Pipeline

Our lead asset, laru-zova, is an investigational gene therapy program for the treatment of XLRP. It includes a proprietary AAV capsid designed to efficiently transduce both rods and cones. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Laru-zova uses a modified gene cassette that expresses the full length RPGR protein, thereby potentially addressing the full complement of photoreceptor damage caused by XLRP.

Our dry AMD program features an intravitreally (IVT) delivered novel AAV based gene therapy. 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 patients.

Our Cadherin Related Family Member 1 (CDHR1) program targets an inherited Cone-rod Dystropy (CRD). CRD is a group of inherited eye disorders that affect the light sensitive cells of the retina called the cones and rods. The program was licensed from the laboratory of Professor Robert MacLaren, Professor of Ophthalmology at the University of Oxford.