New Preclinical Data on CNS Programs to be presented at American Society of Gene & Cell Therapy 26th Annual Meeting 2023
London, UK, 6 April 2023 – 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 pre-clinical data from two of its brain-targeted programs, BGT-INAD and BGT-NPC, will be featured in posters at the upcoming American Society of Gene & Cell Therapy (“ASGCT”) 26th Annual Meeting 2023 to be held on 16-20 May 2023 in Los Angeles, USA.
ASGCT Poster Details:
Title: AAV9-mediated gene therapy in a knock-in mouse model of infantile neuroaxonal dystrophy
Session: Wednesday Poster Session
Poster Board Number: 685
Presenter: Amy Geard, University College London
Title: A novel small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann-Pick Type C1 Disease
Session: Wednesday Poster Session
Poster Board Number: 696
Presenter: Wenfei Liu, University College London
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Enquiries
JW Communications
Julia Wilson
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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 UCL and launched in October 2022 with £5M in Seed funding from UCL Technology Fund and is underpinned by world-leading gene therapy and rare disease expertise from the Company’s academic founders, Professor Paul Gissen, Professor Manju Kurian, Professor Ahad Rahim and Professor Simon Waddington. Bloomsbury is building a pipeline of highly differentiated first- or best-in-class programs. For more information, please visit www.bloomsburygtx.com
About BGT-INAD for Infantile Neuroaxonal Dystrophy (INAD)
BGT-INAD is a neuron-targeted AAV9 investigational gene therapy designed to provide a potentially curative solution to INAD patients following a one-time injection in the cerebrospinal fluid (CSF). BGT-INAD is currently completing preclinical studies, with compelling preclinical efficacy data already demonstrated.
About BGT-NPC DTDS for Niemann-Pick Disease Type-C (NPC)
BGT-NPC is a neuron-targeted AAV9 investigational gene therapy designed to provide a potentially curative solution to NPC patients following a one-time injection in the cerebrospinal fluid (CSF). BGT-NPC is currently completing preclinical studies, with compelling preclinical efficacy data already demonstrated.
About infantile neuroaxonal dystrophy (INAD)
INAD is a devastating and fatal autosomal recessive paediatric neurodegenerative disease caused by mutations in the PLA2G6 gene that affects the catalytic activity of its protein product, a calcium-independent phospholipase A2 protein located in mitochondria and axons. When PLA2G6 is defective, mitochondrial inner membrane integrity is damaged, and phospholipid accumulation in axons causes neuronal damage. Given greater disease awareness and advances in next generation sequencing technologies, INAD is increasingly diagnosed at a younger age, with the majority of affected children now diagnosed by 2 years of age. Symptoms usually begin between 6 months and 3 years of age and include a rapid decline in previously acquired skills such as walking and talking, with the subsequent evolution of seizure, axonal neuropathy and generalised spasticity leading to loss of neurodevelopmental skills and complete dependence for all daily living activities. From presentation, there is gradual loss of motor and cognitive skills, most losing ambulation by 5 years of age with the average age of death around 10 years of age. There are currently no disease-modifying treatments for INAD and affected patients derive limited clinical benefit from available medical therapies used for symptom control. The incidence of INAD is poorly documented and around 300 patients suffering from INAD have been identified currently worldwide.
About Niemann-Pick Disease Type-C (NPC)
NPC is a rare, inherited and fatal neurodegenerative disease. 95% of NPC cases are caused by mutations in the Npc1 gene that encodes for NPC1, a large glycoprotein which is embedded in the lysosomal membrane. Even though the exact function of NPC1 is unknown, when it is mutated or absent, both cholesterol and sphingolipids accumulate in the brain, liver, lungs, bone marrow, and spleen. Sphingolipids are known to play important roles in signal transduction and nerve-fibre insulation, and their abnormal storage leads to irreversible neurological damage. There are several disease subtypes which are categorised by the age of onset of neurological disease. The patients develop progressive disabilities such as impairment in swallowing and speech, epilepsy, cerebellar ataxia (an inability to coordinate balance, gait, extremity and eye movements), and progressive dementia. The speed of progression of symptoms depends on the age of onset, which ranges from just after birth through to late adulthood and after disease onset. There are no approved treatments for NPC in the US. Miglustat is approved in the EU, but its use only slows disease progression. There are no curative treatments currently approved for NPC and there is a significant need to improve the standard of care. The incidence of NPC is estimated to be ~1:100,000 live births worldwide.
