Bloomsbury Genetic Therapies' new pipeline programme offers Parkinson's hope

6 September 2023

UCLB spinout Bloomsbury Genetic Therapies Limited has disclosed its new pipeline programme BGT-PD: an investigational gene therapy candidate for Parkinson’s disease (PD).

The clinical-stage biotechnology company is developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies.

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 programme, 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 reported “compelling” preclinical efficacy and safety, including neuroprotective effects, which suggested a potential therapeutic benefit in Parkinson’s Disease, 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 knowledge and experience gained from the BGT-DTDS programme, 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 described as “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 indicates a potential effect on non-motor symptoms, with a reduction in anxiety behaviours observed in animals treated with BGT-PD.

“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.”

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