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…
New FDA cell and gene therapy oversight system comes with risks
At a Food and Drug Administration cell and gene therapy (CGT) roundtable last June, most speakers pushed for…
Novel immunotherapy targeting pancreatic cancer shows promising results in phase 1/2 trial
A recent publication in Nature Medicine describes a novel immunotherapy targeting pancreatic cancer that has shown promising results in a…
Eyes of the world’s longest-living vertebrate, the Greenland shark, show little ageing
Swimming at a crawl with cloudy eyes and mottled skin, the Greenland shark looks like it’s seen better…
AskBio Announces FDA Acceptance of Investigational New Drug (IND) Application for AB-1009 Gene Therapy for Treatment of Late-Onset Pompe Disease (LOPD)
Durham, N.C., Jan. 08, 2026 (GLOBE NEWSWIRE) — AB-1009 program advances to Phase 1/Phase 2; clinical trial initiated…
7 medical breakthroughs of 2025
For most diseases, the challenge is not a lack of effort but the limits of existing tools and…
the events to watch for in the coming year
India’s Aditya-L1 spacecraft launched in 2023. Next year, it will observe the Sun during its peak activity phase.Credit:…
Challenges and opportunities in delivering gene therapies for sickle cell disease and beta thalassemia
The first study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia…
Novo Nordisk backs biotech start-up targeting aging
General Control enters Novo Nordisk’s Co-Creation Greenhouse to advance multiplex epigenetic editing for complex chronic disease. General Control,…