This page compiles media coverage about the PaVe-GT program.

2023
Virtual No More, Rare Disease Day Comes Charging Back to the NIH
The NIH Catalyst. May 4th, 2023. Satabdi Nandi.
NIH Project Aims to Make Gene Therapy ‘Playbook’ Public
MedPage Today. January 18th, 2023. Kristina Fiore.
2022
Families Push Research Forward in Rare Diseases
MedPage Today. December 26th, 2022. Kristina Fiore.
NCATS’ “Many-Diseases-At-A-Time” Approach to Gene Therapy
CheckRare. June 24th, 2022. James Radke.
2020
Gene Therapy: A Researcher’s Perspective
RareCast (Podcast). November 13th, 2020. Daniel Levine.
Individualized Therapeutics Could Get Boost From NIH Platform Gene Therapy Pilot
Pink Sheet. April 3rd, 2020. Sue Sutter.
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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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