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Events

August 2018


August 2018

The Growing Promise of Gene Therapy Approaches to Rare Diseases

Aug. 20-21, 2018

National Institutes of Health (Main Campus)
NIH Clinical Center (Bldg. 10), Masur Auditorium
10 Center Drive
Bethesda, Maryland 20892

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, and (6) gene therapy as a precision medicine tool.

There is no admission fee to attend, and the workshop also will be videocast at https://videocast.nih.gov.

WebsiteAgenda (PDF - 158KB) • Register