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, has announced that an article highlighting first preclinical data from BGT-NPC, as a potential new brain-targeted therapeutic for the treatment of Niemann-Pick Type C Disease (NPC), was published in Cells.
The article, titled “A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease”, is available online.
The work, conducted by our colleagues at University College London (UCL) led by Professor Ahad Rahim, described for the first time the novel, minimal NPC1 endogenous promoter used in BGT-NPC demonstrating greater therapeutic effects as compared to a broad range of common promoters used in gene therapy. Furthermore, its small size allows for the large NPC1 gene to be efficiently accommodated in our AAV9 vector, which removes downstream manufacturing hurdles. Following intracerebroventricular (ICV) administration in neonatal NPC mice, BGT-NPC showed enhanced NPC1 protein expression, resulting in a significant improvement of animal survival, attenuated neuropathology, as well as improved locomotor deficit.
Following the recent positive feedback received from the UK Medicines and Healthcare products Regulatory Agency (MHRA), confirming the Company’s preclinical plans prior to initiating a registrational Phase 1/2/3 clinical trial for BGT-NPC, further pre-clinical studies are currently underway in juvenile NPC1 mice which will be pivotal to support advancement into the final toxicology and biodistribution study planned for 2024.
“We are pleased to announce the publication of our work leading to the identification of a novel, minimal NPC1 promoter used in BGT-NPC, demonstrating the significant therapeutic potential of brain-targeted AAV-mediated gene therapy for the treatment of NPC1.” said Professor Ahad Rahim (pictured), Professor of Translational Neuroscience at University College London (UCL), Wellcome Chair in Pharmacology and the Head of Pharmacology Department at the UCL School of Pharmacy. “We look forward to continuing our efforts with our partner, Bloomsbury Genetic Therapies, to accelerate the translation of this program into the clinic and provide a novel, effective treatment to patients affected by this devastating disease”.