Ocugen Targets 2027 BLA Filings as Gene Therapy Programs Near Key Readouts

Ocugen is targeting 2027 BLA filings for its OCU400 retinitis pigmentosa and OCU410ST Stargardt disease gene therapies, with potential launches as early as 2028 if development stays on track.

Key clinical readouts are expected next year: Phase III OCU400 data in the first quarter and Phase II/III OCU410ST data in the second quarter.

The company says it has treated more than 325 patients across its programs without observing serious treatment-related adverse events.

A $130 million convertible-note financing is expected to fund operations into 2028, while Ocugen evaluates regional partnerships and non-dilutive funding; additional equity financing could be considered to support U.S. commercialization.

Ocugen NASDAQ: OCGN is positioning its ophthalmology gene therapy portfolio around treatments for inherited retinal diseases and dry age-related macular degeneration, with pivotal-stage programs in retinitis pigmentosa, Stargardt disease and geographic atrophy, Chairman, CEO and Co-Founder Shankar Musunuri said during a Canaccord discussion.

Musunuri said the company is targeting biologics license applications, or BLAs, next year for its retinitis pigmentosa and Stargardt programs, while its geographic atrophy program is expected to follow on a longer timeline.

He said the company’s objective is to pursue approvals in major markets, including the U.S., Europe and Japan, and to improve patient access in parallel with regulatory work.

Ocugen’s approach differs from gene-specific therapies by using “modifier genes,” which Musunuri described as genes that regulate networks of functions in retinal cells.

The company’s technology originated from work by Dr. Neena Haider at Harvard, according to Musunuri.

For retinitis pigmentosa, Ocugen is developing OCU400, which uses the Nr2e3 modifier gene. Musunuri said Nr2e3 affects phototransduction pathways, which are particularly relevant in retinitis pigmentosa as patients can lose peripheral and night vision before central vision.

For Stargardt disease and geographic atrophy, Ocugen is developing treatments using the RORA modifier gene. Musunuri said RORA is intended to regulate multiple disease-related pathways, including oxidative stress, lipid metabolism, inflammation and the complement system. He said the company believes the therapy may help restore cellular homeostasis and create a healthier environment for retinal cells.

Musunuri said Ocugen’s intellectual property extends beyond ophthalmology into neurological applications, though he characterized that area as a future opportunity.

OCU410ST, the company’s Stargardt disease candidate, is in a Phase II/III study enrolling patients ages 3 and older, from early through advanced stages of disease.

Musunuri said the study was endorsed by the European Medicines Agency and that the FDA allowed Ocugen to convert its Phase II study into a combined Phase II/III trial following a small Phase I study.

The company expects top-line data from the Stargardt study in the second quarter of next year, Musunuri said.

Ocugen then plans to file a BLA and pursue market authorization in parallel within several months of the results. If development proceeds according to plan, he said approval and launch could occur in 2028.

Musunuri contrasted the program with oral therapies that may target individual disease pathways and require ongoing dosing. He said Ocugen’s subretinal gene therapy is designed as a one-time administration, while emphasizing that efficacy and safety data will determine its potential differentiation.

OCU410, Ocugen’s geographic atrophy candidate, uses the same RORA...