Bloomsbury Genetic Therapies Showcases its First Preclinical Data on Parkinson’s Disease Program at the American Society of Gene & Cell Therapy 2024 Annual Meeting

London, UK, 9 May 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 presented new preclinical data on its gene therapy candidate, BGT-PD, in a poster presentation at the 27th Annual Meeting of American Society of Gene & Cell Therapy (ASGCT).

BGT-PD is a first-in-class AAV2 gene therapy designed to rebalance dopamine transporter activity in Parkinson’s disease (PD) patients affected by levodopa-induced dyskinesia (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 50% of PD patients are affected by LID after 5 years of levodopa treatment increasing to up to 90% after 10 years, with current treatment options providing only partial therapeutic benefit on LID. 1

In the poster presentation, titled “Proof-of-Concept Efficacy of BGT-PD, a Novel AAV-Based Gene Therapy for the Treatment of Levodopa-Induced Dyskinesia in Parkinson’s Disease” (Click here to view the poster), we showcased, for the first time, data from the pre-clinical proof-of-concept study of BGT-PD, which provide initial evidence of the therapeutic potential of our gene therapy approach to completely reverse LID by reinstating Dopamine Transporter (DAT) activity. DAT is critical for dopamine neurotransmission and its impaired activity has been implicated in PD pathophysiology and LID.2 A single administration of BGT-PD to the putamen of hemiparkinsonian rats with established LID demonstrated a profound and sustained anti-dyskinetic effect, with complete suppression of LID and no treatment-associated side effects, indicating a preliminary positive safety profile of the approach. Additionally, non-motor assessments have shown a potential treatment effect on anxiety-like behaviours. Extensive histopathology assessments have also been conducted, further confirming the safety and mechanism of action of the proposed approach, details of which will be disclosed in a future publication. The company is currently planning follow-up studies to evaluate the long-term efficacy and safety of this approach for LID as well as additional therapeutic benefits in PD, including disease-modifying effects.

“Findings from this study are highly promising and provide compelling evidence of the potential of this innovative gene therapy approach to treat LID and preserve levodopa’s therapeutic benefit in PD,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury “We look forward to continuing our pre-clinical development efforts and generating further data to confirm the long-term safety and efficacy of our approach, which will bring us closer to providing a potentially transformative treatment to the PD community, with potential therapeutic benefits beyond motor function.”

 

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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 a first-in-class investigational AAV2 gene therapy designed to provide increased dopamine transporter (DAT) activity in the nigrostriatal system of Parkinson’s disease (PD) patients affected by levodopa-induced dyskinesia (LID) 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.

 

References:

  1. Fabbrini, G., Brotchie, J. M., Grandas, F., Nomoto, M., & Goetz, C. G. (2007). Levodopa-induced dyskinesias. Movement disorders : official journal of the Movement Disorder Society, 22(10), 1379–1389. https://doi.org/10.1002/mds.21475
  2. Bu, M.,  Matthew J Farrer, M.J., Khoshbouei, H. (2021). Dynamic control of the dopamine transporter in neurotransmission and homeostasis. NPJ Parkinsons Dis, 7(1):22. doi: 10.1038/s41531-021-00161-2.