BMN 349 treatment reduced pro-necro -inflammatory processes in the liver and improved overall biosynthetic liver function, suggesting BMN 349 may slow or prevent AATD liver disease …

2024年9月4日 · BMN 349 for the treatment of Alpha-1 Antitrypsin Deficiency with a unique mechanism of action that has the potential to transform liver health and with target POC in 2026

2024年11月18日 · BMN 349, an investigational treatment for AATD, is a small molecule chaperone that binds to Z -AAT, preventing polymerization and increasing monomer AAT …

2023年9月12日 · Meanwhile, BioMarin has two INDs expected by the end of the year for its existing pipeline, including BMN 349 for alpha-1 antitrypsin deficiency. The uncommon …

2024年4月25日 · First up on the discard pile is BMN 331, a gene therapy being assessed in a phase 1/2 trial for patients with hereditary angioedema, a rare inherited swelling condition. The …

2024年12月31日 · A new study in the United States is recruiting patients to evaluate the safety and efficacy of a single dose of BMN 349, an oral gene therapy for the treatment of patients …

2024年9月5日 · Our pipeline includes investigational medicines across four areas of focus: central nervous system, cardiovascular, musculoskeletal/metabolic and non-oncology hematology. …

2025年2月27日 · The goal of this clinical trial is to assess the safety and tolerability of a single oral dose of BMN 349 in participants with PiZZ or PiMZ/MASH.

2024年8月6日 · A first-in-human study of the oral small molecule BMN 349, for which BioMarin has ambitions to become a best-in-class treatment for Alpha-1 antitrypsin deficiency (AATD) …

BMN 349, A SMALL MOLECULE FOR ALPHA-1 ANTITRYPSIN ASSOCIATED LIVER DISEASE, STABILIZES HUMAN Z-AAT AND REDUCES SEVERITY OF LIVER DISEASE IN THE PIZ …