Beam Therapeutics Secures FDA Orphan Drug Designation for BEAM-302 in AATD
Beam Therapeutics Inc. announced that the FDA has granted Orphan Drug Designation to its investigational therapy, BEAM-302, for the treatment of alpha-1 antitrypsin deficiency (AATD). BEAM-302 is a liver-targeting lipid nanoparticle (LNP) formulation containing a guide RNA and an mRNA encoding a base editor designed to correct the underlying genetic mutation responsible for AATD. The designation provides Beam with potential development incentives, including tax credits, user fee exemptions, and up to seven years of market exclusivity upon approval.
“Receiving orphan drug designation for ...
