Prime editing reverses symptoms of severe childhood neurological disease
Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder that begins in infancy....
FDA Grants Regenerative Medicine Advanced Therapy Designation to Gene Therapy for Osteoarthritis
The FDA awarded Regenerative Medicine Advanced Therapy (RMAT) designation to GNSC-001, a potential gene therapy from Genascence that blocks interleukin 1 (IL-1) for the treatment of knee osteoarthritis (OA). The product, which received FDA...
StockWatch: Sarepta Shares Nosedive after LGMD Gene Therapy Patient Dies
The death of a third patient tied to a Sarepta Therapeutics (NASDAQ: SRPT) gene therapy this year sent the company’s shares nosediving 36% Friday, halting the momentum the stock generated just a day earlier...
Researchers honored for groundbreaking work in cancer neuroscience
The University of Virginia School of Medicine's Harald Sontheimer, PhD, and Stanford's Michelle Monje, MD, PhD, have received the 2025 International Prize in Translational Neuroscience from the Gertrud Reemtsma Foundation for their pioneering research...
Thermo Fisher Agrees to Acquire Sanofi’s Steriles Manufacturing Site
Thermo Fisher Scientific signed an agreement to acquire Sanofi’s steriles manufacturing site in Ridgefield, NJ, and will continue to manufacture a portfolio of therapies for Sanofi. In addition, Thermo Fisher will expand the use...
Targeting GLUD1 shows promise in restoring muscle function in Duchenne muscular dystrophy
New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated that inhibiting GLUD1 significantly enhances muscle strength...
Research suggests a new strategy to improve blood flow to the brain to battle...
New University of Virginia School of Medicine research suggests an unexpected way doctors may be able to improve blood flow to the brain to battle Alzheimer's and other neurodegenerative diseases. Scientists led by Ukpong...
Gut Microbiota Metabolite Could Point to Atherosclerosis Diagnostics, Therapeutics
Cardiovascular disease is the leading cause of death worldwide and often originates in atherosclerosis, a chronic condition in which inflammation and fat deposits cause arteries to harden and narrow. Although clinical practice already targets...
Optogenetic platform enables precise discovery of anti-aging therapeutics
A landmark study published in Cell has unveiled a first-of-a-kind optogenetic screening platform, developed by scientists at Integrated Biosciences, a biotechnology company integrating optogenetics, chemistry and AI to discover small molecule therapeutics for age-related...
Qureight analyze of Insilico Medicine’s Phase IIa rentosertib data support preliminary efficacy results and...
Qureight, a techbio company accelerating drug development using its proprietary deep-learning image biomarkers and clinical data analytics platform, today announced the first results from a collaboration with Insilico Medicine ("Insilico"), a clinical-stage biotechnology company...
