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Human Gene Therapy for Rare Diseases

Origin/Publisher:

FDA, Office of Communication, Outreach and Development (OCOD), 10903 New Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring,

Document Type:
Draft Guidance
Content:
This guidance provides recommendations to stakeholders developing a human gene therapy (GT) product1 intended to treat a rare disease2 in adult and/or pediatric patients regarding the manufacturing, preclinical, and clinical trial design issues for all phases of the clinical development program. Such information is intended to assist sponsors in designing clinical development programs for such products, where there may be limited study population size and potential feasibility and safety issues, as well as issues relating to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the GT product itself.

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