FDA Approves Kresladi Gene Therapy for Rare LAD-1 Disease
The FDA has approved Rocket Pharma's Kresladi for LAD-1, a rare immunodeficiency affecting roughly 25 US patients annually, marking a gene therapy milestone.
By Robert Kim
gene therapy
2 articles tagged "gene therapy"
In Vivo Base Editing for Familial Hypercholesterolemia: Early Evidence from a Phase 1 Trial
A phase 1 trial of YOLT-101 gene therapy demonstrated sustained reductions in PCSK9 and LDL cholesterol levels in patients with heterozygous familial hypercholesterolemia, though larger studies are required to establish clinical efficacy.