Wave Life Sciences Announces Positive Update from Ongoing RestorAATion-2 Trial of WVE-006 in Alpha-1 Antitrypsin Deficiency

Achieved durable production of serum AAT protein at levels associated with a lower risk of AATD liver and lung diseases following repeat 200 mg doses of WVE-006 (11.9 µM total AAT, 7.2 µM M-AAT) First-ever demonstration of therapeutically restored physiological serum AAT production in a Pi*ZZ individual during a non-drug related acute phase response (20.6 µM total AAT, 10.3 µM M-AAT) Single dose of 400 mg achieved 12.8 µM total AAT and 5.3 µM M-AAT; ongoing 400 mg multidose cohort has potential to deliver ...