Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...

Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...

The cell and gene therapy sector has never lacked attention, but all too often, the narrative swings from one extreme to ...

Alzheimer’s may be driven far more by genetics than previously thought, with one gene playing an outsized role. Researchers ...

Live Science's health channel editor makes predictions about the medical breakthroughs and public health shifts to come in 2026. When you purchase through links on our site, we may earn an affiliate ...

NEW ORLEANS, LA—An investigational CRISPR-Cas9 gene-editing therapy that targets angiopoietin-like protein 3 (ANGPTL3), which has a role in regulating lipid metabolism, appears to safely lower levels ...

"Regulatory flexibility must be tailored for cell and gene therapies," said FDA Commissioner Marty Makary, M.D., M.P.H. "These are common-sense reforms that will address the unique characteristics of ...

Collaboration Aims to Establish Sustainable Access to a Newly Approved Therapy Targeting a Rare Immunodeficiency, Filling Urgent Gap Left by For-Profit Efforts CAMBRIDGE, Mass. and ROME, Dec. 15, 2025 ...

The new method is designed to focus specifically on pain-related signals, without interfering with normal activity in other parts of the brain. A new preclinical study has identified a gene therapy ap ...

Astellas Gene Therapies remains intent on treating X-linked Myotubular Myopathy (XLMTM) through a gene therapy—but instead of AAV-based resamirigene bilparvovec (AT132), Astellas is now partnering ...

A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...