First-in-the-world gene therapy delivers missing gene directly to infant’s brain, marking historic milestone in precision medicine
Jun 11 2026 An eight-month-old baby from Israel has become the first human ever to receive an experimental…
Renowned physician‑scientist to lead Houston Methodist’s cell and gene therapy research
Malcolm Brenner, an internationally recognized physician‑scientist renowned for his leadership in cell and gene therapy, has been tapped to lead…
Gene therapies have high price tags. A financing model could help
A new class of medicines is transforming health care. Gene therapies can now cure diseases like sickle cell…
Regeneron (REGN) Says EMA Accepted Review for Otarmeni Application
Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) is one of the 10 Best Debt-Free S&P 500 Stocks to Buy Now. On…
First precise genome editing of human embryos triggers praise and alarm
A four-celled human embryo. Credit: Juan Gaertner/Science Photo Library Researchers say they have used a precise genome-editing technique…
Gene therapies to fix failing hearts gain steam after years in the doldrums
Heart-muscle cells (in close-up, artificially coloured) do not normally proliferate in adult mammals, but scientists are trying to…
Breakthrough Parkinson’s Drug Targets Disease at Its Genetic Roots
Scientists have reported promising early results from a novel Parkinson’s treatment designed to suppress the activity of a…
New gene therapy improves hearing in patients with rare genetic deafness
A new international study co-led by investigators from Mass General Brigham and the Eye & ENT Hospital of…
A mother’s 20-year quest fuels a startling Duchenne breakthrough
A year after the worst day of her life, Debra Miller received a voicemail she couldn’t quite make…
‘Deafness reversed’: Scientists restore hearing in 10 patients with a single-injection gene therapy breakthrough |
A clinical study has delivered one of the clearest signs yet that gene therapy can restore hearing in…
The Medicine Maker | mRNA Expands into Fertility Medicine
Credit: Mito Kanatsu-Shinohara
Researchers in Japan have demonstrated that messenger RNA (mRNA) therapy…
Genetic mismatch identified as major risk factor in cord blood transplantation
A Japanese study of more than 7,400 patients has identified a genetic mismatch that sharply increases the risk…