VISTA Trial
We are now pre-screening for a phase 2/3 clinical trial for X-Linked Retinitis Pigmentosa. Learn more about the clinical trial or how to qualify for no-cost genetic testing and/or prescreening.
In numbers:
- 1:40,000
- Young males affected
- <10 years old
- Early onset night blindness
- 45 years old
- Median age patients become legally blind
- 50%
- Genetic diagnosis rate for RP
If you suspect you have XLRP and are considering a clinical trial…
Related Programmes:
Disease/program | Phase | |||
---|---|---|---|---|
Preclinical | Phase 1 | Phase 2 | Phase 3 | |
X-Linked Retinitis Pigmentosa (XLRP) |
Phase 2/3 | |||
AGTC-501 |
Our lead asset, AGTC-501, is a gene therapy program currently in a Phase 2/3 clinical trial for the treatment of XLRP that was acquired as part of Syncona’s acquisition of AGTC in November 2022. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Unlike other approaches in the space, AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. |