Tag: gene therapy
Gene Therapy Restores Muscle Function in Nemaline Myopathy Type 6: New Hope for Rare Genetic Disorder
A new study published in Science Translational Medicine demonstrates that reducing Kbtbd13 gene expression restores muscle function in nemaline myopathy…
Roche Discontinues Huntington’s Gene-Silencing Program Amid Competitive Pressures
Roche has discontinued its Huntington's disease gene-silencing program, highlighting competitive pressures in neurodegenerative therapeutics. The decision comes as ARPA-H launches…
Harvard researchers develop electric silicon chip for rapid DNA synthesis—a breakthrough toward portable genetic engineering
Harvard researchers have engineered a silicon microchip that synthesizes dozens of DNA sequences simultaneously using electricity and water-based enzymes, replacing…
A Father’s Fight: How One Family’s Quest Is Reshaping Rare Disease Drug Development
A father's advocacy for his daughter with ultra-rare NGLY1 deficiency is reshaping how experimental gene therapies reach patients when traditional…
Gene Therapy Shows Promise for Inherited Cholesterol Disorder in First-in-Human Trial
First-in-human gene therapy trial shows preliminary safety and efficacy for treating homozygous familial hypercholesterolemia, a rare inherited cholesterol disorder. Three…
First Dual-Target Gene Therapy for Parkinson’s Disease Shows Promise in Early Trial
First-in-human trial of BBM-P002 dual-target gene therapy shows sustained 12-month motor improvements in Parkinson's disease patients with excellent safety profile.…
Gene Therapy Financing Crisis: $2 Million Cures Lack Payment Infrastructure
Gene therapies costing up to $2 million per treatment face critical financing barriers despite proven therapeutic benefits. Healthcare economist calls…
CRISPR Gene Therapy Shows Promise for Children with Sickle Cell Disease and Beta-Thalassemia
CRISPR gene therapy exa-cel achieved 95% efficacy in eliminating blood transfusions for children with beta-thalassemia and sickle cell disease. New…