uniQure Provides FDA Update on AMT-130; BLA Timing Unclear
uniQure (NASDAQ: QURE) on Nov. 3, 2025 announced FDA feedback…
New gene-editing method can correct many disease-causing mutations in mammalian cells
Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person’s genome, often…
fixing them is about to get a lot easier
CRISPR gene editing has made its way into every corner of modern biology, but not into every corner…
U.S. Cell And Gene Therapy Clinical Trials Market Trends
Dublin, Oct. 03, 2025 (GLOBE NEWSWIRE) — The “U.S. Cell And Gene Therapy Clinical Trials Market Size, Share…
U.S. Cell And Gene Therapy Clinical Trials Market Trends
Dublin, Oct. 03, 2025 (GLOBE NEWSWIRE) — The “U.S. Cell And Gene Therapy Clinical Trials Market Size, Share…
A clinical milestone for CRISPR in sickle-cell disease
The haemoglobinopathies transfusion-dependent β-thalassemia (TDT) and sickle-cell disease (SCD) are two globally prevalent and severe monogenic blood disorders.…
Next-generation gene therapy tools built by UH scientist
Reading time: 2 minutesAssistant Professor Jesse Owens using a program to model the structure of his integrase protein.…
Huntington’s disease treated for first time using gene therapy
A brain scan of a person with Huntington’s disease, which causes a loss of brain volume as neurons…
New gene therapy for Huntington’s ‘changes everything’, say researchers
UK researchers say they have slowed the progression of the fatal neural condition Huntington’s disease for the first…
U.S. Cell & Gene Therapy Contract Research Organizations
Dublin, Sept. 18, 2025 (GLOBE NEWSWIRE) — The “U.S. Cell & Gene Therapy Contract Research Organizations Market Size,…
The Medicine Maker | Combatting Deafness with Gene Therapy
A study from researchers at Tel Aviv University reports that restoring a single protein…
Diversity challenges and reconciles genetics in facioscapulohumeral muscular dystrophy
Wang LH, Tawil R. Facioscapulohumeral dystrophy. Curr Neurol Neurosci Rep. 2016;16:66. Article PubMed Google Scholar Mul K, Lassche…