Shares in Wave Life Sciences rose by 74% after it reported clinical data with a pioneering new RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD). Wave claims this is the first clinical ...
Chi - Submitted regulatory filing for first-in-human clinical study of KRRO-110 in patients with alpha-1 antitrypsin deficiency (AATD) - Formed Clinical Advisory Board (CAB) of leading lung and ...
Krystal Biotech (KRYS) clinical data updates for both KB408 and KB407, the Company’s clinical-stage, inhaled genetic medicine programs in Phase 1 for the treatment of rare respiratory diseases.
along with its BEAM-302 base-editing candidate for alpha-1 antitrypsin deficiency (AATD) and BEAM-301, an in vivo lipid nanoparticle (LNP) therapy for glycogen storage disease 1a. Pfizer is ...