Bloomsbury Genetic Therapies

Bloomsbury Genetic Therapies is a clinical-stage biotechnology company headquartered in London, UK, dedicated to developing transformative adeno-associated virus (AAV) gene therapies for rare metabolic and neurological disorders. Spun out from University College London (UCL) in 2022, the company leverages the expertise of world-leading gene therapy pioneers to address conditions with significant unmet medical needs. Its lead candidate, BGT-OTCD, is a liver-directed gene therapy for ornithine transcarbamylase deficiency currently in Phase 1/2 clinical development. The company's broader pipeline includes programs for Niemann-Pick disease type C (BGT-NPC), dopamine transporter deficiency syndrome (BGT-DTDS), and G6PC3 deficiency (BGT-GO). By utilizing a validated AAV platform and strategic academic collaborations, Bloomsbury aims to provide one-time, curative treatments for patients worldwide.