Now pre-screening for a phase 2/3 clinical trial of an investigational gene therapy in people with X-Linked Retinitis Pigmentosa. Learn more about the clinical trial or how to qualify for pre-screening.
Several clinical trials have been conducted or are planned for Beacon Therapeutics’ lead development candidate, AGTC-501, a gene therapy program for the treatment of XLRP.
Horizon in an ongoing Phase 1/2 clinical trial which has completed enrollment of 29 male participants and all participants are in long-term follow-up. Participants received a single dose of AGTC-501 in one eye to obtain initial data about safety and potential efficacy of AGTC-501 gene therapy. The 12-month post treatment data results demonstrate AGTC-501 is generally safe and well tolerated.
The Horizon protocol was expanded to add a randomized, masked, multi-center, Phase 2 trial (Skyline) comparing two doses of AGTC-501. Skyline is an ongoing clinical trial which has completed enrollment of 14 male participants who received gene therapy in one eye to further assess safety and potential efficacy of AGTC-501 gene therapy. AGTC- 501 has demonstrated positive and clinically important and significant improvement in retinal sensitivity and a clear differentiation between dose groups were demonstrated at month 12. An efficacious dose is evident. AGTC-501 is generally safe and well tolerated. The benefit: risk profile is favorable and supports continued clinical development for the treatment of patients with XLRP.
Vista is a planned randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety, and tolerability of 2 doses of AGTC-501 compared to an untreated control group. With the Vista study, AGTC will use the data generated from the Vista trial, in combination with data from the Phase 1/2 Horizon and Phase 2 Skyline studies, including all available long-term data, to support a BLA in the US and a MAA in Europe for the treatment of patients with XLRP.
FOR PATIENTS OR CAREGIVERS
If you are an individual with XLRP or know someone with XLRP who may be interested in participating in the VISTA Trial.
FOR HEALTHCARE PROVIDERS
If you are a HealthCare Provider who is interested in obtaining more information about the VISTA clinical trial, including how your patients might participate.
Achromatopsia (ACHM) is an inherited condition associated with extremely poor visual acuity, caused by mutations in one of several genes, with the two most common being mutations in either the CNGB3 or CNGA3 genes. AGTC completed enrollment for the Phase 1/2 gene therapy clinical trials for individuals with a mutation in either CNGA3 or CNGB3 genes. Upon review of the portfolio, Beacon Therapeutics has decided not to continue the development of either achromatopsia product candidates at this time. We will however continue to follow these patients for the full course of the trial, which includes a full four years of follow-up.
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