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Debbie Drell: FDA wants to hear your perspective on individualized cellular and gene therapies (CGTs)
Nov 24, 2023, 13:49

Debbie Drell: FDA wants to hear your perspective on individualized cellular and gene therapies (CGTs)

Debbie Drell, Senior Director of Membership at National Organization for Rare Disorders, shared on LinkedIn:

“Scientific Challenges and Opportunities To Advance the Development of Individualized Cellular and Gene Therapies; FDA Request for Information.

**FDA comment period ends 11/20/23**

FDA wants to hear your perspective on individualized cellular and gene therapies (CGTs) and the formal public comment period is ending soon! CGTs are the hope for small rare disease populations but they are extremely expensive and difficult to make.

The FDA seeks public feedback about current challenges and opportunities in the development of CGTs.

How can patient groups, sponsors, and researchers collaborate effectively to move this field forward? What are your perspectives on data from animal models, computational simulations, or tissue-culture in lieu of or in addition to some clinical data?

What can patient groups do to help prioritize investments in CMC to streamline processes and make these products faster, cheaper, and more reliable to manufacture?

What else should FDA be thinking about in regulating CGTs? Submit comments here.

For more details click here.
Source: Debbie Drell/LinkedIn