Gene therapy pipeline accelerates as Ocugen, Inc. targets approvals for RP and Stargardt disease

Gene therapy pipeline accelerates as Ocugen, Inc. targets approvals for RP and Stargardt disease
Ocugen, Inc. targets gene therapy approvals

Ocugen, Inc. is accelerating development of potentially one-time gene therapies targeting inherited retinal diseases such as retinitis pigmentosa (RP), Stargardt disease, and geographic atrophy (GA).

Speaking at the Retina World Congress in Fort Lauderdale, CEO Dr. Shankar Musunuri outlined the company’s goal to achieve three Biologics License Application (BLA) filings by 2028. The initiatives aim to bring transformative therapies to patients with severe vision disorders, leveraging advanced gene editing and delivery technologies. Ocugen, Inc.’s progress suggests a shift toward more durable and long-lasting treatments in the retina disease landscape.

Ocugen previously detailed plans to pursue three Biologics License Applications for blindness gene therapies by 2028, seeking broader patient access through a mass-market strategy. Company executives also presented updates on gene therapy platform developments and regulatory timelines at major biotech events earlier this year, according to company disclosures in April. These actions reflect Ocugen’s continued focus on advancing regulatory milestones for inherited retinal disease treatments.

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