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Literature and Guidance Documents

Regulatory issues in human gene therapy. Blood Cells Mol Dis 2003; 31: 51–56. This paper will review the current oversight agencies and identify areas of evolving regulations that pose particular challenges to gene therapy investigators.

Regulatory issues for clinical gene therapy trials. Hum Gene Ther. 2002; 13(10):1143-9. In this review, the basic tenets of human subject research are discussed in the context of the regulatory bodies which oversee this work. The challenges faced by academic research are outlined, including new and proposed regulations which impact human gene therapy investigators.

NIH Guidelines for Research Involving Recombinant DNA Molecules (September, 2009)

FDA Guidance for Industry: Gene Therapy Clinical Trials - Observing Subjects for Delayed Adverse Events (November 28, 2006)

FDA Guidance for Industry: Supplemental Guidance on Testing for Replication Competent Retrovirus in Retroviral Vector Based Gene Therapy Products and using Follow-up of Patients in Clinical Trials Using Retroviral Vectors (November 28, 2006)

The Sponsor’s Guide to Regulatory Submissions For an Investigational New Drug. A practical guide for bench Scientists entering the clinical and regulatory realm (March, 2005)

 

FDA - CBER News

  • What’s New for Biologics
    21 June 2024
    Latest news from the Center for Biologics Evaluation and Research
  • Jobs at the Center for Biologics Evaluation and Research (CBER)
    21 June 2024
    CBER has unique opportunities for individuals with backgrounds in science or administration to fill positions involved in the regulation and research of blood, vaccines and therapeutics.
  • ELEVIDYS
    20 June 2024
    Treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.
  • FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy
    20 June 2024
    The FDA expanded the approval of Elevidys, a gene therapy for the treatment of Duchenne muscular dystrophy for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene.
  • International Activities
    20 June 2024
    CBER’s international activities can be categorized in the following functional areas: regulatory harmonization, regulatory capacity building, information sharing, international standards setting, and collaborative research.