The Medicines and Healthcare Products Regulatory Agency in the United Kingdom has accepted a clinical trial application from GenSight Biologics for the PIONEER phase 1/2 study of its gene therapy GS030 for patients with retinitis pigmentosa, according to a company press release.
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, the release said.
“I am particularly excited to have GS030 moving into the clinic. This represents a major milestone for the company,” Bernard Gilly, GenSight CEO and co-founder, said in the release. “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.”
Three cohorts of three subjects will be administered an increasing dose of the therapy with a single intravitreal injection, while a fourth extension cohort will receive the highest tolerated dose, the release said.