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 ...

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 ...

Rising prevalence of genetic respiratory disorders, expanding healthcare infrastructure, and strong R&D investments fuel sustained market growth worldwide.Austin, Texas, Jan. 13, 2026 (GLOBE NEWSWIRE) ...

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 ...

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 ...

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 ...

Study designed to demonstrate impact of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% (Alpha-1 15%), an alpha 1 antitrypsin treatment, compared with Liquid Alpha1-Proteinase Inhibitor (Human) ...

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 ...

MOLINE, Ill. — November is Alpha-1 Awareness Month, a time dedicated to increasing understanding of Alpha-1 Antitrypsin Deficiency, a rare genetic condition that can cause serious lung and liver ...

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; ...