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 ...

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

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

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

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 ...

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 ...

For those born with certain types of congenital deafness, the cochlear implant has been a positive and enabling technology.

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 ...

"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 ...

Ace Therapeutics announces its preclinical ophthalmic drug development services to help expedite the discovery of ...