First Preclinical Data on Parkinson’s Disease Program to be presented at the American Society of Gene & Cell Therapy 27th Annual Meeting 2024

London, UK, 03 April 2024 – Bloomsbury Genetic Therapies Limited (“Bloomsbury”), 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 new preclinical data supporting BGT-PD, the Company’s AAV2 gene therapy candidate for the treatment of levodopa-induced dyskinesia (LID) in Parkinson’s disease (PD), will be featured in a poster presentation at the upcoming 27th Annual Meeting of American Society of Gene & Cell Therapy (ASGCT), which is being held in Baltimore, USA, from 7-11 May 2024.

BGT-PD is a first-in-class AAV2 gene therapy designed to rebalance dopamine transporter activity in PD patients affected by LID following a single intra-brain injection. LID is a prevalent and debilitating side effect of chronic levodopa treatment, the current gold-standard for managing PD motor symptoms. Around half of PD patients are affected by LID after 5 years of levodopa treatment and up to 90% after 10 years, with current treatment options providing only partial therapeutic benefit on LID.

The Company will present, for the first time, data from an initial proof-of-concept study, which demonstrated a profound anti-dyskinetic effect of BGT-PD, with no side effects observed indicating a positive preliminary safety profile of the approach.

Details of the poster presentation at ASGCT Annual meeting:

Title: Proof-of-Concept Efficacy of BGT-PD, a Novel AAV-Based Gene Therapy for the Treatment of Levodopa-Induced Dyskinesia (LID) in Parkinson’s Disease

Session: Thursday Poster Session – Neurological Diseases

Abstract Number: 1112

Presenter: Dr Giulia Leoni, Bloomsbury Genetic Therapies

 

 

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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 www.bloomsburygtx.com

 

About BGT-PD

BGT-PD is an investigational AAV2 gene therapy designed to provide increased dopamine transporter (DAT) activity in the nigrostriatal system of Parkinson’s disease (PD) patients following a one-time intra-brain injection. The investigational therapy was originally developed as a potentially curative solution for Dopamine Transporter Deficiency Syndrome (DTDS), a rare, monogenic disorder caused by mutations in DAT gene leading to parkinsonism-like clinical features and premature death in childhood/teenage years.  BGT-PD is currently being investigated in preclinical studies as a potentially symptomatic and disease modifying treatment for PD patients. For more information, please visit www.bloomsburygtx.com/pipeline/bgt-pd/

 

About Parkinson’s Disease

PD is a progressive neurodegenerative disorder characterised by gradual loss of dopamine-producing brain cells resulting in progressive impairment of motor function (tremors, muscle rigidity, bradykinesia and postural instability) as well as non-motor function (e.g., cognitive impairment, mood alterations, sleep disturbance). Global estimates in 2019 showed over 8.5 million individuals affected by PD.

Standard of care for PD patients involves a multidisciplinary approach to manage and alleviate symptoms and improve overall quality of life. There are no approved disease-modifying therapies for PD and levodopa, a precursor of dopamine, remains the most effective, first-line treatment to control motor symptoms in early disease stages.  However, as the disease progresses, chronic treatment with levodopa can lead to the development of motor fluctuations and levodopa-induced dyskinesia in a majority of PD patients, limiting its long-term efficacy.