Product Description
for the treatment of patients with Lebers Congenital Amaurosis due to mutations in the RPE65 gene (LCA2) (Sourced from: https://investors.meiragtx.com/news-releases/news-release-details/meiragtx-receives-rare-pediatric-disease-designation-a001)
Mechanisms of Action: Gene Therapy, RPE65
Novel Mechanism: No
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: MeiraGTx
Company Location: Eastern America
Company Founding Year: None
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Recent & Upcoming Milestones
Highest Development Phases
Phase 2: Leber Congenital Amaurosis
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT02946879 |
LCA2 | N/A |
Completed |
Leber Congenital Amaurosis|Retinal Dystrophies|Blindness |
2023-07-01 |
2024-04-12 |
Patient Enrollment|Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments |
|
2015-003418-25 |
Gene Therapy Trial for LCA2 OPTIRPE65 (AAV2/5-OPTIRPE65) | P1 |
Completed |
Leber Congenital Amaurosis |
2018-12-07 |
2025-07-02 |
Treatments |
|
2016-000898-20 |
Long term follow-up gene therapy study for LCA2 OPTIRPE65 | P2 |
Unknown status |
Leber Congenital Amaurosis |
2023-10-04 |
2022-03-13 |
Treatments |
Recent News Events
Date |
Type |
Title |
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