Drugs / BALURETGENE PARVEC
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT05203939 | Jan 2022 → Mar 2027 expected | Leber congenital amaurosis, retinitis pigmentosa | Ocugen | Active not recruiting | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT06574997 | — | retinitis pigmentosa | Ocugen | No longer available | No outcome recorded |
News releases announcing trial results or a regulatory action · 24
| Date | Issuer | Release |
|---|---|---|
| 2025-02-03 | Ocugen | Regulatory Ocugen Announces Positive Opinion of European Medicines Agency’s Committee for Advanced Therapies for Advanced Therapy Medicinal Product Classification for Modifier Gene Therapy Candidate OCU400 for Retinitis Pigmentosa 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 Commission has provided a positive opinion from the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) for OCU400 Advanced Therapy Medicinal Product (ATMP) classification. |
| 2025-01-13 | Ocugen | Results Ocugen, Inc. Announces Positive 2-Year Data Across Multiple Mutations from Phase 1/2 Clinical Trial of OCU400 —A Novel Modifier Gene Therapy for Retinitis Pigmentosa ocugen.com ↗
This treatment effect was statistically significant ( p=0.01 ) in all subjects, regardless of mutation at two years, validating the gene-agnostic mechanism of action for OCU400. |
| 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. |
| 2024-08-26 | Ocugen | Regulatory Ocugen, Inc. Announces Health Canada Approval to Initiate Phase 3 Clinical Trial for OCU400 – Modifier Gene Therapy for Broad Retinitis Pigmentosa Indication ocugen.com ↗ |
| 2024-08-05 | Ocugen | Regulatory Ocugen, Inc. Announces FDA Approval of Expanded Access Program for Patients with Retinitis Pigmentosa ocugen.com ↗ |
| 2024-04-10 | Ocugen | Regulatory Ocugen, Inc. Announces Positive Scientific Advice from the European Medicines Agency Related to the Approval Pathway for OCU400—Modifier Gene Therapy for Broad Retinitis Pigmentosa Indication ocugen.com ↗
today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) reviewed the study design, endpoints and planned statistical analysis of the pivotal OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) and provided acceptability of the U.S.-based trial for submission of a Marketing Authorization Application (MAA). |
| 2024-04-08 | Ocugen | Regulatory Ocugen, Inc. Announces U.S. FDA Clearance of IND Amendment to Initiate OCU400 Phase 3 Clinical Trial — First Gene Therapy to Enter Phase 3 with a Broad Retinitis Pigmentosa Indication ocugen.com ↗ |
| 2023-12-19 | Ocugen | Regulatory Ocugen Announces OCU400 Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for Treatment of Retinitis Pigmentosa Associated with RHO Mutations ocugen.com ↗
today announced that the FDA has granted RMAT designation to Ocugen’s investigational product OCU400 for the treatment of retinitis pigmentosa (RP) associated with RHO mutations. |
| 2023-09-13 | Ocugen | Results Ocugen Announces Positive Clinical Study Update from the Phase 1/2 Trial of OCU400, a Modifier Gene Therapy Product Candidate, for the Treatment of Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) ocugen.com ↗
today announced a clinical study update for Retinitis Pigmentosa (RP) participants treated in the Phase 1/2 trial to assess the safety and efficacy of OCU400 |
| 2023-04-14 | Ocugen | Results Ocugen Announces Positive Preliminary Safety and Efficacy Results from the Phase 1/2 Trial of OCU400, a Modifier Gene Therapy Product Candidate, for the Treatment of Retinitis Pigmentosa and Leber Congenital Amaurosis ocugen.com ↗
These preliminary results provide support that OCU400, Ocugen’s first-in-class therapeutic approach utilizing a proprietary modifier gene therapy platform, has the potential to be a gene-agnostic therapeutic for RP and LCA patients with inherited retinal degeneration. |
| 2023-03-27 | Ocugen | Regulatory Ocugen Announces FDA Approval for Enrollment of Pediatric Patients in Ongoing OCU400 Phase 1/2 Clinical Trial for the Treatment of Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) ocugen.com ↗ |
| 2022-12-15 | Ocugen | Regulatory Ocugen Announces OCU400 Receives Orphan Drug Designations for Retinitis Pigmentosa and Leber Congenital Amaurosis ocugen.com ↗
today announced that the FDA granted orphan drug designations to OCU400—human nuclear hormone receptor subfamily 2 group E member 3 (h NR2E3 )—for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). |
| 2022-12-07 | Ocugen | Results Ocugen Announces Update on OCU400 Phase 1/2 Clinical Trial Targeting Retinitis Pigmentosa and Leber Congenital Amaurosis ocugen.com ↗ |
| 2022-10-12 | Ocugen | Results Ocugen Announces Completion of Dosing in OCU400 Phase 1/2 Cohort 2 ocugen.com ↗
Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the Independent Data and Safety Monitoring Board (DSMB) for the OCU400 Phase 1/2 clinical trial completed a review of safety data for subjects enrolled in Cohort 2 and recommends proceeding to enroll subjects in Cohort 3. |
| 2022-04-25 | Ocugen | Results Ocugen, Inc. Announces Positive DSMB Recommendation for OCU400-101 Clinical Trial ocugen.com ↗
The DSMB recommended that the Company continue enrolling the remaining study subjects in this current cohort at the target dose level. |
| 2021-12-09 | Ocugen | Regulatory Ocugen, Inc. Announces U.S. FDA Acceptance of Investigational New Drug Application to Initiate a Phase 1/2 Clinical Trial for Gene Therapy Candidate OCU400 to Treat Inherited Retinal Degeneration ocugen.com ↗ |
| 2021-11-09 | Ocugen | Regulatory Ocugen Provides Business Update and Third Quarter 2021 Financial Results ocugen.com ↗
Two Investigational New Drug submissions within a span of two weeks is a phenomenal achievement resulting from the work of international teams aligned around serving people with serious diseases. |
| 2021-11-08 | Ocugen | Regulatory Ocugen, Inc. Announces Submission of Investigational New Drug Application with U.S. FDA to Initiate a Phase 1/2 Clinical Trial Evaluating Gene Therapy Candidate OCU400 (AAV-NR2E3) to Treat Inherited Retinal Degeneration ocugen.com ↗ |
| 2021-02-23 | Ocugen | Regulatory European Commission Grants Ocugen Orphan Medicinal Product Designation for Gene Therapy Product Candidate, OCU400, For the Treatment of Both Retinitis Pigmentosa and Leber Congenital Amaurosis ocugen.com ↗ |
| 2020-08-10 | Ocugen | Regulatory Ocugen Receives Fourth FDA Orphan Drug Designation for the Same Product, OCU400 (AAV-NR2E3) Gene Therapy, for the Treatment of Another Key Inherited Retinal Disease (IRD), PDE6B Gene Mutation-Associated Retinal Diseases ocugen.com ↗
Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced the U.S. Food and Drug Administration (FDA) granted the fourth Orphan Drug Designation (ODD) for OCU400 in the treatment of PDE6B gene mutation-associated retinal diseases. |
| 2020-07-27 | Ocugen | Regulatory Ocugen Granted FDA Orphan Drug Designation for OCU400 (AAV-hNR2E3) Gene Therapy for the Treatment of RHO Mutation-Associated Retinal Degenerative Disease ocugen.com ↗
Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced the U.S. Food and Drug Administration (FDA) granted the third Orphan Drug Designation (ODD) for OCU400 in the treatment of RHO mutation-associated retinal degeneration. |
| 2020-03-03 | Ocugen | Results Nature Gene Therapy Publishes Preclinical Data of Ocugen’s OCU400 (NR2E3-AAV) Genetic Modifier to treat Retinitis Pigmentosa (RP) ocugen.com ↗ |
| 2019-09-18 | Ocugen | Regulatory Ocugen Granted FDA Orphan Drug Designation for OCU400 (NR2E3) Gene Therapy for the Treatment of CEP290 Mutation Associated Retinal Disease ocugen.com ↗
today announced the U.S. Food and Drug Administration (FDA) granted the second orphan drug designation for OCU400, Ocugen’s novel gene therapy, for the treatment of CEP290 mutation associated retinal disease. |
| 2019-02-14 | Ocugen | Regulatory Ocugen Granted FDA Orphan Drug Designation for OCU400 Gene Therapy for the Treatment of NR2E3 Mutation-Associated Retinal Degenerative Disease ocugen.com ↗ |
All press releases naming this drug 32 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | BALURETGENE PARVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL6068483 ↗ |
| Known as | OCU400 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06574997 ↗ |
| Modality | Other / unclassified | “modifier gene therapy product” NCT06574997 ↗ |
| Route | Other | “single subretinal injection of OCU400 in one study eye” NCT05203939 ↗ |