Bloomsbury Genetic Therapies Completes Successful Scientific Advice Meeting with the MHRA regarding its BGT-NPC Program

– Company intends to move forward with toxicology and biodistribution study in rodents ahead of a single, Phase 1/2/3 trial in Niemann-Pick Disease Type C (NPC) –


London, UK, 12 June 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, today announced that a successful scientific advice meeting was held with the UK Medicines and Healthcare products Regulatory Agency (MHRA) on 26 April 2023 confirming the formal toxicology and biodistribution requirements to commence a single, Phase 1/2/3 clinical trial with the Company’s product candidate, BGT-NPC, as a potential new therapeutic for the treatment of NPC.

The Company met with the MHRA to discuss its preclinical data package and plans. The MHRA confirmed that it was supportive of the Company’s proposal to conduct a single toxicology and biodistribution study in rodents supplemented by published data from precedent intra-brain AAV9 gene therapies, prior to initiating a registrational, Phase 1/2/3 clinical trial for BGT-NPC in the UK. There was no requirement to conduct studies in a larger animal model. Delivering accelerated development is especially important in indications such as NPC, where no curative treatments are currently approved and there is a significant need to improve the standard of care.

The Company is currently completing efficacy studies for BGT-NPC in collaboration with its partner University College London. As a result of the advice and formal feedback received from the MHRA, subject to initiating a manufacturing campaign with its chosen vector Contract Development and Manufacturing Organisation (CDMO) partner, the Company intends to initiate a toxicology & biodistribution study in 2024.

“Following our recent, positive regulatory interactions regarding our BGT-DTDS program, this is another success for Bloomsbury’s pragmatic development approach,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “This result confirms our ability to deliver accelerated clinical translation and capital-efficient development by delivering CTA-enabling activities in rodents, as opposed to more expensive and time-consuming studies in non-human primates or larger animals.”


– ENDS –



JW Communications

Julia Wilson

Tel: +44 (0)7818 430877


About Bloomsbury

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


BGT-NPC is a neuron-targeted AAV9 investigational gene therapy designed to provide a potentially curative solution to NPC patients following a one-time injection in the cerebrospinal fluid (CSF). BGT-NPC is currently completing preclinical studies, with compelling preclinical efficacy data already demonstrated.

About Niemann-Pick Disease Type-C (NPC)

NPC is a rare, inherited and fatal neurodegenerative disease. 95% of NPC cases are caused by mutations in the Npc1 gene that encodes for NPC1, a large glycoprotein which is embedded in the lysosomal membrane. Even though the exact function of NPC1 is unknown, when it is mutated or absent, both cholesterol and sphingolipids accumulate in the brain, liver, lungs, bone marrow, and spleen. Sphingolipids are known to play important roles in signal transduction and nerve-fibre insulation, and their abnormal storage leads to irreversible neurological damage. There are several disease subtypes which are categorised by the age of onset of neurological disease. The patients develop progressive disabilities such as impairment in swallowing and speech, epilepsy, cerebellar ataxia (an inability to coordinate balance, gait, extremity and eye movements), and progressive dementia. The speed of progression of symptoms depends on the age of onset, which ranges from just after birth through to late adulthood and after disease onset. There are no approved treatments for NPC in the US. Miglustat is approved in the EU, but its use only slows disease progression. There are no curative treatments currently approved for NPC and there is a significant need to improve the standard of care. The incidence of NPC is estimated to be ~1:100,000 live births worldwide.