GlobalData on MSN

Beacon of hope for XLRP patients on Phase III gene therapy success

Beacon states this is the first pivotal XLRP trial to meet its primary endpoint.
Hosted on MSN

CRISPR roadblocks: Scientists identify genes blocking gene therapy success

Like a delivery driver navigating crowded city streets, a gene-therapy-toting lipid nanoparticle faces a gauntlet of potential detours on its journey toward a cell's nucleus. First, there's entering ...
CNN

FDA approves first gene therapy for inherited deafness, shown to restore hearing for children with rare condition

The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time treatment that proved to be life-changing for a small number of children in a ...
markets.financialcontent

Gene Therapy Remains Top-of-Mind in Hemophilia Despite Recent Market Contraction, as Physicians and Patients Raise the Bar for Success

Gene Therapy Remains Top-of-Mind in Hemophilia Despite Recent Market Contraction, as Physicians and Patients Raise the Bar for Success ...