Bloomsbury Genetic Therapies Outlines Pre-Clinical Data on CNS Programs Presented at the American Society of Gene and Cell Therapy 26th Annual Meeting

Bloomsbury Genetic Therapies Outlines Pre-Clinical Data on CNS Programs Presented at the American Society of Gene and Cell Therapy 26th Annual Meeting

London, UK, 18 May 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, announced today that pre-clinical data from two of its brain-targeted programs, BGT-INAD and BGT-NPC for the treatment of Infantile Neuroaxonal Dystrophy (INAD) and Niemann-Pick Type C Disease (NPC) respectively, were presented at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting in Los Angeles, California.

The presentation entitled “AAV9-Mediated Gene Therapy in a Knock-In Mouse Model of Infantile Neuroaxonal Dystrophy” (Poster No. 685) highlighted pre-clinical proof-of-concept results for BGT-INAD, a first-in-class AAV gene therapy for the treatment of INAD. Treatment of neonatal and juvenile pla2g6-inad mice with BGT-INAD via intracerebroventricular (ICV) administration resulted in significantly extended survival, improved motor functions, as well as ameliorated neurodegeneration in multiple brain regions, supporting the therapeutic potential of this approach. The long-term study in juvenile mice is currently ongoing with results expected in the second half of this year.

A second presentation entitled “A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann-Pick Type C Disease” (Poster No. 696) detailed key pre-clinical in vivo findings demonstrating the superior therapeutic efficacy of the novel, minimal NPC1 endogenous promoter used in BGT-NPC compared to common promoters used in gene therapy. Following ICV administration in clinically relevant NPC1 mouse models, treatment with BGT-NPC demonstrated enhanced NPC1 protein expression, resulting in a significant increase of animal survival, attenuated neuropathology, as well as improved locomotor deficit.

Following positive regulatory engagements with the UK Medicines and Healthcare products Regulatory Agency (MHRA) in April, whose outcomes will be communicated in June, and building on the compelling results achieved to date, further pre-clinical studies are currently underway to support successful clinical translation of these two programs.

“The presentations at this year’s ASGCT meeting have highlighted key positive pre-clinical results for our investigational AAV9-based gene therapies for NPC and INAD, supporting our advancement of these programs,” said Adrien Lemoine, Founder and Chief Executive Officer of Bloomsbury. “We are continuing our efforts, together with our academic partners at UCL, to accelerate the delivery of these promising therapies to patients and families affected by these devastating conditions.”