NIH Gene Therapy Team Reveals Its Path to FDA Orphan Drug and Rare Pediatric Disease Designations

March 30, 2023

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Pave-GT Resources
Rare Pediatric Disease (RPD) Designation Request for AAV9-hPCCA

This pdf file contains the RPD designation request for AAV9-hPCCA (NCATSBL-0746) and associated communications between NCATS and FDA OOPD.

March 21, 2023
Orphan Drug Designation (ODD) Request for AAV9-hPCCA

This pdf file contains the ODD request for AAV9-hPCCA (NCATSBL-0746) and associated communications between NCATS and FDA OOPD.

March 21, 2023
Rare Pediatric Disease (RPD) Designation Request Template Read More
February 27, 2023
Orphan Drug Designation (ODD) Request Template Read More
February 27, 2023
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