Posted by Michael Wonder on 21 Oct 2020
Selecta Biosciences and AskBio receive FDA rare paediatric disease designation for their gene therapy for methylmalonic acidemia
20 October 2020 - Selecta Biosciences and Asklepios BioPharmaceutical today announced the U.S. FDA has granted rare paediatric disease designation to MMA-101 for the treatment of isolated methylmalonic acidaemia due to methylmalonyl-CoA mutase gene mutations.
AskBio and Selecta expect to initiate a Phase 1 clinical trial of MMA-101 and ImmTOR for patients with methylmalonic acidaemia in 1H 2021.
