Alpha-1 antitrypsin deficiency (AATD) ... Fazirsiran for Adults With Alpha-1 Antitrypsin Deficiency Liver Disease: A Phase 2 Placebo Controlled Trial (SEQUOIA). Gastroenterology (2024).

A new approach. Alpha-1 antitrypsin deficiency is a genetic disorder that harms the lungs, the liver or sometimes both. About 80,000 to 100,000 people in the U.S. have it, all because of mutations ...

The global Alpha-1 Antitrypsin Deficiency market is expected to reach $2.96 billion by 2034. Then, you have the other disorder type it is going to develop a Prime Editing therapy for, which is ...

Alpha-1 antitrypsin deficiency (AATD) is an inherited genetic disorder that affects the lungs and/or liver, leading to early onset emphysema and liver disease, and for which there is significant ...

Fazirsiran for Liver Disease Associated with Alpha 1-Antitrypsin Deficiency. 2022-08-11. DOI: 10.1056/NEJMdo006633. ... Mutant AAT protein accumulates in the liver and can lead to liver disease.

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 ...

Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) Provided by GlobeNewswire May 29, 2025, 8:01:00 PM ...

Korro Bio, Inc. announced an interim readout from its Phase 1/2a clinical trial of KRRO-110 for Alpha-1 Antitrypsin Deficiency (AATD) is expected in the second half of 2025, with trial completion ...