Placeholder Banner

BIO Comments on FDA Draft Guidance on Human Gene Therapy for Retinal Disorder and Hemophilia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BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance on Human Gene Therapy for Retinal Disorder and Human Gene Therapy for Hemophilia.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
Read full comment letter below
Discover More
The Biotechnology Innovation Organization (BIO) is pleased to respond to the U.S. Department of Agriculture’s (USDA) Solicitation of Input from Stakeholders on Agricultural Innovations. BIO applauds USDA for taking a proactive approach and seeking…
July 31, 2020 Re: Docket No. FDA–2020-N-0837: Raare Disease Clinical Trial Network; Request for Information. Dear Sir/Madam: The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the…
July 30, 2020 Re: Docket No. FDA–2019-D-5799: FDA’s Public Meeting, Modernizing the Food and Drug Administration’s Data Strategy. Dear Sir/Madam: The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA or…