The UK Medicines and Healthcare Regulatory Agency (MHRA) has approved GenSight Biologics’ clinical trial application (CTA) to initiate the Phase I/II trial of GS030 to treat patients with retinitis pigmentosa (RP).
PIONEER will examine the safety and tolerability of GS030 in RP patients as part of a multi-centre, open-label dose-escalation study.
It expects to enrol patients affected by end-stage non-syndromic RP with vision not better than ‘counting fingers’.
The trial will include three groups of three subjects, which will each receive an increasing dose of GS030-DP through a single intravitreal injection in their worse affected eye.
A fourth extension cohort is also expected to receive the highest tolerated dose.
PIONEER will also include an independent data safety monitoring board (DSMB) that will review safety information of all treated subjects in each group and make recommendations before proceeding to the next higher dose.
The trial's primary objective will be the safety and tolerability at one-year post-injection, with the first patient expected to be treated in the UK during the first quarter of this year.
GenSight Biologics CEO and co-founder Bernard Gilly said: "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."
GenSight’s GS030 is a combination of a gene therapy (GS030-DP) designed to be administered through a single intravitreal injection and a wearable optronic visual stimulation device (GS030-MD).
The solution has already received orphan drug designation in the US and Europe.