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 ...

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Targeted gene repair is a technique used to correct a mutation at a specific site in an episome or chromosome. ... the primary organs affected in alpha-1 antitrypsin deficiency.

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 ...

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) Beam Therapeutics - GlobeNewswire - Thu May 29, 3:01PM CDT ...

About Alpha-1 Antitrypsin Deficiency (AATD)AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease.

BACKGROUND--Previous estimates of the survival times of patients with alpha 1-antitrypsin deficiency have been based on selected patients. METHODS--The survival times of 397 patients with severe alpha ...

The ZZ genotype of alpha-1 antitrypsin deficiency (AATD) is associated with COPD regardless of smoking. Heterozygous MZ-AATD is recognised as a moderate deficiency state, increasing the risk of COPD ...