TOKYO, Japan — Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) announced today that it launched ELEVIDYS® Intravenous Infusion [generic name: delandistrogene moxeparvovec] (hereinafter, “ELEVIDYS”), which received conditional and time-limited approval on May 13, 2025, following its listing on the national health insurance (NHI) reimbursement price list today, as a regenerative medical product for...

LOS ANGELES, Calif. – Researchers at UCLA have developed a nonviral gene-editing strategy that could help pave the way for one-time treatments for people with cystic fibrosis (CF). The experimental therapy uses lipid nanoparticles — tiny fatty molecules — to insert a full, healthy copy of the CFTR gene into human airway cells,...

Boston, MA – PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value, today announced that the U.S. Food and Drug Administration (FDA) and European Commission have granted Orphan Drug Designation to deupirfenidone (LYT-100) for the treatment of idiopathic pulmonary fibrosis...