Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
rAAV2tYF-GRK1-RPGR · 2 trials · 1 indication
Number and proportion of participants experiencing Grade 3 or higher local (ocular) or systemic treatment-emergent adverse events that occur during the 36 months after study agent administration; number and proportion of participants experiencing treatment-emergent AEs, including treatment-emergent serious AEs;
| Arm | Type | Description |
|---|---|---|
| Low Dose Group | EXPERIMENTAL | Male subjects at least 8 y/o treated with a lower dose (Dose 2 in RPGR-001 Horizon Phase 1/2 study) of rAAV2tYF-GRK1-RPGR study drug. |
| High Dose Group | EXPERIMENTAL | Male subjects at least 8 y/o treated with a higher dose (Dose 5 in RPGR-001 Horizon Phase 1/2 study) of rAAV2tYF-GRK1-RPGR study drug. |
| Group 1: Phase 1/2 Dose Escalation | EXPERIMENTAL | Male subjects at least 18 y/o treated with Dose 1 of rAAV2tYF-GRK1-RPGR study drug. |
| Group 2: Phase 1/2 Dose Escalation | EXPERIMENTAL | Male subjects at least 18 y/o treated with Dose 2 of rAAV2tYF-GRK1-RPGR study drug. |
| Group 3 Phase 1/2 Dose Escalation | EXPERIMENTAL | Group 3 male subjects at least 18 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug. |
| Group 4 Phase 1/2 Dose Escalation | EXPERIMENTAL | Group 4 male subjects at least 6 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug. |
| Group 5 Phase 1/2 Dose Escalation | EXPERIMENTAL | Male subjects at least 18 y/o treated with Dose 5 of rAAV2tYF-GRK1-RPGR study drug. |
| Group 6 Phase 1/2 Dose Escalation | EXPERIMENTAL | Male subjects at least 18 y/o treated with Dose 6 of rAAV2tYF-GRK1-RPGR study drug. |
| Name | Type | Description |
|---|---|---|
| rAAV2tYF-GRK1-RPGR | BIOLOGICAL | Adeno-associated virus vector expressing a human RPGR gene |
Inclusion Criteria: * Provide written informed consent or assent (per local regulation), prior to the conduct of any study-related procedure. Subjects who provide assent must have a parent, guardian, or legal representative provide written informed consent. * Be between 8 and 50 years of age (inclu...
rAAV2tYF-GRK1-RPGR is an investigational gene therapy being developed for X-Linked Retinitis Pigmentosa, a rare inherited retinal disease that causes progressive vision loss. It is designed to deliver a functional RPGR gene to retinal cells. The drug is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.
rAAV2tYF-GRK1-RPGR targets the RPGR gene, which is mutated in X-Linked Retinitis Pigmentosa. The therapy uses an adeno-associated virus (AAV) vector to deliver a correct copy of the RPGR gene to retinal cells, aiming to restore their function. This approach is designed to address the underlying genetic cause of the disease.
rAAV2tYF-GRK1-RPGR is being developed by Elevance Health, Inc., a company traded on the stock exchange under the ticker symbol ELV. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational gene therapy for X-Linked Retinitis Pigmentosa.
rAAV2tYF-GRK1-RPGR is currently in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The therapy is being studied in clinical trials to assess its safety and effectiveness in patients with X-Linked Retinitis Pigmentosa.
rAAV2tYF-GRK1-RPGR is being evaluated in two active clinical trials. NCT03316560 is a Phase 1 study with 29 participants, and NCT06333249, also known as the SKYLINE trial, is a Phase 2 study with 14 participants. Both trials are enrolling male patients with X-Linked Retinitis Pigmentosa caused by RPGR mutations in the United States.
Yes, rAAV2tYF-GRK1-RPGR is also known as AGTC-501. The Phase 2 clinical trial NCT06333249, titled SKYLINE, uses the name AGTC-501 to refer to the same investigational gene therapy. Both names describe the same drug being developed for X-Linked Retinitis Pigmentosa.