FDA Rejects Capricor’s Cell Therapy for Duchenne Muscular Dystrophy

FDA rejection; Capricor Therapeutics; deramiocel; Duchenne muscular dystrophy; DMD; cell therapy; cardiomyopathy; clinical data; Phase 3 HOPE-3 trial; Complete Response Letter

FDA Investigates Patient Deaths Linked to Sarepta’s Elevidys Gene Therapy for Duchenne Muscular Dystrophy

FDA; Sarepta; Elevidys; Duchenne muscular dystrophy; gene therapy; patient deaths; acute liver failure; liver toxicity; regulatory investigation; non-ambulatory patients

Nuvalent Reports Positive Pivotal Data for ROS1-Targeted Lung Cancer Drug; Rolling FDA Submission Planned

Nuvalent; ROS1-positive lung cancer; zidesamtinib; FDA submission; ARROS-1 trial; NSCLC; tyrosine kinase inhibitor; objective response rate; brain metastases; oncology

Compass Pathways Achieves Phase 3 Win for Psilocybin in Depression, Yet Stock Tumbles

Compass Pathways; COMP360; psilocybin; Phase 3 trial; depression; treatment-resistant depression; clinical trial; stock decline; investor reaction; FDA