The UK Medicines and Healthcare Regulatory Agency has signed off on GenSight Biologics' (NASDAQ:GNST) Clinical Trial Application for a Phase 1/2 study assessing GS030 in patients with retinitis pigmentosa (RP), the leading cause of hereditary blindness in the world.
The trial will involve three cohorts of three subjects each who will receive an increasing dose of GS030-DP via a single intravitreal injection in their worse eye. A fourth extension cohort will receive the highest tolerated dose. The primary endpoint is safety and tolerability at one year post-injection.
Orphan Drug-tagged GS030 is a gene therapy that introduces a light-sensitive protein into specific cells in the retina. Treated patients will wear an external device that amplifies the light signal by stimulating the transduced cells thereby further enhancing vision.
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