In September 2023, The US Food and Drug Administration has approved Krystal Biotech’s Investigational New Drug (IND) ...

Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder associated with liver disease, ranging from fibrosis to hepatocellular carcinoma. The disease remains asymptomatic until its final ...

Background: Influenza vaccination is recommended for all subjects with COPD, including α 1-antitrypsin deficiency (AATD), but immunization practices are below US national goals. Influenza ...

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 ...

This first-in-class RNA editing oligonucleotide is being developed for treating alpha-1 antitrypsin deficiency (AATD). WVE-006 is a GalNAc-conjugated, subcutaneously administered therapy that does ...

The new Inhibrx will also start with $200 million in funding from the French drugmaker. AATD is an inherited disorder whose primary consequence is life-threatening pulmonary emphysema, resulting ...

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.

A few months prior, Intellia had nixed an earlier program for alpha-1 antitrypsin deficiency, also known as AATD. At the time, Intellia said it intended to focus on enrolling pivotal studies of ...

Duchenne muscular dystrophy (DMD), Huntington’s disease (HD), and alpha-1 antitrypsin deficiency (AATD). The newly initiated Phase 1 INLIGHT clinical trial for WVE-007, targeting obesity ...