12 January 2018 - - The UK Medicines and Healthcare Regulatory Agency (MHRA) has accepted Paris, France-based clinical-stage biopharma company GenSight Biologics' (Paris, Euronext: SIGHT) clinical trial application to initiate the PIONEER Phase I/II study of GS030 in patients with retinitis pigmentosa, the company said.
PIONEER is a first-in-man, multi-center, open label dose-escalation study to evaluate the safety and tolerability of GS030 in subjects affected by end-stage non-syndromic retinitis pigmentosa with vision not better than "counting fingers."
GS030 is the combination of a gene therapy (GS030-DP) administered via a single intravitreal injection, and a wearable optronic visual stimulation device (GS030-MD).
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 will review safety data of all treated subjects in each cohort and make recommendations before moving to the next higher dose.
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.
GenSight Biologics is focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders.
Its pipeline leverages two core technology platforms, the mitochondrial targeting sequence and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases.