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.

BTX-001 is an adeno-associated virus (AAV)-based gene therapy candidate being evaluated for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). The therapy is designed to target the clinically-validated complement pathway through delivery of a C5 inhibitor administered as a single intravitreal injection, combining the potential for sustained treatment effect with the convenience of in-office delivery. 

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.