Bloomsbury Genetic Therapies receives Rare Pediatric Disease Designation from the US FDA for BGT-INAD for the Treatment of Infantile Neuroaxonal Dystrophy (INAD)

29 August 2023

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Bloomsbury Genetic Therapies Limited, a UCLB spinout developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, has been granted Rare Pediatric Disease Designation (RPDD) for BGT-INAD by the U.S. Food and Drug Administration (FDA) for 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 programmes 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.

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