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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 being studied for the treatment of Leber Congenital Amaurosis, a rare inherited retinal disease. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
rAAV2-CB-hRPE65 is designed to deliver a functional copy of the RPE65 gene to retinal cells. The RPE65 gene provides instructions for an enzyme essential for normal vision, and mutations in this gene cause Leber Congenital Amaurosis.
rAAV2-CB-hRPE65 is being developed by Elevance Health, Inc., a company traded on the New York Stock Exchange under the ticker symbol ELV. The drug is in Phase 1 clinical trials for Leber Congenital Amaurosis.
rAAV2-CB-hRPE65 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. One Phase 1/2 clinical trial has been completed.
rAAV2-CB-hRPE65 has been studied in one completed clinical trial, identified as NCT00749957. This Phase 1/2 study evaluated the safety and efficacy of the AAV-RPE65 vector in 12 participants with Leber Congenital Amaurosis in the United States.
Yes, rAAV2-CB-hRPE65 is also known as AAV-RPE65. The clinical trial NCT00749957, titled 'Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis,' used this agent.