P.J. Brooks, PhD, Acting Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), describes the Platform Vector Gene Therapy (PaVe-GT) pilot project.

As Dr. Brooks explains, adeno-associated virus (AAV) gene therapies use a modified version of AAV to deliver a working copy of a defective gene into the relevant cells for a given disease, including cells in the eye, liver, brain, muscle and other organs. AAV gene therapy is a promising platform for many single-gene rare diseases and recently the Food and Drug Administration (FDA) have approved AAV gene therapies for a number of diverse rare diseases. 

In theory, the testing of new AAV gene therapies for safety and efficacy could be streamlined during preclinical investigations to save both time and money during the final clinical stages. 

Dr. Brooks goes on to explain that the goal of NCATS’ PaVe-GT pilot program is to test whether the efficiency of gene therapy trial startup can be significantly increased by using a standardized process, with the same capsid and manufacturing methods, for four different rare diseases. NCATS plans to make the results of the program publicly available so that other researchers can use the information to develop more AAV gene therapies.

To stay up to date with the latest news in rare diseases, sign up for one of our monthly newsletters