Pipeline

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.

Our dryAMD program features an intravitreally (IVT) delivered novel AAV based gene therapy for dry Age-related Macular Degeneration (dryAMD). Dry AMD is a leading cause of irreversible vision loss in people over 60. IVT delivery is less invasive, requires less clinician training and can be delivered in clinic rather than via surgery, hence providing greater access to more patients.

Our Cadherin Related Family Member 1 (CDHR1) program targets Cone-rod Dystrophy (CRD) is a rare, monogenic recessive disorder caused by a null mutation in the CDHR1 gene. The program was licensed from the laboratory of Professor Robert MacLaren, Professor of Ophthalmology at the University of Oxford.