Vertex’s Gene Therapy Casgevy Shows Strong Efficacy in Younger Children with Blood Disorders
Boston, December 6, 2025Vertex Pharmaceuticals announced new clinical data demonstrating that its gene-editing therapy Casgevy (exagamglogene autotemcel, exa-cel) produced robust therapeutic benefits ...
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FDA Approves First Gene Therapy for Rare Immune Disorder Wiskott-Aldrich Syndrome, Expanding Treatment to Patients Aged 6 Months and Older
Washington, Dec. 9, 2025 — The U.S. Food and Drug Administration (FDA) has approved Waskyra (etuvetidigene autotemcel), a gene therapy developed by nonprofit organization Fondazione Telethon ETS, for...
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FDA Approves BioCryst’s Oral Pellet Form of Orladeyo, First Oral Prophylactic Therapy for Children Aged 2–11 with HAE
Durham, NC — Dec. 12, 2025 — The U.S. Food and Drug Administration (FDA) has approved an oral pellet formulation of Orladeyo (berotralstat) from BioCryst Pharmaceuticals for prophylactic use in chil...
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Sanofi Signs Up to $1.04 Billion Alzheimer’s Drug Development Deal with South Korea’s ADEL
Paris / Seoul — Dec. 15, 2025 — French pharmaceutical company Sanofi has entered into a strategic collaboration with South Korean biotech firm ADEL worth up to USD 1.04 billion to jointly develop an...
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UAE Becomes Second Country to Approve Gene Therapy Itvisma for Spinal Muscular Atrophy
The United Arab Emirates has become the second country in the world to approve the gene therapy Itvisma for the treatment of spinal muscular atrophy (SMA), marking another milestone in the global adop...
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FDA Approves Omeros’ Yartemlea as First Treatment for Transplant-Associated Thrombotic Microangiopathy
Washington, Dec. 24, 2025 — The U.S. Food and Drug Administration has approved Yartemlea, a monoclonal antibody developed by Omeros Corporation, as the first treatment for transplant-associated throm...
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