Bloomsbury Genetic Therapies Announces CTA Filing for a Phase I/II Study for its Lead Gene Therapy Program
– Clinical trial in patients with significant unmet medical need to start in 2023 –
London, UK, 7 December 2022 – Bloomsbury Genetic Therapies Limited (“Bloomsbury”), a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, today announced that University College London (UCL) has submitted an application for a Clinical Trial Authorisation (CTA) to the Gene Therapy Advisory Committee (GTAC) and UK Medicines and Healthcare products Regulatory Agency (MHRA) for the development of BGT-OTCD as a potential new therapeutic for the treatment of Ornithine Transcarbamylase Deficiency (OTCD). BGT-OTCD has demonstrated curative potential in preclinical efficacy studies and has completed comprehensive preclinical safety and biodistribution assessments.
The Phase I/II trial of BGT-OTCD, Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn (HORACE: NCT05092685) is a safety and dose finding study in paediatric patients with OTCD and is expected to begin in the UK in 2023. UCL are the sponsor of the trial with financial support to be provided by Bloomsbury.
Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury said, “We are focused on delivering the promise of gene therapy, so this first step on the journey to bring our potentially curative treatment to all OTCD patients who need it is a significant milestone for us.”
ENDS
Enquiries
JW Communications
Julia Wilson
Tel: +44 (0)7818 430877
About Bloomsbury Genetic Therapies
Bloomsbury Genetic Therapies (“Bloomsbury”) is a clinical-stage biotechnology company, developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies. The Company was spun out of UCL and launched in October 2022 with £5M in Seed funding from UCL Technology Fund and is underpinned by world-leading gene therapy and rare disease expertise from the Company’s academic founders, Professor Paul Gissen, Professor Manju Kurian, Professor Ahad Rahim and Professor Simon Waddington. Bloomsbury is building a pipeline of highly differentiated first- or best-in-class programs. For more information, please visit www.bloomsburygtx.com
About BGT-OTCD
BGT-OTCD is a liver-targeted AAV-LK03 gene therapy designed to provide a potentially curative solution to OTC deficiency patients following a one-time intravenous injection.
About Ornithine transcarbamylase deficiency
Ornithine transcarbamylase deficiency (OTCD) is a genetic disease that causes too much ammonia to accumulate in the blood (hyperammonemia). Ammonia is toxic when levels are too high and especially affects the nervous system. Signs and symptoms may include lack of energy and appetite, poorly-controlled breathing rate and body temperature, unusual body movements, seizures, which can all progress to coma or death. While later onset disease can occur in adults with a milder form of the disorder, symptoms present within a few days of birth of males with severe OTCD. Current standard of care involves protein-restricted diets and ammonia-scavenger medications; however, these approaches can have a significant impact on patients’ quality of life and patients still face lifelong risk of decompensation and neurological damage resulting in intellectual disability, developmental delays, and movement disorder. Liver transplant is the only curative option, but is often unavailable and comes with significant morbidity/mortality risk and lifelong immunosuppression and arginine supplementation. Over 10,000 patients suffering from OTCD have been identified worldwide.
About University College London
University College London (UCL) is a diverse global community of world-class academics, students, industry links, external partners, and alumni. Its powerful collective of individuals and institutions work together to explore new possibilities. Since 1826, UCL has championed independent thought by attracting and nurturing the world’s best minds. Its community of more than 43,800 students from 150 countries and over 14,300 staff pursues academic excellence, breaks boundaries and makes a positive impact on real world problems. UCL is consistently ranked among the top 10 universities in the world and is one of only a handful of institutions rated as having the strongest academic reputation and the broadest research impact. For more information, please visit www.ucl.ac.uk
