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NCATS and FDA Gene Therapy Approaches to Rare Diseases Workshop
August 20 @ 8:00 am - August 21 @ 4:00 pm
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.
To help facilitate discussion among academic, government and industry researchers, NCATS and the Food and Drug Administration’s Center for Biologics Evaluation and Research will co-host “The Growing Promise of Gene Therapy Approaches to Rare Diseases” workshop on Aug. 20-21, 2018. The event will take place at the National Institutes of Health main campus in Bethesda, Maryland. The goals of this two-day workshop include:
- Reviewing the state of current gene therapy approaches;
- Identifying challenges to and strategies for advancing gene therapy research; and
- Discussing how to collaboratively scale and accelerate gene therapy development to benefit patients with rare diseases that currently lack effective treatments.