Beam receives Orphan Drug status for genetic disorder asset BEAM-302

21 hours ago 1

The U.S. FDA has granted Orphan Drug designation to Beam Therapeutics' (NASDAQ:BEAM) BEAM-302, a liver-targeting lipid-nanoparticle formulation of a guide RNA and an mRNA encoding a base editor to treat the genetic disorder alpha-1 antitrypsin deficiency (AATD).
The candidate works

Recommended For You