Ocugen’s Stargardt Disease Treatment Advances: Green Light for High-Dose Trial

Key points:

  • DSMB approves progression to high-dose cohort in OCU410ST clinical trial for Stargardt disease.
  • Six patients have been dosed in low and medium dose cohorts, with no serious adverse events reported.
  • OCU410ST represents a potential one-time treatment for Stargardt disease, addressing a significant unmet medical need.

Ocugen, Inc. has reached a significant milestone in its clinical trial for OCU410ST, a gene therapy candidate for Stargardt disease. The Data and Safety Monitoring Board (DSMB) has approved proceeding with high-dose cohort dosing in the Phase 1/2 GARDian clinical trial.

The trial has successfully completed dosing in the low and medium dose cohorts, with six patients treated so far. The DSMB’s decision to proceed with the high-dose cohort underscores the favorable safety and tolerability profile of OCU410ST observed to date.

Dr. Peter Y. Chang, DSMB Chair, emphasized the importance of this development, stating, “This is an important next step in the clinical progress for OCU410ST and encouraging for patients living with this most common form of inherited retinal disease.”

Ocugen’s Chief Medical Officer, Dr. Huma Qamar, expressed enthusiasm about the potential of OCU410ST as a one-time treatment for Stargardt disease, which currently has no FDA-approved therapeutic options. The company plans to share a clinical trial update later this year.

The Phase 1/2 GARDian trial will ultimately include up to 42 subjects, assessing the safety and efficacy of OCU410ST across different dosages in both adult and pediatric patients with Stargardt disease.

Human eye.

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