On July 11, the FDA released a total of 6 guidance documents intended to help advance the field of gene therapy. This includes three new draft guidance documents that address the development of gene therapy products for specific disease categories (Hemophilia, Retinal Disorders and Rare Diseases) and updates to three existing guidances that address manufacturing issues related to gene therapy.
Statement from FDA Commissioner Scott Gottlieb, M.D. on agency’s efforts to advance development of gene therapies
Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs); Draft Guidance for Industry (PDF - 486KB)
Long Term Follow-up After Administration of Human Gene Therapy Products; Draft Guidance for Industry (PDF - 294KB)
Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Draft Guidance for Industry (PDF - 124KB)
Human Gene Therapy for Hemophilia; Draft Guidance for Industry (PDF - 371KB)
Human Gene Therapy for Rare Diseases; Draft Guidance for Industry (PDF - 136KB)
Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry (PDF - 172KB)
View the most recent NA LRA Regulatory WatchDog Report from the June Telegraft issue.
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