Ocugen Doses First Patient in Phase 2/3 GARDian3 Trial for Stargardt Disease Gene Therapy

Ocugen Inc, a biotech innovator in gene therapies for blindness, has announced a major milestone—the first patient has been dosed in its Phase 2/3 GARDian3 clinical trial for OCU410ST, a modifier gene therapy designed to treat all forms of Stargardt disease.

Why This Matters

• Stargardt disease affects over 1 million people globally, with 100,000 cases in the US and Europe.
• No approved treatments currently exist for this progressive form of inherited macular degeneration.
• OCU410ST is designed to be a one-time therapy for life, offering hope to families long without options.

A Pivotal Milestone

“Dosing the first patient is an especially significant milestone and brings us closer to addressing the unmet need that exists for all Stargardt patients,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen.

The Phase 2/3 trial builds on strong data from the Phase 1 GARDian study, where:

• 48% slower lesion growth was observed at 12 months in treated eyes vs untreated.
• Treated eyes showed a clinically meaningful 2-line gain in visual acuity.
• Results were statistically significant (p=0.031).

Key Features of the GARDian3 Trial

• Study Design:
  • Total of 51 participants diagnosed with Stargardt disease.
  • 34 will receive OCU410ST via one-time subretinal injection in the eye with poorer vision.
  • 17 will serve as an untreated control group.
• Primary Objective:
  • Measure reduction in atrophic lesion size.
• Secondary Endpoints:
  • Improvement in:
    • Best Corrected Visual Acuity (BCVA)
    • Low luminance visual acuity
• Dosing Details:
  • 200 µL injection at a concentration of 1.5 × 10¹⁰ vector genomes/mL.
• Trial Design:
  • Adaptive protocol includes a masked interim analysis at 8 months on 24 subjects to assess early efficacy and safety signals.

Expert and Community Reactions

“This trial brings us closer to a one-time gene therapy that could transform quality of life for years to come,” said Dr. Victor H. Gonzalez, Principal Investigator, Valley Retina Institute.

“We’re efficiently optimizing study conduct to generate meaningful data for regulatory approvals,” added Dr. Huma Qamar, CMO of Ocugen.

Safety and Regulatory Outlook

• No serious adverse events reported so far.
• OCU410ST shows a favorable safety and tolerability profile, with no signs of:
  • Ischemic optic neuropathy
  • Vasculitis
  • Intraocular inflammation
  • Endophthalmitis
  • Choroidal neovascularization
• The data will support a planned Biologics License Application (BLA) submission in 2027, as part of Ocugen’s goal to file three BLAs over the next three years.

How the Therapy Works?

• OCU410ST uses an AAV (adeno-associated virus) delivery system for retinal administration.
• It introduces the RORA gene (RAR-Related Orphan Receptor A), a nuclear hormone receptor that modulates key pathways involved in Stargardt disease:
  • Lipofuscin buildup
  • Oxidative stress
  • Inflammation
  • Complement formation
  • Photoreceptor cell survival

About Stargardt Disease

• A genetic retinal disorder, it leads to progressive loss of central vision.
• Caused by degeneration of photoreceptor cells in the macula—the central part of the retina.
• Peripheral vision often remains intact.
• Typically begins in childhood or adolescence, but the rate of progression varies.
• The retinal pigment epithelium (RPE), which supports photoreceptors, is also affected.

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