alpha 1-Antitrypsin Deficiency
Sanofi’s efdoralprin alfa (INBRX-101) achieves all endpoints in Phase 2 alpha-1 antitrypsin deficiency (AATD) trial
Sanofi; efdoralprin alfa; INBRX-101; AATD; alpha-1 antitrypsin deficiency; Phase 2; Inhibrx; acquisition; rare disease; plasma-derived therapy; Zemaira
Wave RNA Editing Restores Enzyme in Genetic Disorder but Fails to Impress Investors
Wave Life Sciences; RNA editing; AATD; alpha-1 antitrypsin deficiency; enzyme restoration; clinical trial; investor reaction; WVE-006
Wave Life Sciences Shares Dip After Mixed Data Update on GSK-Partnered AATD Therapy
Wave Life Sciences; GSK; AATD; RNA editing; WVE-006; clinical trial; protein levels; stock dip; biotech
Korro Bio Lays Off 20% of Workforce to Extend Runway for Genetic Medicine Trials and Novo Nordisk Partnership
Korro Bio; layoffs; biotech; genetic medicine; RNA editing; KRRO-110; Novo Nordisk; clinical trial funding; restructuring; AATD
Revolutionizing AATD Treatment: Emerging Therapies to Replace Augmentation Therapy
Alpha-1 Antitrypsin Deficiency (AATD), Genetic Disorder, New Therapies, Augmentation Therapy, RNA Interference (RNAi), Gene Editing, CRISPR
Vertex Pharmaceuticals Abandons Two Phase I AATD Drug Candidates Due to Disappointing Data
Vertex Pharmaceuticals, AATD, alpha-1 antitrypsin deficiency, drug candidates, Phase I trials, disappointing data
Biotech Startup Airna Secures $60M to Advance RNA-Editing Therapy for Alpha-1 Antitrypsin Deficiency
Airna, RNA editing, alpha-1 antitrypsin deficiency, AATD, biotech startup, genetic medicine, ADAR, CRISPR, RNA therapy
Sanofi gains AATD hopeful with $1.7-billion deal for Inhibrx
sanofi, Inhibrx, Acquisition (action), alpha 1-Antitrypsin Deficiency, Market