Guess the Therapy Answer February 11, 2026

Which of Ocugen’s investigational gene therapies is currently in development for Stargardt Disease?

Answer: OCU410ST

See below for further reading on OCU410ST:

Ocugen’s Gene Therapy OCU410ST Continues to Show Efficacy in Phase 1 Stargardt Disease Trial

Ocugen’s investigational adeno-associated virus (AAV)–based gene therapy OCU410ST demonstrated a +6-letter improvement in best-corrected visual acuity (BCVA) among BCVA-evaluable patients without confounders in the phase 1 GARDian1 clinical trial (NCT05956626). These findings, along with additional study results, were published in Eye.

The BCVA analysis included 6 of the 9 patients treated in the study. At 12 months posttreatment, these patients experienced a mean improvement of +4.5 ± 2.20 ETDRS letters in treated eyes, compared with a decline of −1.5 ± 2.33 letters in untreated fellow eyes, corresponding to a net +6-letter gain. Investigators noted that visual acuity in all treated eyes was either stable (±4 letters) or improved (≥5 letters).

EMA's CHMP Gives Ocugen the Green Light to Submit MAA for OCU410ST Based on GARDian3 Trial

The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has provided Ocugen with a positive opinion regarding the use of data from the United States–based phase 2/3 GARDian3 clinical trial (NCT05956626) to support a marketing authorization application (MAA) for OCU410ST, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of Stargardt disease.

The EMA’s decision was based on data from the phase 1 GARDian clinical trial (NCT05956626), in which evaluable treated eyes showed 48% slower lesion growth compared with untreated eyes at 12 months of follow-up. In addition, evaluable treated eyes showed improvement in best-corrected visual acuity (BCVA) of almost 2 lines or 9 letters compared with untreated eyes, a changed deemed clinically meaningful and statistically significant (P = .031).