LogicBio Therapeutics’ (NASDAQ:LOGC) investigational pediatric genome editing therapy, LB-001, received FDA orphan drug designation for the treatment of methylmalonic acidemia (MMA).
MMA, a rare disease caused by mutations in the MUT gene, begins in early childhood and prevents the body from processing certain fats and proteins. This results in a toxic accumulation of metabolites that causes infections, neurodevelopmental disabilities, chronic kidney disease and even death. There are currently no approved therapies for MMA.
LB-001 – currently in pre-IND studies – is a genome editing therapy designed to incorporate a functioning version of the faulty MUT gene into the genome of MMA patients.
“We believe that LB-001 has potential to transform the treatment of this devastating disease, and receiving this designation represents a step forward for our program,” Fred Chereau, LogicBio’s CEO, said in a statement.
