NCATS PaVe-GT: Paving the Way for Rare Disease Gene Therapies

The National Institutes of Health (NIH) National Center for Advancing Translational Sciences (NCATS) Platform Vector Gene Therapy (PaVe-GT) pilot project is led by NCATS and includes collaborators at different institutes across NIH.

The goal of this pilot program is to test whether the efficiency of gene therapy clinical trial startup can be significantly increased by using a standardized process, with the same capsid and manufacturing methods, for four different rare diseases. This hypothesis is tested in a transparent manner so that results, documents and learnings resulting from the program will be made publicly available (see: PaVe-GT Resources) and can be used to benefit subsequent AAV gene therapy development.

Note: this project provides a US perspective on gene therapy development.

More information: 

• Other Resources section contains a list of highly relevant resources on gene therapy development for researchers and patient groups.
• The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup