On track to file IND and/or CTA for Wilson Disease and AATD programs in 1H 2026 and mid-2026, respectively; initial clinical data for both ...

Shares of Vertex Pharmaceuticals (VRTX) were lower after the drugmaker said that it would not pursue VX-864, a drug to treat alpha 1 antitrypsin deficiency, into late-stage development. At last check ...

TSRA-196 is designed to precisely correct the genetic mutation underlying AATD, with the goal of restoring production of functional alpha-1 antitrypsin (AAT) protein through a one-time, durable ...

Bernstein has upgraded Beam Therapeutics (NASDAQ:BEAM) to outperform from market perform, citing upcoming data on the company’s drug candidate for alpha-1 antitrypsin deficiency, or AATD. The ...

Wave, marked by positive clinical data sets in obesity and AATD that further demonstrated the broad potential of our unique ...

Recent studies attempted to improve the risk of recurrence while reducing the risk of adverse effect Alvelestat is an investigational oral neutrophil elastase inhibitor designed to protect AATD ...

A particular protein deficiency that leads to liver and lung damage is currently treatable only with decades-old therapies that all have limitations. Biotech startup AIRNA is part of a field of ...

A Beam Therapeutics gene-editing therapy designed to fix a mutation at the root of a rare liver protein deficiency now has early signs of efficacy along with safety data that ease some concerns about ...

CARLSBAD, Calif., Oct. 11, 2011 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has designated the second development candidate, ISIS-AATRx, in its collaboration with ...

(Boston)—Andrew A. Wilson, MD, associate professor of medicine at Boston University Chobanian & Avedisian School of Medicine, recently received a gift of $100,000 to establish the Katharine Howe ...