Ocugen Aims for 2027 BLA Submissions as Gene Therapy Initiatives Approach Crucial Milestones

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Ocugen Inc. (NASDAQ: OCGN), a clinical-stage biopharmaceutical company based in Malvern, Pennsylvania, is advancing its ophthalmology gene therapy portfolio focused on inherited retinal diseases and dry age-related macular degeneration. CEO Shankar Musunuri indicated that the company aims to submit biologics license applications (BLAs) next year for its programs targeting retinitis pigmentosa and Stargardt disease, with potential approvals anticipated in 2028.

The Phase II/III trial for Ocugen’s Stargardt disease candidate, OCU410ST, is currently enrolling patients aged 3 and older, with expected top-line data in Q2 2024. For retinitis pigmentosa, results from the Phase III trial of OCU400 involving 140 patients are expected in Q1 2024. To date, Ocugen has treated over 325 patients across its clinical trials, reporting no serious adverse events related to its gene therapies.

In funding news, Ocugen secured $130 million in convertible note financing to support operations through 2028, with plans to explore regional partnerships and non-dilutive funding opportunities to aid in commercialization efforts.

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