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OCU400

Gene therapy · Gene replacement · developed by Ocugen

Phase 1/2 Most advanced trial

OCGN Developer

2 Registered trials

What is OCU400?

OCU400 is a gene therapy administered by subretinal injection for inherited retinal diseases including retinitis pigmentosa and Leber congenital amaurosis.

Summary written with AI from the sources listed at the end of this page.

At a glance

Drug type: Gene therapy
Mechanism: Gene replacement
Therapy area: Ophthalmology
Developer: Ocugen (OCGN)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 1/2
UNII: 72M7L7U43V

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT05203939	Phase 1/2	Retinitis Pigmentosa	Active not recruiting	OCGN
NCT06574997	N/A	Retinitis Pigmentosa	No longer available	OCGN

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Leber Congenital AmaurosisRetinitis Pigmentosa

More from Ocugen

OCU200 Phase 1 OCU410 Phase 3 OCU410ST Phase 2/3

Sources

Updated Oct 3, 2026. Something wrong or missing? Report it on the OCGN page.

Follow OCGN, the company behind OCU400

See OCGN's upcoming FDA decisions, trial readouts, cash runway and dilution risk, updated every day from SEC, FDA and ClinicalTrials.gov filings.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z