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 ...
The drug’s success in a Phase 2 study in alpha-1 antitrypsin deficiency “validates” Sanofi’s 2024 acquisition of Inhibrx, ...
Zacks Investment Research on MSN
SNY's Efdoralprin Alfa Meets Key Endpoints in Rare Disease Study
Sanofi SNY announced that the phase II ElevAATe study, which evaluated its pipeline candidate, efdoralprin alfa, for the ...
Sanofi’s efdoralprin alfa met all primary and key secondary endpoints in alpha-1 antitrypsin deficiency emphysema phase 2 studyEfdoralprin alfa ...
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 ...
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 ...
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 ...
(RTTNews) - Beam Therapeutics Inc. (BEAM) presented additional data from the Phase 1/2 clinical trial of BEAM-302 in patients with alpha-1 antitrypsin deficiency (AATD) at the 2025 Alpha-1 Foundation ...
Wave Life Sciences Ltd. (NASDAQ:WVE) stock is trading lower on Wednesday after the company shared data from the ongoing Phase 1b/2a RestorAATion-2 Trial of WVE-006 in alpha-1 antitrypsin deficiency.
Alpha-1 antitrypsin (AAT) deficiency is a genetic disease where your body doesn’t make enough normal functioning AAT. AAT is a protein your liver makes. AAT has many important roles in your body, ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback