we have uncovered early molecular triggers of how alpha-1 antitrypsin deficiency progresses. This points toward actionable targets that could lead to improved therapies for patients.” Pavel Strnad, a ...

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 ...

The two most common inherited liver diseases are hemochromatosis and alpha-1 antitrypsin deficiency. Hemochromatosis is a disease in which deposits of iron collect in the liver and other organs.

According to recent projections by Future Market Insights (FMI), the global alpha-1 antitrypsin deficiency market is poised for significant growth, reaching an estimated USD 10 Billion by 2033, up ...

today 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 7th Global Research Conference and ...

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 ...

WASHINGTON (dpa-AFX) - 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 ...

today unveiled a preclinical program for the treatment of alpha-1 antitrypsin deficiency (AATD), the next program within its liver franchise. Prime Medicine expects to file an investigational new ...

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 ...