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Also known as AAV8.hLCA5
OPGx-LCA5 · 1 trial · 1 indication
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Qualitative Assessment of SD-OCT image
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| Arm | Type | Description |
|---|---|---|
| Dose Group 1 | EXPERIMENTAL | A single, unilateral, subretinal injection of low dose (1E10 vg/eye) OPGx-001 is injected into two LCA5 adults (18 yo or above) in a sentinel fashion. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, a subsequent LCA5 adolescent (13-17) may be treated in a sentinel fashion, with a unilateral, subretinal injection of OPGx-001. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, two remaining adolescents (13-17) are eligible to be treated with a single, unilateral, subretinal injection of a low dose of OPGx-001. Total cohort size is 5 (2 adults and 3 adolescents). |
| Dose Group 2 | EXPERIMENTAL | A single, unilateral, subretinal injection of an intermediate dose (3E10 vg/eye) OPGx-001 is injected into two LCA5 adults (18 yo or above) in a sentinel fashion. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, a subsequent LCA5 adolescent (13-17) may be treated in a sentinel fashion, with a unilateral, subretinal injection of OPGx-001. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, two remaining adolescents (13-17) are eligible to be treated with a single, unilateral, subretinal injection of an intermediate dose of OPGx-001. Total cohort size is 5 (2 adults and 3 adolescents). |
| Dose Group 3 | EXPERIMENTAL | A single, unilateral, subretinal injection of high dose (1E11 vg/eye) OPGx-001 is injected into two LCA5 adults (18 yo or above) in a sentinel fashion. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, a subsequent LCA5 adolescent (13-17) may be treated in a sentinel fashion, with a unilateral, subretinal injection of OPGx-001. A 30-day safety evaluation and data committee review and approval of continued dosing occurs. If additional dosing is recommended, two remaining adolescents (13-17) are eligible to be treated with a single, unilateral, subretinal injection of a high dose of OPGx-001. Total cohort size is 5 (2 adults and 3 adolescents). |
| Part B Observational | NO_INTERVENTION | Part B will occur concurrently with Dose Groups 1-3. Sixteen patients will be assessed for 6 months to assess the natural course of LCA5-IRD, then will be treated with OPGx-001 which will be described in a future protocol amendment. |
| Name | Type | Description |
|---|---|---|
| AAV8.hLCA5 | BIOLOGICAL | Adeno-associated virus vector expressing human LCA5 gene |
Inclusion Criteria: 1. Are willing and able to provide written informed consent (ICF) and, where appropriate, willing to sign an assent prior to any study procedures. 2. Are willing to adhere to the clinical protocol and able to perform testing procedures. 3. In part A participants must be 13 years...
OPGx-LCA5 is an investigational gene therapy being developed for LCA5-associated inherited retinal degeneration, a rare genetic form of vision loss caused by mutations in the LCA5 gene. It is designed to treat patients with this specific inherited retinal disease. It is not approved for any use and remains in clinical development.
OPGx-LCA5 is being developed by Opus Genetics, Inc., a clinical-stage biopharmaceutical company focused on gene therapies for inherited retinal diseases. Opus Genetics trades on the Nasdaq under the ticker symbol IRD.
OPGx-LCA5 is in Phase 1 clinical development. The program has one active, recruiting trial and no completed trials. It is investigational and has not been approved by the FDA for any indication.
OPGx-LCA5 is being evaluated in a single Phase 1 trial, NCT05616793, titled a study of safety and tolerability of subretinal OPGx-001 for LCA5-associated inherited retinal degeneration. The trial is recruiting in the United States, has an estimated enrollment of 22 participants, and includes a non-interventional arm with untreated patients.
Yes, AAV8.hLCA5 is another name for OPGx-LCA5. Both refer to the same investigational gene therapy developed by Opus Genetics for LCA5-associated inherited retinal degeneration.
OPGx-LCA5 has received three FDA designations: Rare Pediatric Disease designation, Regenerative Medicine Advanced Therapy (RMAT) designation, and Orphan Drug designation. These designations are intended to support and expedite the development of therapies for serious rare conditions.