Tag: gene therapy
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…
Africa Needs Infrastructure Investment for Next-Generation Sickle Cell Therapies, BMJ Study Shows
BMJ Global Health analysis reveals African healthcare systems need major infrastructure investment to deliver next-generation gene therapies for sickle cell…
Small-Molecule Drugs Enable Safer, More Precise Therapeutic Genome Editing
New research shows small-molecule drugs can precisely control therapeutic genome editing, achieving 85% improved accuracy while reducing off-target effects by…