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Ocugen, Inc.

Ophthalmology Phase 3

Nasdaq: OCGN

Founded: 2013

Corporate Overview

Ocugen, Inc.

Ocugen, Inc. is a pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach.

Unlike traditional gene therapies and gene editing, Ocugen's modifier gene therapies address the entire disease—complex diseases that are potentially caused by imbalances in multiple gene networks. Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe.
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Ocugen, Inc. NASDAQ: OCGN

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Drug Pipeline

AAV modifier therapies, biologics, and inhaled vaccines, targeting Retinitis Pigmentosa, Stargardt, and diabetes-related eye disease.

Pre-IND IND Phase 1 Phase 2 Phase 2/3 Phase 3
OwnershipKwangdong PharmaKorea rights
TargetNR2E3 (NHR)NR2E3 is a nuclear hormone receptor target linked to photoreceptor gene regulation in inherited retinal disease.
Enrollment150For Phase 3 inherited retinal studies, 150 participants is a robust sample designed for powered functional endpoints.
Next Signal

Primary Endpoint

LDNA responder rate at Week 52.

Secondary Endpoints

BCVA, LLVA, and patient-reported visual function.
OwnershipWholly owned
TargetRORA (NHR)RORA is a transcriptional regulator involved in retinal metabolism and inflammation pathways relevant to Stargardt progression.
Enrollment51This sample size aligns with rare-disease Stargardt programs where active global patient pools are limited.
Next Signal

Primary Endpoint

Change in atrophic lesion size at 12 months.

Secondary Endpoints

BCVA and LLVA in treated vs untreated eyes.
OwnershipWholly owned
TargetRORA (NHR)In GA secondary to dry AMD, RORA modulation is intended to improve retinal resilience and reduce degenerative signaling.
Enrollment60 (Phase 1/2)This size is typical for dose-finding plus early efficacy in ophthalmology programs before larger confirmatory expansion.
Next Signal

Primary Endpoint

Change in GA lesion size at 12 months.

Secondary Endpoints

Retinal structural and functional preservation signals.
OwnershipWholly owned
TargetalphaVbeta3 IntegrinalphaVbeta3 integrin is associated with angiogenesis and vascular leakage, central mechanisms in retinal edema and neovascular disease.
Enrollment~24 (4 cohorts)For first-in-human biologic studies, ~24 patients across dose cohorts is a common structure for safety and MTD evaluation.
Next Signal

Primary Endpoint

Safety and maximum tolerated dose (MTD).

Secondary Endpoints

Exploratory efficacy via BCVA and retinal thickness change.
OwnershipNIAID Partnership
TargetSARS-CoV-2The target is respiratory mucosal immune protection against SARS-CoV-2, aiming for local airway response in addition to systemic immunity.
EnrollmentTBDEnrollment is partner-defined at this stage; Phase 1 vaccine cohorts are often adjusted by dose arm and immunogenicity design.
Next Signal

Primary Endpoint

Safety and immunogenicity after inhaled dosing.

Commercial Note

NIAID partnership is strategically meaningful; Ocugen does not solely control Phase 1 timing.
OwnershipWholly owned
TargetFlu / Flu+COVIDCombination respiratory targets are positioned for seasonal protection with one inhaled platform covering influenza and COVID antigens.
EnrollmentN/A (pre-IND)Pre-IND programs generally finalize enrollment after regulatory feedback and protocol agreement for first-in-human studies.
Next Signal

Primary Endpoint

Expected safety and combined immunogenicity in first-in-human trials.

Secondary Endpoints

Mucosal antibody profiles and breadth across relevant strains.