• Organizations: Ocugen
FDA clears Ocugen for phase 2/3 trial on Stargardt gene therapy
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FDA clears Ocugen for phase 2/3 trial on Stargardt gene therapy

IND approval enables continued clinical evaluation of OCU410ST, a modifier gene therapy with a favorable safety and tolerability profile; plans include a 2027 BLA submission.
FDA grants Rare Pediatric Disease designation to Stargardt gene therapy
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FDA grants Rare Pediatric Disease designation to Stargardt gene therapy

OCU410ST could receive a priority review voucher if approved; company plans to initiate a phase 2/3 trial in coming weeks—along with a BLA filing in 2027.
FDA OKs Ocugen's RP expanded access program in adult patients
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FDA OKs Ocugen's RP expanded access program in adult patients

EAP enables RP patients with at least minimal retinal preservation to benefit from OCU400 ahead of an expected 2026 BLA submission.
Ocugen doses first patient in phase 3 RP gene therapy trial
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Ocugen doses first patient in phase 3 RP gene therapy trial

Prior clinical data supports OCU400 in improving baseline vision among patients with RP associated RHO and N2ER3 mutations.
Ocugen's IND amendment gets FDA OK for phase 3 RP gene therapy trial
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Ocugen's IND amendment gets FDA OK for phase 3 RP gene therapy trial

Investigational candidate OCU400 is the first potential treatment for RP associated with rhodopsin mutations.
Ocugen's OCU400 receives FDA RMAT designation for RP
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Ocugen's OCU400 receives FDA RMAT designation for RP

New status marks progress in advancement of the first potential treatment of RP associated with rhodopsin mutations.
Ocugen reports positive preliminary data from RP, LCA gene therapy trial
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Ocugen reports positive preliminary data from RP, LCA gene therapy trial

Phase 1/2 trial is assessing the safety and efficacy of OCU400.
FDA approves pediatric patient enrollment in RP, LCA gene therapy trial
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FDA approves pediatric patient enrollment in RP, LCA gene therapy trial

Ocugen’s ongoing phase 1/2 trial is assessing OCU400 as a gene therapy candidate.