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,…
Cutting-edge treatments in amyotrophic lateral sclerosis: the role of molecular pathogenesis in targeted therapies | Stem Cell Research & Therapy
Feldman EL, Goutman SA, Petri S, Mazzini L, Savelieff MG, Shaw PJ, et al. Amyotrophic lateral sclerosis. Lancet.…
Harmful cholesterol levels cut in half with one-time gene editing drug in early trial
Altering a single gene may help people lower dangerously high levels of cholesterol and other fats in the…
TAU researchers develop groundbreaking gene therapy to treat hearing, balance disabilities
“These findings highlight the potential of self-complementary AAVs to reduce dose requirements, minimize toxicity, and broaden clinical use…
New method could improve manufacturing of gene-therapy drugs | MIT News
Some of the most expensive drugs currently in use are gene therapies to treat specific diseases, and their…