The global alpha-1 antitrypsin deficiency (AATD) market is expanding, driven by advances in diagnosis, treatment, and growing awareness. Key players like Grifols, CSL Behring, and Pfizer lead ...

CAMBRIDGE, Mass., May 29, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that ...

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 ...

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 ...

Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) Published May 29, 2025 4:01pm EDT ...

BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the PiZ mutation. ... About Alpha-1 Antitrypsin Deficiency (AATD) ...

Abnormalities in alpha-1 antitrypsin (AAT) proteins are risk factors for human disease. While the most common is AAT deficiency, a genetic disorder associated with chronic obstructive pulmonary ...

A gene-editing delivery system developed by UT Southwestern Medical Center researchers simultaneously targeted the liver and lungs of a preclinical model of a rare genetic disease known as alpha-1 ...

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