GenSight Biologics has enroled the first patient in the Phase I/II PIONEER clinical trial to assess the safety and tolerability of GS030 for the treatment of patients with retinitis pigmentosa.
The subject has also been treated at the Moorfields Eye Hospital in London, UK.
GenSight is expected to enrol 18 patients as part of the first-in-man, open-label, dose-escalation trial, with registration expected to close by the last quarter of next year.
The first three cohorts, consisting of three subjects each, will include patients affected by end-stage non-syndromic retinitis pigmentosa with no light perception (NLP) or light perception (LP) level of visual acuity.
The trial’s extension cohort will comprise less severe patients, with hand motion (HM) and counting fingers (CF) levels of visual acuity.
A fourth extension cohort will also be added to receive the highest tolerated dose.
GenSight's GS030 combines a gene therapy, GS030-DP, which can be given via a single intravitreal injection with a wearable optronic visual stimulation device, GS030-MD.
During the multi-centre trial, patients will receive an increasing dose of GS030-DP in their worse affected eye.
Safety data of the trial will be reviewed by an independent data safety monitoring board (DSMB) to make recommendations on whether to move to the next dose.
The trial’s primary outcome analysis is the safety and tolerability at one-year post-injection.
GenSight plans to report early findings for the first cohort in the first half of next year and topline results from the trial by the last quarter of 2020.