Posted by Michael Wonder on 17 Jul 2019
LogicBio Therapeutics receives rare paediatric disease designation for LB-001 for the treatment of methylmalonic acidaemia
16 July 2019 - LogicBio Therapeutics today announced the U.S. FDA has granted rare paediatric disease designation to LB-001, a recombinant adeno-associated viral vector with human methylmalonyl-COA mutase gene for the treatment of methylmalonic acidaemia.
In addition to rare paediatric disease designation, LogicBio previously received orphan drug designation from the FDA for LB-001.
LB-001 is an investigational paediatric genome editing therapy based on LogicBio’s GeneRide technology.
