance released by the US Food and Drug Administration (FDA), regulators announced today. Background In July 2013, FDA's Center for Biologics Evaluation and Research (CBER) released a draft guidance it said would help industry in designing early-stage clinical trials for CTs and GTs. The guidance was specifically aimed at investiga...
  • Safety Main Challenge to Preparing INDs for Cellular and Gene Therapy Products, Says FDA

    The US Food and Drug Administration (FDA) has released a new draft guidance document intended to outline the considerations industry should take into account when designing early-phase clinical trials for cellular therapy (CT) and gene therapy (GT) products. Background The draft guidance, released on 2 July 2013 by FDA's Center for Biologics Evaluation and Research (CBER), is specifically targeted at investigational new drug applications (INDs), which are used by compan...