LogicBio Therapeutics, Inc. (LOGC), a genome editing company, reported that the US Food and Drug Administration (FDA) has granted orphan drug designation to LB-001, a recombinant adeno-associated viral vector with human methylmalonyl-COA mutase (MUT) gene for the treatment of methylmalonic acidemia (MMA).
“We believe that LB-001 has potential to transform the treatment of this devastating disease,” said Chief Executive Fred Chereau, “and receiving this designation represents a step forward for our program.”
Orphan drug designation is granted to drugs and biologics intended to treat, diagnose or prevent rare diseases and disorders that affect fewer than 200,000 people in the US. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the US following FDA marketing approval.
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