The recent conference, `Alpha 1-antitrypsin deficiency and other conformational diseases', held in Airlie, Virginia, USA (27–30 June, 2000) focused on some of the common pathways by which cells ...

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 ...

which is being evaluated in a phase 1/2 study for the treatment of patients with Alpha-1 Antitrypsin deficiency [AATD]. Initial results from this early-stage study are expected to be released in 2025.

AVG-002 is being developed by AlveoGene using its InGenuiTy® platform, which employs a unique pseudotyped lentiviral vector to deliver a functional SP-B gene directly to the neonaal deep lung alveolar ...

KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD) lung disease In September, after initiating dosing in Cohort 2, the Company amended the protocol for its KB408 Phase 1 SERPENTINE-1 ...

The company has also begun Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE) this year. Meanwhile, NTLA-3001, which targets alpha-1 antitrypsin deficiency, is scheduled to ...