Bloomsbury Genetic Therapies Ltd.

– HORACE trial initiated to evaluate the safety, efficacy and tolerability of BGT-OTCD in paediatric patients diagnosed with OTCD – LONDON, UK I November 06, 2023 I 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 enrolled the first patient 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 to have initiated 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 About 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 University College London and launched in October 2022 with funding from UCL Technology Fund. Bloomsbury is building a pipeline of highly differentiated first- or best-in-class programs. For more information, please visit www.bloomsburygtx.com About Ornithine transcarbamylase deficiency Ornithine transcarbamylase deficiency (OTCD) is a rare, X-linked genetic disorder that is characterised by complete or partial lack of the OTC enzyme. OTC enzyme is a key component of the urea cycle and patients with OTCD accumulate nitrogen waste in the form of excess ammonia (hyperammonaemia) in the blood, causing hyperammonaemic decompensations with symptoms including vomiting, impaired voluntary movement and progressive lethargy. If left untreated, these may progress to coma and life-threatening complications. 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. Patients are rapidly diagnosed (urine and blood biochemical analyses, gene sequencing) when they present at hospital/are admitted to intensive care with acute hepatic decompensation and hyperammonaemia. 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 a 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 BGT-OTCD BGT-OTCD is an investigational AAV-LK03 gene therapy designed to provide a potentially curative solution to OTCD patients following a one-time intravenous injection. AAV-LK03 was selected for its high tropism for liver cells and its success in other liver disorders such as haemophilia A. BGT-OTCD has been granted Orphan Drug Designation (ODD) for the treatment of OTCD by the European Commission (EC) and the US Food and Drug Administration (FDA) as well as Rare Pediatric Disease Designation (RPDD) from the FDA.  BGT-OTCD is currently being evaluated in HORACE (Halting Ornithine transcarbamylase deficiency with Recombinant AAV in ChildrEn; NCT 05092685), a Phase 1/2 clinical trial initiated in November 2023. SOURCE: Bloomsbury Genetic Therapies

– First in-human phase 1/2 trial to evaluate the safety, efficacy and tolerability of BGT-OTCD in paediatric patients with confirmed diagnosis of OTCD – – University College London expects to initiate the clinical trial in Q3 2023 – LONDON, UK I May 15, 2023 I 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 that the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) and the Gene Therapy Advisory Committee (GTAC) have approved the clinical trial application (CTA) submitted by Bloomsbury’s collaborators at University College London (UCL) for the initiation of a phase 1/2 clinical trial of BGT-OTCD, the Company’s liver-targeted AAV-LK03 gene therapy, in paediatric patients diagnosed with OTCD. The phase 1/2 trial of BGT-OTCD, Halting Ornithine transcarbamylase deficiency with Recombinant AAV in ChildrEn (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 and is expected to begin in the UK in Q3 2023. UCL is the sponsor of the trial with financial support to be provided by Bloomsbury. “This is a significant milestone for Bloomsbury as BGT-OTCD becomes the company’s first investigational gene therapy to be taken into a clinical trial,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury . “We are thrilled to collaborate with the teams at UCL and Great Ormond Street Hospital (GOSH) in London on this trial supporting the ongoing development of the BGT-OTCD program and our goal of bringing a one-time, potentially curative treatment to both children and adult patients suffering from this devasting disease.” “Ornithine transcarbamylase deficiency is a rare genetic disorder that that is characterised by complete or partial lack of the OTC enzyme which causes too much ammonia to accumulate in the body. People with this disease suffer from symptoms including vomiting, impaired voluntary movement and progressive lethargy, which can all progress to brain damage, coma or death if left untreated,” said Dr Anupam Chakrapani, Consultant in Metabolic Medicine, GOSH and the trial’s Principal Investigator . “The capsid used in this investigational AAV gene therapy, AAV-LK03, was selected for its enhanced ability to transduce human hepatocytes, and in particular periportal hepatocytes where the urea cycle preferentially takes place. We hope the trial will show that BGT-OTCD could provide sustained curative effect following a single administration even in children with a growing liver.” About 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 University College London 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 OTCD patients following a one-time intravenous injection.  AAV-LK03 was selected for its high tropism for liver cells and its success in other liver disorders such as haemophilia A. BGT-OTCD has been granted orphan drug designation for the treatment of OTCD by the European Medicines Agency (EMA). About Ornithine transcarbamylase deficiency Ornithine transcarbamylase deficiency (OTCD) is a rare, X-linked genetic disorder that is characterised by complete or partial lack of the OTC enzyme. OTC is a key component of the urea cycle and patients with OTCD accumulate nitrogen waste in the form of excess ammonia (hyperammonaemia) in the blood, causing hyperammonaemic decompensations with symptoms including vomiting, impaired voluntary movement and progressive lethargy. If left untreated, these may progress to coma and life-threatening complications. 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. Patients are rapidly diagnosed (urine and blood biochemical analyses, gene sequencing) when they present at hospital/are admitted to intensive care with acute hepatic decompensation and hyperammonaemia. 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. SOURCE: Bloomsbury Genetic Therapies