Bloomsbury Genetic Therapies Announces CTA Filing for a Phase I/II Study for its Lead Gene Therapy Program

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.”