Ocugen Shares Surge on Promising Gene Therapy Trial Results

Ocugen US67577C1053

Investors in Ocugen, Inc. have received the positive clinical data they were anticipating. The biopharmaceutical company's stock price jumped approximately 19% following the release of encouraging one-year results from its Phase 1 trial for OCU410ST, an investigational gene therapy targeting Stargardt disease. This inherited form of macular degeneration currently has no approved treatment.

The 12-month data from the GARDian1 study were published in the peer-reviewed journal Nature Eye. According to the findings, patients treated with OCU410ST demonstrated both structural and functional improvements in the eye.

Key outcomes from the trial include:
* A 54% reduction in the growth of atrophic lesions in treated eyes versus untreated eyes.
* The rate of lesion spread was halved, measuring 0.10 mm per year compared to 0.19 mm per year in untreated eyes.
* Every treated eye exhibited either stabilization or an improvement in visual acuity.
* On average, treated eyes gained 6 letters in best-corrected visual acuity, while untreated eyes lost 1.5 letters.
* Importantly, the therapy was well-tolerated with no serious adverse events linked to the drug.

A parallel Phase 2/3 trial, named GARDian3, is already underway. Its timeline has been accelerated, with completion now expected by the first quarter of 2026. If successful, this could pave the way for a regulatory submission in the first half of 2027.

Financial and Market Outlook

The addressable patient population for Stargardt disease is significant, with over 100,000 individuals affected across the United States and Europe. This represents a substantial market opportunity for an effective therapy. Reflecting this potential, market analysts maintain a positive outlook on Ocugen's equity. The consensus average price target stands at $9.00, implying an upside of roughly 400% from recent trading levels.

The company strengthened its balance sheet with a $20 million financing round completed in the third quarter of 2025. Beyond OCU410ST, Ocugen's pipeline includes two other gene therapy candidates: OCU400 for retinitis pigmentosa and OCU200 for diabetic macular edema. The firm is scheduled to report its next quarterly financial results on March 3.