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rAAV2tYF-CB-hRS1 · 1 trial · 1 indication
Number of participants with ocular adverse events, assessed by standard ophthalmic examination, including slit lamp biomicroscopy, tonometry, and indirect ophthalmoscopy.
| Arm | Type | Description |
|---|---|---|
| Groups 1A and 1B | EXPERIMENTAL | Subjects at least 18 y/o treated with a lower dose of rAAV2tYF-CB-hRS1 study drug. |
| Groups 2 and 2A | EXPERIMENTAL | Subjects at least 6 y/o treated with a middle dose of rAAV2tYF-CB-hRS1 study drug. |
| Group 3 | EXPERIMENTAL | Subjects at least 18 y/o treated with a higher dose of rAAV2tYF-CB-hRS1 study drug. |
| Group 4 | EXPERIMENTAL | Subjects at least 6 y/o treated with a maximum tolerated dose of rAAV2tYF-CB-hRS1 study drug determined for Groups 1A, 1B, 2 and 3. |
| Name | Type | Description |
|---|---|---|
| rAAV2tYF-CB-hRS1 | BIOLOGICAL | adeno-associated virus vector expressing retinoschisin |
Inclusion Criteria include: 1. Retinal disease consistent with a diagnosis of XLRS and documented mutations in the RS1 gene 2. Male individual at least 18 years of age (dose escalation phase) or at least 6 years of age (maximum tolerated dose phase), 3. Able to perform tests of visual and retinal f...
rAAV2tYF-CB-hRS1 is an investigational gene therapy being developed for X-linked retinoschisis, a genetic retinal disorder that causes vision loss, primarily in males. It is designed to deliver a functional copy of the RS1 gene to retinal cells. The therapy is in Phase 1 clinical development and has not been approved by the FDA.
rAAV2tYF-CB-hRS1 is being studied for the treatment of X-linked retinoschisis (XLRS), an inherited condition that leads to progressive retinal degeneration and vision impairment. It is intended to address the underlying genetic cause by providing a working copy of the RS1 gene. The therapy remains investigational.
rAAV2tYF-CB-hRS1 is being developed by Elevance Health, Inc., which trades under the ticker symbol ELV. The company is responsible for the clinical program evaluating this gene therapy in patients with X-linked retinoschisis.
rAAV2tYF-CB-hRS1 is in Phase 1 clinical development. A single Phase 1 trial has been completed, and no active trials are currently listed. The drug is investigational and has not received FDA approval for any indication.
rAAV2tYF-CB-hRS1 has been evaluated in one completed Phase 1 clinical trial, NCT02416622, titled "Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)." The trial enrolled 27 male participants aged 6 years and older in the United States. It was a controlled study without randomization or double-blinding.