Solid Biosciences Inc.’s SGT-003 has been granted orphan drug designation by the FDA. The company’s next-generation Duchenne muscular dystrophy gene therapy candidate was also granted fast track designation last month.
Beam Therapeutics Inc. has offered a progress update on its genetic disease franchise. BEAM-302, the company’s priority genetic disease program, is a potential treatment for α1-antitrypsin deficiency (AATD).
