Can We Streamline How AAV Gene Therapies Are Developed?

  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...

Panel Discussion: The High Cost of Rare Diseases  

  Joni Rutter, PhD, Acting Director at the National Center for Advancing Translational Sciences (NCATS) and Annie Kennedy, Chief of Policy and Advocacy at the EveryLife Foundation discuss their organizations’ collective studies showing the direct and...