Sarepta’s Duchenne Muscular Dystrophy Drugs Fail Confirmatory Trial, Company Pursues Full FDA Approval Regardless

Sarepta Therapeutics; Duchenne muscular dystrophy; confirmatory trial; AMONDYS 45; VYONDYS 53; Elevidys; FDA approval; clinical trial failure; accelerated approval; statistical significance

UCB Wins First FDA Approval for Ultra-Rare Mitochondrial Disease: Kygevvi for Thymidine Kinase 2 Deficiency (TK2d)

UCB; FDA approval; Kygevvi; mitochondrial disease; Thymidine Kinase 2 deficiency; TK2d; doxecitine; doxribtimine; rare diseases; Orphan Drug; Breakthrough Therapy; Priority Review; Rare Pediatric Disease

TScan Cuts Workforce by 30% While Iovance Advances Amtagvi Program Amid Layoffs

TScan Therapeutics; layoffs; Iovance Biotherapeutics; Amtagvi; tumor-infiltrating lymphocyte (TIL) therapy; melanoma; lung cancer; strategic restructuring; biotech layoffs; cell therapy

Eli Lilly to Invest $3 Billion in New European Factory for Weight-Loss Pill Orforglipron

Eli Lilly; Orforglipron; obesity pill; Leiden Bio Science Park; Netherlands; GLP-1 receptor agonist; manufacturing facility; European market; advanced manufacturing; global supply chain

M42 and Oracle Join Forces to Advance Healthy Longevity and Disease Prevention

M42; Oracle Health; healthy longevity; disease prevention; precision medicine; Emirati Genome Program; pharmacogenomics; electronic health record (EHR); personalized healthcare; UAE