WVE-006 is a first-in-class RNA editing therapeutic candidate designed to correct the root cause of disease for the 200,000 individuals in the U.S. and Europe living with AATD; no currently approved ...

Prime Medicine announces promising preclinical results for a novel AATD treatment using advanced Prime Editing technology, aiming for clinical trials by mid-2026. Prime Medicine, Inc. announced the ...

Austin, Texas, Jan. 13, 2026 (GLOBE NEWSWIRE) -- Alpha-1 Antitrypsin Deficiency Market Size & Growth Analysis: According to SNS Insider, The Alpha-1 Antitrypsin Deficiency Market was valued at USD ...

Alpha-1 antitrypsin deficiency (AATD) is a hereditary condition characterised by the misfolding of the alpha-1 antitrypsin (AAT) protein, resulting in its hepatic accumulation and compromised protease ...

Korro Bio, Inc., a biopharmaceutical company focused on RNA editing therapies, announced that the European Medicines Agency (EMA) has granted orphan drug designation for its investigational drug ...

Alpha-1-antitrypsin is a so-called protease inhibitor, a type of enzyme inhibitor. It is produced in the liver but exerts its effects in the lungs, where it regulates immune cell activity. This ...

Researchers at Saint Louis University's School of Medicine, in collaboration with Arrowhead Pharmaceuticals and Takeda Pharmaceuticals, report the first effective drug to treat a rare, genetic liver ...

Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder characterized by the misfolding and accumulation of the mutant variant of alpha-1 antitrypsin (AAT) within hepatocytes, which limits its ...

CAMBRIDGE, Mass., November 12, 2025 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA ...

New Data Demonstrate Proportion of Corrected M-AAT Reached a Mean of 91% of Total AAT in Circulation at Day 28 Following BEAM-302 Treatment in 60 mg Cohort (n=3) Mean Decrease of 79% in Mutant Z-AAT ...

David H. Perlmutter, MD, Vira I. Heinz Endowed Chairman, Department of Pediatrics, Distinguished Professor of Pediatrics, Professor of Cell Biology, University of Pittsburgh School of Medicine; ...