This page compiles news and media about the PaVe-GT program from participating NIH Institutes and external sources.
On the NIH Rare Disease Day, Richa Lomash, PhD, speaks about the PaVe-GT program and its goal of streamlining gene therapies for rare diseases using an AAV-based platform approach.
The NIH Catalyst | May 4, 2023
Rodica Stan, PhD, provides an overview of PaVe-GT’s overarching goals, challenges, and its impact on the rare disease community.
Patient Worthy | April 23, 2023
The PaVe-GT team discusses the regulatory process of receiving an Orphan Drug Designation and a Rare Pediatric Disease designation from the FDA.
NCATS News | March 29, 2023
Elizabeth Ottinger, PhD, and P.J. Brooks, PhD, announce PaVe-GT’s plans to streamline AAV gene therapy development and disseminate project details publicly.
MedPage Today | January 18, 2023
Charles Venditti, MD, PhD, shares his journey in leading the development of AAV gene therapies for rare diseases that don’t attract large pharmaceutical companies.
MedPage Today | December 26, 2022
The PaVe-GT team shares key insights on compiling applications for Rare Pediatric Disease and Orphan Drug Designations.
PaVe-GT News | December 7, 2022
P.J. Brooks, PhD, describes NCATS’ strategy of targeting more than one disease at a time to increase efficiency of developing treatments for rare diseases.
CheckRare | June 24, 2022
PaVe-GT’s investigational gene therapy for the treatment of PCCA-related propionic acidemia has received an Orphan Drug Designation from the FDA.
PaVe-GT News | May 16, 2022
P.J. Brooks, PhD, speaks about the PaVe-GT program’s aim to reduce the time and cost of gene therapy development for a range of rare diseases.
RareCast (Podcast) | November 13, 2020
Insights from the PaVe-GT program could help foster partnerships in manufacturing AAV gene therapies for rare diseases.
Pink Sheet | April 3, 2020
Donald C. Lo, PhD gives an overview of the Platform Vector Gene Therapy (PaVe-GT) program and Philip J. Brooks, PhD gives an overview of the Bespoke Gene Therapy Consortium (BGTC) program. These NCATS-led sister initiatives adopt two distinct approaches to increase the accessibility of gene therapies for patients with rare genetic diseases.
NIH Videocast | March 23, 2022
Philip J. Brooks, PhD explains the Platform Vector Gene Therapy (PaVe-GT) program’s unique approach of streamlining the development of adeno-associated virus (AAV) gene therapies and the reason why the program was launched.
CheckRare YouTube Channel | March 17, 2022
Meet Carsten G. Bönnemann, MD, Senior Investigator, at National Institute of Neurological Disorders and Stroke (NINDS) and Charles Venditti, MD, PhD, Chief, Metabolic Medicine Branch, at National Human Genome Research Institute (NHGRI), and learn about their teams who are developing AAV (adeno-associated virus) gene therapies as part of the Platform Vector Gene Therapy (PaVe-GT) program for four rare genetic diseases.
NCATS NIH YouTube Channel | August 17, 2021
Anne R. Pariser, M.D. gives a brief overview of the NCATS-led Platform Vector Gene Therapy (PaVe-GT) program, which aims to increase the efficiency of gene therapy trial startup by focusing on processes that are common to many rare diseases. By standardizing such processes, PaVe-GT aims to hasten the development of gene therapies for many rare diseases.
NCATS NIH YouTube Channel | March 4, 2021