Published in Pipeline

GenSight Biologics releases 1-year data from phase 1/2 trial of RP treatment

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GenSight Biologics S.A. announced the release of data from its PIONEER phase 1/2 clinical trial at 1-year post-gene therapy administration of GS030 for the treatment of retinitis pigmentosa (RP).

Tell me more about GS030.

GS030 is a combination of optogenetic and adeno-associated virus (AAV2)-based gene therapy (GS030-DP) that is administered via a single intravitreal injection. The therapy incorporates GenSight’s optogenetics technology platform—which uses ocular gene therapy and tailored light-activation of treated retinal cells—through the use of light-stimulating goggles (GS030-MD).

Anything else?

The FDA granted GS030 Orphan Drug designation in 2017 and Fast Track designation in 2021 for the treatment of RP.

Talk about the trial.

The PIONEER study is a first-in-human, multicenter, open-label dose escalation trial assessing the tolerability and safety of GS030 in patients with end-stage RP. Patients were separated into three cohorts and each administered one of three doses of GS030-DP—5e10 vg; 1.5e vg; and 5e11 vg—via an intravitreal injection into their worst RP-affected eye.

A Data Safety Monitoring Board (DSMB) assessed all treated patients’ safety data to determine which dosage to use for the extension study, selecting the highest dose (5e11 vg).

What were the findings?

Results from the three cohorts noted only mild and moderate ocular adverse effects (AEs), with the most common being mild intraocular inflammation (70%) in response to corticosteroid treatment.

A follow-up extended to 4 years (n = 1).

GS030-MD (light-stimulating goggles) was first used 8 weeks after injection and well tolerated in all patients.

What else?

One-year post-injection data from patients in the 5e11 vg dose cohort allowed investigators to assess efficacy signals for all three cohorts; a portion of patients’ vision improved significantly, with the best results seen with those receiving the highest dose.


With no current treatment approved for RP, the PIONEER’s 1-year data could provide hope and encouragement for patients suffering from this condition. GenSight expects to receive further data from the 4-year extension cohort, which is under recruitment.