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 ...

In a poster presentation entitled “Optimized RESTORE+ oligonucleotides for an efficacious and safe RNA base editing treatment for alpha-1 antitrypsin deficiency,” AIRNA’s rAIR-100 research ...

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.

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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 ...

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.

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

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 ...

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Alpha-1 Antitrypsin Deficiency Companies are Kamada Pharmaceuticals, Arrowhead Pharmaceuticals, Takeda, Mereo BioPharma, AstraZeneca, Inhibrx, Novo Nordisk, and others. (Albany, USA) DelveInsight ...

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 ...

KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency Provided by GlobeNewswire Mar 14, 2025 12:00pm. CAMBRIDGE, Mass., March 14, ...