Clinical Stage Biotech Bloomsbury Genetic Therapies Shuttering its Doors
The company’s CEO announced via LinkedIn that the company is shutting down its operations, citing a “difficult funding environment.”
Bloomsbury Genetic Therapies, a biotech company developing several gene therapy candidates for ultra-orphan rare diseases, is in the process of shutting down its operations.1
The announcement came via a LinkedIn post made by the company’s cofounder and chief executive officer, Adrien Lemoine, MSc, who noted alongside the news that he will be stepping down from his role as CEO. Lemoine cited a “difficult funding environment which doesn’t do justice to the quality of the science nor the acuteness of the unmet medical needs” as the reason for the company’s closure.
In the post, Lemoine stated that he is proud of what the company was able to accomplish with the £5M in capital that it had raised, highlighting “compelling” clinical data from the phase 1/2 HORACE clinical trial (NCT05092685) evaluating BGT-OTCD, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat ornithine transcarbamylase deficiency (OTCD).1,2 Lemoine stated that the aforementioned data will be presented in a poster at the upcoming American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, which will be held May 13 to 17, 2025, in New Orleans, LA. Among other achievements of the company, he pointed out that Bloomsbury garnered 7 orphan drug designations (ODDs) and 4 rare pediatric disease designations.
“I am especially proud of the outcomes of our successful interactions with the FDA via 2 type B pre-investigational new drug (IND) meetings and the Medicines and Healthcare products Regulatory Agency (MHRA) via 3 Scientific Advice meetings, which paved the way for the implementation of our vision of a pragmatic, faster, and more cost-effective approach to gene therapy development,” Lemoine wrote in the post.1 “I will continue working as a consultant for Bloomsbury to assist the company with a number of transactions which will seek to provide a good home for each of its programs, about which announcements will be made in due course.”
Notably, BGT-OTCD's development constituted a collaborative effort between Bloomsbury and University College London (UCL).2 The FDA granted ODD to the therapy in August 2023 and HORACE began recruiting patients in November 2023.2,3 BGT-OTCD is delivered via a single intravenous injection and utilizes the AAV-LK03 vector, with high specificity for the cells of the liver. UCL also submitted a clinical trial application to the MHRA in December 2022 that was later cleared by the agency in May 2023.4,5
“OTCD is a rare genetic disorder that is characterized by complete or partial lack of the OTC enzyme which causes too much ammonia to accumulate in the body,” Anupam Chakrapani, a consultant in metabolic medicine at Great Ormond Street Hospital and HORACE’s principal investigator, said in a May 2023 statement.5 “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. 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.”
In addition to BGT-OTCD, Bloosmbury’s pipeline also includes preclinical programs such as BGT-NPC, an investigational AAV vector-based gene therapy intended to treat Niemann-Pick disease type C, and BGT-DTDS, an investigational AAV vector-based gene therapy intended to treat dopamine transporter deficiency syndrome.6,7 BGT-NPC received rare pediatric disease designation (RPDD) from the FDA in August 2023 and BGT-DTDS received the same designation from the agency in May 2023. The company also has preclinical programs for Parkinson disease and infantile neuroaxonal dystrophy.8
REFERENCES
1. Adrien LemoineAdrien Lemoine. LinkedIn post. May 1, 2025. Accessed May 6, 2025. https://www.linkedin.com/posts/adrien-lemoine-a805aa_dear-network-it-is-with-a-heavy-heart-that-activity-7323625722093182976-vDoz/?utm_source=share&utm_medium=member_desktop&rcm=ACoAAAL-ytUB8-b9ftTDxr8xUk-LggBDpR8i5Io
2. Bloomsbury Genetic Therapies receives orphan drug designation from the U.S. FDA for BGT-OTCD for the treatment of ornithine transcarbamylase deficiency (OTCD). News release. Bloomsbury Genetic Therapies Limited. August 9, 2023. Accessed May 6, 2025. https://bloomsburygtx.com/bloomsbury-genetic-therapies-receives-orphan-drug-designation-from-the-u-s-fda-for-bgt-otcd-for-the-treatment-of-ornithine-transcarbamylase-deficiency-otcd/
3. 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). News release. Bloomsbury Genetic Therapies. November 16, 2023. Accessed May 6, 2025. https://bloomsburygtx.com/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/
4. Bloomsbury Genetic Therapies announces CTA filing for a phase I/II study for its lead gene therapy program. News release. Bloomsbury Genetic Therapies Limited. December 7, 2022. Accessed May 6, 2025. https://bloomsburygtx.com/bloomsbury-genetic-therapies-announces-cta-filing-for-a-phase-i-ii-study-for-its-lead-gene-therapy-program/
5. Bloomsbury Genetic Therapies announces clinical trial application accepted by U.K. regulatory agency to initiate phase 1/2 gene therapy trial of BGT-OTCD for the treatment of ornithine transcarbamylase deficiency (OTCD). News release. Bloomsbury Genetic Therapies Limited. May 15, 2023. Accessed May 6, 2025. https://bloomsburygtx.com/bloomsbury-genetic-therapies-announces-clinical-trial-application-accepted-by-u-k-regulatory-agency-to-initiate-phase-1-2-gene-therapy-trial-of-bgt-otcd-for-the-treatment-of-ornithine-transcarbamylas/
6. Bloomsbury Genetic Therapies receives rare pediatric disease designation from the U.S. FDA for BGT-NPC for the treatment of Niemann-Pick disease type C (NPC). News release. Bloomsbury Genetic Therapies Limited. August 21, 2023. Accessed May 6, 2025. https://bloomsburygtx.com/bloomsbury-genetic-therapies-receives-rare-pediatric-disease-designation-from-the-u-s-fda-for-bgt-npc-for-the-treatment-of-niemann-pick-disease-type-c-npc/
7. Bloomsbury Genetic Therapies receives rare paediatric disease designation from the U.S. FDA for BGT-DTDS for the treatment of dopamine transporter deficiency syndrome (DTDS). News release. Bloomsbury Genetic Therapies Limited. May 3, 2023. Accessed May 6, 2025. https://bloomsburygtx.com/bloomsbury-receives-rare-paediatric-disease-designation-from-the-u-s-fda-for-bgt-dtds-for-the-treatment-of-dopamine-transporter-deficiency-syndrome-dtds/
8. Pipeline overview. Bloomsbury Genetic Therapies. Website. Accessed May 6, 2025. https://bloomsburygtx.com/pipeline/
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