Ocugen Publishes Positive Phase 1 GARDian1 Trial Results for OCU410ST Gene Therapy

Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company focused on gene therapies for blinding diseases, announced the publication of positive Phase 1 GARDian1 clinical trial results for its experimental modifier gene therapy, OCU410ST, in the peer‑reviewed journal Nature Eye. The data represent the first human trial evaluating safety, tolerability, and exploratory efficacy of the therapy in adults with Stargardt disease, the most common form of inherited macular degeneration, which currently has no approved treatments. In the small cohort, treated eyes demonstrated a 54 % reduction in atrophic lesion growth over 12 months compared with untreated fellow eyes and 50 % slower lesion expansion, falling below natural history progression rates. Best‑corrected visual acuity (BCVA) improved by an average of +6 letters in treated eyes versus a −1.5‑letter decline in untreated eyes, with all treated eyes stabilizing or improving. No drug‑related serious adverse events were observed, supporting a favorable safety profile. OCU410ST uses an AAV5 vector to deliver the RORA gene to retinal cells, targeting multiple disease mechanisms independent of the patient’s ABCA4 mutation. Ocugen says the results validate its modifier gene therapy approach and support ongoing development. The Phase 2/3 GARDian3 trial continues enrolling, with anticipated completion in Q1 2026 and a potential Biologics License Application (BLA) filing in H1 2027. #Ocugen #GeneTherapy #StargardtDisease #ClinicalTrialSuccess #SlimScan #GrowthStocks #CANSLIM