Developing AAV Gene Therapies for BEST1-Associated Inherited Retinal Disease That Ensures IND Success
Time: 4:30 pm
day: Conference Day One
Details:
- Engineering of a safe, efficacious RPE selective AAV vector for delivery of BEST1 in dominant and recessive forms of the disease
- Proof of concept in cellular and large animal models of BEST1
- Development of clinical endpoints for a P1/2 BEST1 clinical trial
