Studying a monogenic condition like Alpha-1 antitrypsin deficiency provides a unique opportunity to better understand disease ...

Studying a monogenic condition like Alpha-1 Antitrypsin Deficiency provides a unique opportunity to better understand disease progression. This work deepens our insight into protein folding disorders ...

AIRNA Raises Oversubscribed $155 Million Series B Financing to Fund Phase 1/2 Clinical Trial for Alpha-1 Antitrypsin Deficiency and ... drive health through human genetics, and is developing ...

Korro Bio, Inc. announced that its investigational drug KRRO-110 has received orphan drug designation from the FDA for treating Alpha-1 Antitrypsin Deficiency ... March 14, 2025 (GLOBE NEWSWIRE ...

BEAM-302 demonstrated safety and efficacy in increasing AAT levels and reducing mutant protein in alpha-1 antitrypsin deficiency patients. Beam Therapeutics announced promising initial results ...

Shares in Wave Life Sciences rose by 74% after it reported clinical data with a pioneering new RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD). Wave claims this is the first clinical ...

and others also participated AIRNA expected to file clinical trial application for Alpha-1 antitrypsin deficiency (AATD) RNA editing product candidate (AIR-001) with potential best-in-class ...