June 10th 2025
The industry is diversifying pipelines from traditional small-molecule drugs to embrace complex and exciting new modalities.
Gene Therapy Holds Promise for Blood Disorders
January 11th 2019Researchers at the University of Delaware have made a step forward in gene therapy by engineering microparticles that deliver gene-regulating material to hematopoietic stem and progenitor cells, which live deep in bone marrow and direct the formation of blood cells.
Sarepta Therapeutics, Aldevron to Partner on Gene Therapy Development
January 4th 2019The partnership provides Sarepta with capacity and manufacturing slots for GMP-grade plasmid production for its micro-dystrophin Duchenne muscular dystrophy gene therapy program, as well as plasmid capacity for future gene therapy programs.