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Treatment for infantile neuroaxonal dystrophy a step closer as Bloomsbury Genetic Therapies receives Orphan Drug Designations

23 October 2023

Microscope in lab

UCLB spinout Bloomsbury Genetic Therapies Limited 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 clinical-stage biotechnology company is developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies.

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.”

Further Information: 

Bloomsbury Genetic Therapies