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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with rare, severe, and debilitating neuromuscular diseases by applying its world-leading platform in myostatin biology, today announced that the FDA review of the apitegromab Biologics License Application (BLA) for the treatment of children and adults with spinal muscular atrophy (SMA) is progressing with the Company’s second fill-finish facility. The B...

HOUSTON--(BUSINESS WIRE)--IPS HEART, Inc., a biopharmaceutical company pioneering induced pluripotent stem cell (iPSC)-derived cell replacement therapies, announced that the FDA has granted Rare Pediatric Drug Designation for ISX9-CPC, for the treatment of cardiomyopathies associated with dystrophinopathies. Redefining the Therapeutic Paradigm: Generating New Human Muscle Duchenne muscular dystrophy (DMD) leads to progressive skeletal muscle loss, severe cardiac fibrosis, and premature death fr...

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today reported financial results for the second quarter ended June 30, 2026, and provided a business update. “The second quarter marked a defining moment for Precision BioSciences and the hepatitis B field, with the first clinical biopsy evidence that a therapeutic...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Amylyx Pharmaceuticals Reports Second Quarter 2026 Financial Results...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology, today reported financial results for the second quarter ended June 30, 2026, and provided an update on recent company developments. “We are on the threshold of securing the world’s first ever regulatory approval of a myostatin inhibitor, whi...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vedanta Biosciences, a late clinical-stage biopharmaceutical company developing microbiome-based oral therapies for gastrointestinal diseases, today announced securing $60 million in funding, a clinical update for its pivotal Phase 3 RESTORATiVE303 study of VE303 for the prevention of recurrent C. difficile infection, and new appointments to its Board of Directors. The funding includes a $40 million financing round led by existing investors AMR Action Fund and...

RAHWAY, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside of the United States and Canada, announced today that the U.S. Food and Drug Administration (FDA) has accepted a supplemental Biologics License Application (sBLA) for ENFLONSIA™ (clesrovimab-cfor) to expand its indication to include prevention of respiratory syncytial virus (RSV) lower respiratory tract disease (LRTD) in children under two years of age at increased risk for severe RSV disease through their second RSV season....

OSAKA, Japan & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Takeda (TSE:4502/NYSE:TAK) announced that the U.S. Food and Drug Administration (FDA) approved ORZEYFUL™ (oveporexton), an oral orexin receptor 2 (OX2R) agonist, for the treatment of narcolepsy type 1 (NT1, narcolepsy with cataplexy) in adults.* The persistent 24-hour nature of NT1 is driven by orexin deficiency and can severely impact people’s lives. As a first-in-class orexin treatment, ORZEYFUL is the only medicine indicated in the U.S. to tr...

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today reported financial results and operational highlights for the second quarter ended June 30, 2026. “At Adicet, we continue to advance prula-cel toward our next important clinical milestone, with a Phase 1 clinical update now expected in the third quarter of 2026. The additional f...

ALAMEDA, Calif.--(BUSINESS WIRE)--Exelixis, Inc. (Nasdaq: EXEL) today reported financial results for the second quarter of 2026, provided an update on progress toward achieving key corporate objectives, and outlined its commercial, clinical and pipeline development milestones. “Exelixis continues to execute across the key pillars of our business, positioning the company to deliver on our strategic objectives for 2026 and beyond,” said Michael M. Morrissey, Ph.D., President and Chief Executive O...