Why are patients with mitochondrial disease more susceptible to infections? The cell at the top right is dying through caspase-11 dependent pyroptosis, the mechanism West and colleagues identified in this paper. It's mitochondria are gone and the nucleus also condensed. ©The Jackson Laboratory Patients with rare mitochondrial diseases already face a…..
GNT0004 product confirms its clinical efficacy with stabilization of motor functions in patients treated at the effective dose for up to 2 years Genethon has unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting of the American Society…..
Launch of UMBRELLA study to personnalise post-treatment follow-up for cancer patients On May 19, 2025, Gustave Roussy announced the launch of UMBRELLA, a ground-breaking phase III clinical trial that could profoundly transform how cancer patients are monitored after curative treatment. The objective is to personalise post-treatment follow-up based on the…..
8.3 million euros for doctoral programme in regenerative medicine and ATMP Marie Jönsson, grant manager Stem Cell Center and Mattias Magnusson, director of RAMP-UP at the Lund Research School in Stem Cell Biology and group leader at the Stem Cell Center. ©Alexis Bento Luis Lund University in Sweden has been…..
National Initiative for RNA Biology and its Applications launched in Singapore RNA biology and its applications has been identified as a rapidly expanding field of critical importance to Singapore’s future health and economic needs. Singapore Deputy Prime Minister Mr Heng Swee Keat officiated at the inauguration of the National Initiative…..
Study suggests new molecular strategy for treating fragile X syndrome Building on more than two decades of research, a study by MIT neuroscientists at The Picower Institute for Learning and Memory reports a new way to treat pathology and symptoms of fragile X syndrome, the most common genetically-caused autism spectrum…..
Novel gene therapy trial for sickle cell disease launches in the US The study uses CRISPR to correct sickle cell mutation and aims to free patients of the disease. UCSF Benioff Children's Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The…..
Preclinical data for AAV-FGF21 gene therapy shows durable reversal of liver fibrosis UAB researchers have cured liver disease MASH in mouse models by a one-time gene therapy . The results were obtained with a single intramuscular administration of the therapeutic viral vectors AAV-FGF21. The research has also determined that most…..
Merck KGaA to triple its ADC manufacturing capacity in St. Louis The German science and technology group Merck KGaA has announced a € 70 million expansion of its antibody-drug conjugates (ADC) manufacturing capabilities and capacity at its Bioconjugation Center of Excellence facility in St. Louis, Missouri, USA. This investment…..
An epigenome editing toolkit to dissect the mechanisms of gene regulation A study from the Hackett group at EMBL Rome led to the development of a powerful epigenetic editing technology, which unlocks the ability to precisely program chromatin modifications. Creative depiction of the epigenetic editing toolkit: each building represents the…..
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