The Growing Promise of Gene Therapy Approaches to Rare Diseases Workshop

Gene-based therapy is a promising and cutting-edge approach for the treatment of rare diseases. While these tailored therapies offer hope for patients, there are still many unanswered questions.

NCATS and the Food and Drug Administration’s Center for Biologics Evaluation and Research are co-sponsoring a workshop to review the state of current gene therapy approaches, identify challenges and strategies to overcome them, and discuss how to collaboratively scale and accelerate gene therapy development to benefit patients with rare diseases for which there is no effective treatment.

Proposed panel sessions will focus on

  1. pre-clinical development
  2. clinical innovation
  3. manufacturing
  4. partnerships and transitions
  5. business models and patient access
  6. gene therapy as a precision medicine tool


There is no admission fee to attend, and the workshop also will be videocast at is external).

NIH main campus - 10 Center Drive Bethesda, Maryland 20892