Biopharma company GenSight Biologics (Euronext: SIGHT), a provider of gene therapies for retinal neurodegenerative diseases and central nervous system disorders, on Monday declared favourable safety data and encouraging efficacy signals at one year post-gene therapy administration for the PIONEER Phase I/II clinical trial of GS030 for the treatment of treat retinitis pigmentosa (RP).

The company stated that RP is a genetic blinding disease for which there is currently no treatment. It affects between 15,000 and 20,000 new patients each year in the US and the EU.

This PIONEER first-in-human, multi-centre, open-label dose escalation clinical trial evaluated GS030 in nine RP patients, with a follow-up up to four years (n=1).

GS030 is an optogenetic treatment candidate combining an AAV2-based gene therapy (GS030-DP) with the use of light-stimulating goggles (GS030-MD) in patients with end-stage RP. This therapeutic approach is independent of the causal mutation and therefore applicable to potentially all patients suffering from end-stage RP, the company added.