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BIO Comments on FDA Draft Guidance on Human Gene Therapy for Retinal Disorder and Hemophilia

November 14, 2018

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance on Human Gene Therapy for Retinal Disorder and Human Gene Therapy for Hemophilia.

BIO says the guidance will assist stakeholders developing human gene therapy (GT) products to treat rare diseases, including hemophilia and retinal disorders, and provided specific suggestions for the FDA to clarify both guidances. The topics discussed in BIO's comments include preclinical in vitro and in vivo proof-of-concept studies and disease-specific animal models, science-based regulatory flexibility when evaluating appropriateness of animal models, the use of the word "pharmacokinetics," biodistribution studies, reproductive/developmental toxicity studies, fit-for-purpose clinical trial designs, and patient experience.

Download Full Comments Below
FINAL BIO Letter GT For Hemophilia And GT For Retinal Disorder 12-10-18
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