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

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

- Company intends to move forward with a single, Phase 1/2/3 trial in dopamine transporter deficiency syndrome (DTDS) -

London, UK, 26 April 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 1 March  2023 confirming the formal preclinical requirements to commence a single, Phase 1/2/3 clinical trial with the Company’s product candidate, BGT-DTDS, as a potential new therapeutic for the treatment of DTDS. 

The Company discussed its preclinical data package with the MHRA and, based upon the preclinical efficacy data generated in the BGT-DTDS program to date and on precedents from other intra-parenchymal AAV2 gene therapies, in particular in aromatic l-amino acid decarboxylase (AADC) deficiency, a condition similar to DTDS, the MHRA confirmed that it was supportive of the Company initiating a clinical trial for BGT-DTDS in the UK without the need for further non-clinical studies. Delivering such accelerated development is especially key in indications such as DTDS where there is high unmet medical need and no effective treatments.

As a result of the advice and formal feedback received from the MHRA, subject to completing a GMP manufacturing campaign with its chosen vector CDMO partner, the Company intends to move forward with its clinical development plans for BGT-DTDS and aims to submit a combined Clinical Trial and Ethics Committee application for a single, Phase 1/2/3 registrational clinical trial to the MHRA in 2024. The Company is also planning to engage with the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) ahead of trial expansion in those geographies, where it has already obtained orphan drug designations.

“We were grateful to have had this constructive dialogue with the MHRA. It confirmed that our preclinical program includes all the studies and data needed to progress the BGT-DTDS program into the clinic,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “We see this outcome as a validation of our development strategy, both for BGT-DTDS and across our portfolio – namely, relying on platforms based on de-risked capsids/routes of administration to deliver accelerated clinical translation and capital-efficient development. We expect to see further benefits resulting from our approach as we progress our programs to registration.”