Bloomsbury Genetic Therapies Receives Orphan Drug Designations from the European Commission and the US FDA for BGT-INAD for the Treatment of Infantile Neuroaxonal Dystrophy and Provides Update on Type B Pre-IND Meeting with the US FDA

– Type B pre-IND meeting with the US FDA provides detailed advice on the Company’s CMC, non-clinical and clinical development plans for BGT-INAD –


London, UK, 23 October 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 Company has received Orphan Drug Designations (ODD) from the US Food and Drug Administration (FDA) and the European Commission (EC) for BGT-INAD, an investigational gene therapy for the treatment of Infantile Neuroaxonal Dystrophy (INAD).

The Company also held a constructive Type B Pre-Investigational New Drug Application (IND) meeting with the US FDA, and received detailed advice on the Company’s plans for chemistry, manufacturing, and controls (CMC), non-clinical and clinical development for BGT-INAD. Following the US FDA advice and the UK Medicines and Healthcare Products Regulatory Agency (MHRA) feedback disclosed in June 2023, the Company plans to complete preclinical development in line with MHRA and FDA’s feedback ahead of a starting a Phase 1/2/3 clinical trial in Europe and the US. The Company is currently completing preclinical studies for BGT-INAD in collaboration with its partner University College London (UCL).

“All four of Bloomsbury’s rare disease gene therapy programs have now received EU and US orphan drug designations and US rare pediatric disease designations, which recognise the significant unmet need that we are seeking to address and further underscores the necessity of bringing these important therapeutic options to patients,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “We are also grateful to the FDA for the collaborative nature of the Type B meeting and their detailed advice on the development plans for BGT-INAD and we look forward to an ongoing, constructive dialogue with the FDA as we advance BGT-INAD into the next phase of development.”

In the US, the FDA grants ODD to a drug or biologic intended to treat a rare disease or condition, which generally includes a disease or condition that affects fewer than 200,000 individuals in the US. ODD provides opportunities for grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the US in the event of regulatory approval.

Similarly, in the EU, ODD is granted by the EC based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products (COMP). It is intended to encourage the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases.  This designation provides special incentives for sponsors, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees. In addition, if approved for marketing, this designation will provide ten years of marketing exclusivity.


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JW Communications

Julia Wilson

Tel: +44 (0)7818 430877


About Infantile Neuroaxonal Dystrophy

Infantile Neuroaxonal Dystrophy (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.



BGT-INAD is an investigational AAV9 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. BGT-INAD has been granted Orphan Drug Designation (ODD) for the treatment of INAD by the European Commission (EC) and the US Food and Drug Administration (FDA), as well as Rare Pediatric Disease Designation (RPDD) from the FDA.


About Bloomsbury Genetic Therapies

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