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Bloomsbury Genetic Therapies Announces Initiation of Recruitment in Phase 1/2 HORACE Clinical Trial of Investigational Gene Therapy BGT-OTCD for the Treatment of Ornithine Transcarbamylase Deficiency (OTCD)

16 November 2023

Gene therapy

– HORACE trial initiated to evaluate the safety, efficacy and tolerability of BGT-OTCD in paediatric patients diagnosed with OTCD –

London, UK, 16 November 2023 – Bloomsbury Genetic Therapies Limited, 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, announced today that the Company’s collaborator, University College London (UCL), has initiated recruitment in the Phase 1/2 clinical trial HORACE (Halting Ornithine transcarbamylase deficiency with Recombinant AAV in ChildrEn, NCT05092685) evaluating BGT-OTCD, Bloomsbury’s investigational AAV-LK03 gene therapy, in paediatric patients diagnosed with OTCD.

HORACE is an open-label, safety, efficacy and dose-finding trial and will enrol up to 12 patients aged between 0 and 16 years old diagnosed with OTCD. UCL is the sponsor of the trial with financial support provided by the Company. HORACE will be conducted at a single treatment site, Great Ormond Street Hospital (GOSH) in London, UK, with three further referring UK sites: Evelina London Children’s Hospital, Royal Manchester Children’s Hospital and Birmingham Children’s Hospital.

“OTCD is a devastating disease, particularly in the paediatric population. Current medical management of children with OTCD and dietary protein restriction do not prevent recurrent metabolic crises and don’t fully address the sub-clinical chronic neuronal damage caused by elevated blood ammonia levels, and its impact on children’s neurological development. I am therefore very pleased that recruitment has been initiated in the HORACE trial and look forward to evaluating BGT-OTCD as a potentially curative solution for children with OTCD”, said Dr Anupam Chakrapani, Consultant in Metabolic Medicine, GOSH and the trial’s Principal Investigator.

“The initiation of our first clinical trial just a year after Bloomsbury’s launch is a landmark moment for us. BGT-OTCD is a highly differentiated gene therapy candidate for OTCD, relying on the best-in-class capsid AAV-LK03, whose efficacy, durability and safety has already been illustrated in the clinic in haemophilia A clinical trials, and we believe it has curative potential for both paediatric and adult patients suffering from OTCD,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “We look forward to UCL sharing initial clinical data with the OTCD community in due course.”

For more information on the Phase 1/2 HORACE trial of BGT-OTCD for the treatment of OTCD, please visit: https://clinicaltrials.gov/study/NCT05092685 and for more information about participation to the trial, please contact cctu.horace@ucl.ac.uk.

Further information: 

Bloomsbury Genetic Therapies