Bloomsbury Genetic Therapies Announces New Program in Parkinson’s Disease

Bloomsbury Genetic Therapies Announces New Program in Parkinson’s Disease

London, UK, 6 September 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, is pleased to disclose its new pipeline program BGT-PD, an investigational gene therapy candidate for Parkinson’s disease (PD).

BGT-PD is an investigational AAV2 gene therapy designed to rebalance dopamine transporter (DAT) activity in the nigrostriatal system of idiopathic Parkinson’s disease (PD) patients following a single, targeted intra-brain injection. BGT-PD relies on the same gene therapy vector encoding for the DAT protein used in the Company’s BGT-DTDS program, which is being developed concurrently for the treatment of Dopamine Transporter Deficiency Syndrome (DTDS), a rare, monogenic disorder caused by mutations in the DAT gene leading to parkinsonism-like clinical features and premature death in childhood/teenage years. BGT-DTDS has already demonstrated compelling preclinical efficacy and safety, including neuroprotective effects, which suggested a potential therapeutic benefit in PD, and the Company has recently received positive feedback from the UK Medicines and Healthcare products Regulatory Agency (MHRA) supporting the filing of a clinical trial application for BGT-DTDS. BGT-PD will leverage the extensive knowledge and experience gained from the BGT-DTDS program, from DAT biology and its central role in both diseases to product manufacturing and characterisation, providing both de-risking and acceleration of a potential clinical translation in PD.

The Company has initiated a preclinical proof-of-concept study for BGT-PD which will be completed before the end of 2023. Preliminary data following single administration of BGT-PD in the caudate/putamen of PD animals are very promising, indicating a profound effect of the gene therapy on levodopa-induced dyskinesia compared to vehicle-treated controls, with a magnitude of effect markedly superior to that achieved by existing medication for dyskinesia. Data also indicate a potential effect on non-motor symptoms, with a reduction in anxiety behaviours observed in animals treated with BGT-PD. Building on these compelling preliminary findings, the Company is developing a comprehensive preclinical program to further evaluate the therapeutic potential of BGT-PD as a disease-modifying approach.

“We are excited to announce BGT-PD as our fifth program. It is a true case of developments in an ultra-orphan, monogenic disease de-risking the development of the same drug candidate in a complex, multifactorial disease,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “We look forward to sharing our preclinical findings as we progress our novel therapeutic strategy for this debilitating disease with limited treatment options.”