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

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 ...

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 ...

Neurodegenerative diseases (NDDs) represent a profound challenge to global health, characterized by progressive neuronal dysfunction and loss, leading to ...

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 ...

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 ...

"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 ...

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

The Gene Therapy R&D market presents robust opportunities, driven by clinical advancements in CAR-T therapies and a strong pipeline, particularly for cancer and rare diseases. Despite challenges from ...

An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...