Bloomsbury Genetic Therapies

We are a biotechnology company with a mission of bringing potentially curative treatments to patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies. Our approach leverages well-characterised AAV capsids, optimised vector constructs and novel routes of administration to create highly differentiated programs. We are tackling diseases with clear and translatable biology and high unmet medical needs. All of our four programs are first or best in class and have already demonstrated compelling preclinical efficacy. We are now working relentlessly to bring them to patients. We are on track to file a clinical trial application in the UK for the P1/2 for our lead program for ornithine transcarbamylase deficiency, BGT-OTCD, in December 2022.