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 ...

To the point. Hereditary disease alpha-1-antitrypsin deficiency: Researchers have discovered why some patients remain healthy despite a genetic defect, while others develop severe liver fibrosis Deep ...

About Alpha-1 Antitrypsin Deficiency (AATD) and KRRO-110. AATD is a genetic disorder most commonly caused by a single missense mutation (G-to-A) in the SERPINA1 gene.

AIRNA’s lead program has the potential to be a best-in-class therapeutic for alpha-1 antitrypsin deficiency (AATD). AIRNA has headquarters in Cambridge, MA, with research operations in Tübingen ...

CAMBRIDGE, Mass. &, TÜBINGEN, Germany, May 13, 2025--AIRNA today announced preclinical proof-of-concept data supporting its best-in-class RNA editing strategy for alpha-1 antitrypsin deficiency ...

Korro Bio announces FDA orphan drug designation for KRRO-110, targeting Alpha-1 Antitrypsin Deficiency in clinical trials. ... March 14, 2025 — 08:12 am EDT.

In a dose-dependent manner, fazirsiran reduced serum and liver concentrations of Z-AAT and also improved histological measures in patients with homozygous ZZ alpha-1 antitrypsin deficiency ...

More information: Natalia Brzozowska et al, Selection for somatic escape variants in SERPINA1 in the liver of patients with ...

Mar 14, 2025 8:00am EDT. ... About Alpha-1 Antitrypsin Deficiency (AATD) and KRRO-110. AATD is a genetic disorder most commonly caused by a single missense mutation (G-to-A) in the SERPINA1 gene.

AIRNA's lead program has the potential to be a best-in-class therapeutic for alpha-1 antitrypsin deficiency (AATD). AIRNA has headquarters in Cambridge, MA, with research operations in Tübingen ...