Posted by Michael Wonder on 30 Aug 2023
Bloomsbury Genetic Therapies receives rare paediatric disease designation from the US FDA for BGT-INAD for the treatment of infantile neuroaxonal dystrophy
29 August 2023 - Bloomsbury Genetic Therapies Limited announced today that the US FDA has granted rare paediatric disease designation for BGT-INAD, the Company’s investigational gene therapy for the treatment of infantile neuroaxonal dystrophy,
In June, the Company announced that the UK 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.
