The Growing Promise of Gene Therapy Approaches to Rare Diseases by National Center for Advancing Translational Sciences (NCATS)

The Growing Promise of Gene Therapy Approaches to Rare Diseases is organized by National Center for Advancing Translational Sciences (NCATS) and will be held from Aug 20 - 21, 2018 at National Institutes of Health campus, Bethesda, Maryland, United States of America.

Conference Description :
NCATS and the U.S. 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.

The proposed sessions will focus on:
• Pre-clinical development.
• Clinical development.
• Manufacturing.
• Partnerships and transitions.
• Business models and patient access.
• Ensuring that no patient and no disease is left behind.

Additional details will be posted as soon as they are available.