Bloomsbury Genetic Therapies Receives Rare Pediatric Disease Designation from the U.S. FDA for BGT-INAD for the Treatment of Infantile Neuroaxonal Dystrophy (INAD) and Announces Upcoming Type B Pre-IND Meeting with the U.S. FDA

– RPDD and Type B pre-IND meeting granted by the FDA –


London, UK, 29 August 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 the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for BGT-INAD, the Company’s investigational gene therapy for the treatment of INAD.

The FDA has also granted the Company a Type B Pre-IND meeting to be held in October to discuss the Company’s plans for BGT-INAD’s remaining safety and biodistribution studies and clinical development strategy. In June, the Company announced that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) supported the Company’s plans to conduct a single toxicology and biodistribution study in rodents ahead of a single, registrational Phase 1/2/3 clinical trial. The Company is currently completing efficacy studies for BGT-INAD in collaboration with its partner University College London (UCL). 

“This is our fourth RPDD in as many months, following those already obtained for our programs BGT-DTDS, BGT-OTCD and BGT-NPC, which is a testament to the productivity of our highly experienced team” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “We are also very pleased that the FDA has granted us a Type B pre-IND meeting where we will be able to discuss our global development plans for the program, following our successful interaction with the MHRA earlier this year, and are looking forward to a productive exchange”.

The FDA grants RPDD for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If a Biologics Licensing Application (BLA) for BGT-INAD for the treatment of INAD is approved by the FDA, Bloomsbury may be eligible to receive a Priority Review Voucher (PRV) that can be redeemed to receive a priority review for any subsequent marketing application, or may be sold or transferred.


– ENDS –



JW Communications

Julia Wilson

Tel: +44 (0)7818 430877


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



BGT-INAD is an investigational AAV9 gene therapy designed to provide a potentially curative solution to Infantile Neuroaxonal Dystrophy (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. BGT-INAD has been granted Rare Pediatric Disease Designation (RPDD) by the U.S. Food and Drug Administration (FDA) for the treatment of INAD.


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 prevalence of INAD is poorly documented, it is estimated to be approximately 0.3/100,000 worldwide.