Bloomsbury Genetic Therapies is focused on developing innovative gene therapy solutions for rare neurological and metabolic diseases. Their approach utilizes well-characterized adeno-associated virus (AAV) capsids and optimized vector constructs, which are designed to deliver therapeutic genes effectively. The company is particularly dedicated to addressing high unmet medical needs in conditions such as Parkinson's Disease (PD) and Dopamine Transporter Deficiency Syndrome (DTDS). Their investigational gene therapy, BGT-PD, aims to rebalance dopamine transporter activity in PD patients, showing promise in reducing levodopa-induced dyskinesia (LID) and potentially offering disease-modifying effects.

The company’s pipeline includes BGT-PD, which has demonstrated compelling preclinical results, indicating a significant impact on LID compared to existing treatments. This therapy relies on the same gene therapy vector as their BGT-DTDS program, which targets a rare genetic disorder linked to parkinsonism. Bloomsbury Genetic Therapies is committed to advancing these programs through rigorous preclinical studies, leveraging partnerships with academic institutions and contract development organizations to enhance their research and development capabilities. Their vision is to establish a robust pipeline of genetic therapies that can transform treatment options for patients suffering from these challenging conditions.

United Kingdom

Bloomsbury Genetic Therapies ("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.

Central Nervous System (CNS)

Genetic Diseases

Lymphatic Diseases

Metabolic Diseases

Nervous System Diseases

Bloomsbury Genetic Therapies

Ornithine transcarbmylase deficiency

BGT-OTCD

Gene Therapy

Dopamine Transporter Deficiency Syndrome

BGT-DTDS

Niemann Pick Disease Type C

BGT-NPC

Infantile Neuroaxonal Dystrophy

BGT-INAD

Cambridge, Cambridgeshire, United Kingdom

Europe - West

Adeno-associated viral vector (AAV)

Biologics

Therapeutic

Bloomsbury Genetic Therapies

Company Details