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rAAV2-CB-hRPE65 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| 1 | EXPERIMENTAL | Subjects at least 6 y/o treated with a lower dose of the vector by subretinal injection |
| 2 | EXPERIMENTAL | Subjects at least 6 y/o treated with a higher dose of the vector by subretinal injection |
| Name | Type | Description |
|---|---|---|
| rAAV2-CB-hRPE65 | BIOLOGICAL | Recombinant adeno-associated virus vector expressing RPE65 |
Inclusion Criteria: * Retinal disease consistent with a diagnosis of Leber congenital amaurosis and documented mutations in the RPE65 gene (including null mutations and mutations that code for abnormal RPE65 protein); * At least 6 years of age; * Good general health without significant physical exa...
rAAV2-CB-hRPE65 is an investigational gene therapy developed for Leber Congenital Amaurosis, a rare inherited retinal disorder. It delivers a functional copy of the human RPE65 gene using a recombinant adeno-associated virus serotype 2 vector. It is being studied as a one-time treatment approach for patients with RPE65-related retinal disease.
rAAV2-CB-hRPE65 is being studied for the treatment of Leber Congenital Amaurosis, a rare genetic condition that causes severe vision loss in children. The therapy targets the RPE65 form of the disease, in which mutations impair the visual cycle in the retina. It is administered as a gene therapy vector.
rAAV2-CB-hRPE65 is being developed by Elevance Health, Inc., which trades under the ticker ELV. The company sponsored the Phase 1/2 clinical study of the gene therapy in patients with Leber Congenital Amaurosis in the United States.
rAAV2-CB-hRPE65 is in Phase 1 clinical development. Its single registered trial, NCT00749957, was a Phase 1/2 safety and efficacy study that has been completed. The therapy is investigational and has not been approved by the FDA for any indication.
rAAV2-CB-hRPE65 has been evaluated in one registered clinical trial, NCT00749957, titled a Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis. The trial enrolled 12 participants in the United States, included patients aged 6 years and older, and has been completed.
rAAV2-CB-hRPE65 uses a recombinant adeno-associated virus serotype 2 vector to deliver the human RPE65 gene to retinal cells. The RPE65 gene encodes a protein essential to the visual cycle. Restoring functional RPE65 protein in the retina is intended to address the underlying genetic defect in RPE65-related Leber Congenital Amaurosis.