Bloomsbury Genetic Therapies

11:00 AM - 11:15 AM (EST), Tuesday, February 7, 2023 ・ Music Box
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.
Company Type:
Privately Funded Company
Company HQ State:
Not Provided
Company HQ Country:
United Kingdom
Year Founded:
Main Therapeutic Focus:
Orphan/Rare Diseases
Lead Product in Development:
Development Phase of Primary Product:
Phase II
Number Of Unlicensed Products (For Which You Are Seeking Partners):
Chief Executive Officer
Bloomsbury Genetic Therapies