Drugs / OCU410ST
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 2/3 | NCT05956626 | Aug 2023 → Sep 2026 overdue | Stargardt disease | Ocugen | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 12
| Date | Issuer | Release |
|---|---|---|
| 2026-01-12 | Ocugen | Results Ocugen Announces Publication of Phase 1 GARDian1 Trial Results for OCU410ST Modifier Gene Therapy ocugen.com ↗
The publication reports comprehensive 12-month safety, tolerability, and exploratory efficacy data from the first-in-human Phase 1 trial evaluating OCU410ST in patients with early to advanced Stargardt disease. |
| 2025-08-13 | Ocugen | Regulatory Ocugen, Inc. Announces Positive Scientific Advice from the European Medicines Agency Related to the Approval Pathway for OCU410ST—Modifier Gene Therapy for Stargardt Disease ocugen.com ↗ |
| 2025-06-16 | Ocugen | Regulatory Ocugen, Inc. Announces U.S. FDA Clearance of Investigational New Drug Amendment to Initiate Phase 2/3 Pivotal Confirmatory Clinical Trial of OCU410ST—Modifier Gene Therapy Candidate for Stargardt Disease ocugen.com ↗ |
| 2025-05-27 | Ocugen | Regulatory Ocugen Announces Rare Pediatric Disease Designation Granted for OCU410ST—Modifier Gene Therapy for the Treatment of Stargardt Disease ocugen.com ↗
today announced that the United States Food and Drug Administration (U.S. FDA) has granted Rare Pediatric Disease Designation (RPDD) for OCU410ST |
| 2025-03-03 | Ocugen | Regulatory Ocugen Announces Positive Opinion of EMA’s Committee for Advanced Therapies for ATMP Classification for Novel Modifier Gene Therapy Candidate OCU410 for Geographic Atrophy and OCU410ST for Stargardt Disease ocugen.com ↗
Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the European Commission has provided a positive opinion from the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) for OCU410 and OCU410ST Advanced Therapy Medicinal Product (ATMP) classification. |
| 2025-02-27 | Ocugen | Regulatory Ocugen, Inc. Announces FDA Alignment on Phase 2/3 Pivotal Confirmatory Clinical Trial for Modifier Gene Therapy Candidate OCU410ST for Stargardt Disease ocugen.com ↗ |
| 2024-11-20 | Ocugen | Regulatory Ocugen Announces European Medicines Agency Grants Orphan Medicinal Product Designation for Modifier Gene Therapy Candidate OCU410ST for Treatment of ABCA4-Associated Retinopathies including Stargardt Disease ocugen.com ↗
Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation for OCU410ST for the treatment of ABCA4 -associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3). |
| 2024-11-19 | Ocugen | Results Ocugen Announces Compelling Preliminary Data for OCU410—a Single Dose Novel Modifier Gene Therapy to Treat Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration ocugen.com ↗
Ocugen also announced promising data from the Phase 1/2 OCU410ST GARDian clinical trial for Stargardt disease and data on Leber congenital amaurosis (LCA) from the Phase 1/2 OCU400 clinical trial. |
| 2024-10-22 | Ocugen | Results Data and Safety Monitoring Board Approves Initiation of Phase 2 of OCU410ST GARDian Clinical Trial for Stargardt Disease ocugen.com ↗
No serious adverse events (SAEs) have been reported, and the DSMB determined the high dose to be the maximum tolerated dose (MTD). |
| 2024-06-21 | Ocugen | Results Ocugen Announces Data and Safety Monitoring Board Approves Enrollment in High Dose Cohort 3 in GARDian Study for Stargardt Disease ocugen.com ↗
“The DSMB has recommended moving forward to dose subsequent subjects with Stargardt disease at the targeted high dose,” said Dr. Peter Y. Chang, MD, FACS, DSMB Chair for the OCU410ST clinical trial. |
| 2024-04-01 | Ocugen | Results Ocugen Announces Positive Data and Safety Monitoring Board Review and Initiation of Enrollment in Medium Dose for OCU410ST—a Modifier Gene Therapy—in GARDian Study for Stargardt Disease ocugen.com ↗
No serious adverse events (SAEs) related to OCU410ST have been reported to date. |
| 2023-04-27 | Ocugen | Regulatory Ocugen Announces OCU410ST Receives Orphan Drug Designation for Treatment of ABCA4-Associated Retinopathies ocugen.com ↗
Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the FDA has granted Orphan Drug Designation for its OCU410ST |
All press releases naming this drug 18 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | OCU410ST | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05956626 ↗ |
| Known as | AAV5-hRORA | “Name of Investigational Product: OCU410ST Name of Active Ingredient: Adeno-associated viral vector 5 human RORA (AAV5-hRORA)” NCT05956626 ↗ |
| Action | Restore | “The role of hRORA in regulating these gene pathways strongly suggests OCU410ST could restore homeostasis in the eye and thereby serve as a therapeutic candidate for Stargardt disease.” NCT05956626 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “OCU410ST is an Adeno-Associated Virus serotype 5 containing human RORA for the treatment of Stargardt disease.” NCT05956626 ↗ |
| Route | Other | “Subjects will receive a single subretinal injection of 200 µL OCU410ST in concentration of 1.5×10E11 vg/mL.” NCT05956626 ↗ |