GenSight Biologics is a biopharma focussed on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. The French biotech has now announced UK Medicines and Healthcare Regulatory Agency (MHRA) acceptance of the Company’s Clinical Trial Application (CTA) to initiate the PIONEER Phase I/II study of GS030 in patients with Retinitis Pigmentosa (RP).
The first-in-man, multicenter, open-label, dose-escalation study will evaluate the safety and tolerability of the therapy, which utilizes GenSight’s optogenetics technology platform to introduce gene encoding for a light-sensitive protein into specifically targeted retinal cells.
As per protocol, three cohorts of three subjects each will be administered an increasing dose of GS030-DP via a single intravitreal injection in their worse affected eye. A fourth extension cohort will receive the highest tolerated dose. An independent Data Safety Monitoring Board (DSMB) will review safety data of all treated subjects in each cohort and make recommendations before moving to the next higher dose.
Bernard Gilly, CEO and co-founder of GenSight, commented, “I am particularly excited to have GS030 moving into the clinic. This represents a major milestone for the Company. For the first time ever in ophthalmology, an optogenetic approach combined with a medical device will be tested in humans. If proven safe and effective, this therapy could be transferable from Retinitis Pigmentosa to dry-AMD.”
The primary outcome analysis will be the safety and tolerability at one year post-injection. GenSight expects to treat the first patient in the United Kingdom in the first quarter of 2018.