Comment from Emily's Entourage
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Summary: Emily's Entourage, an organization focused on cystic fibrosis research, supports the FDA's request for comments and proposes a specific workshop topic regarding preclinical data requirements for genetic therapies. They argue that establishing a standardized regulatory framework and master IND protocols would reduce the regulatory burden and accelerate the development of personalized treatments for patients with rare CFTR variants.
Emily’s Entourage (“EE”) is pleased to submit a proposal in response to the Food and Drug Administration’s (“FDA” or “the Agency”) request for comments on FDA Rare Disease Innovation Hub Future Programming. Please see our attached proposal.