FDA Approves Labcorp Cell-Based CDx for Hemophilia Gene Therapy, BEQVEZ
LabCorp
The companion diagnostic will help bring clarity to hemophilia B patients considering BEQVEZ as a treatment option
gene therapy
Organoids Help Uncover the Genetics of Kidney Disease
National Institutes of Health
Innovative disease modeling and gene editing techniques begin to answer long-standing questions
Personalizing Enzyme Therapy to Improve Pancreatitis in Kids
Ochsner Health
When on pancreatic enzyme replacement therapy, the mean acute pancreatitis annual incidence rate decreased from 3.14 to 0.71
Gene Therapy Improves Rare Neurodegenerative Disease in Trial
National Institutes of Health
Treatment for giant axonal neuropathy was well tolerated and slowed loss of motor function
FDA Approves First Gene Therapy for Metachromatic Leukodystrophy
U.S. Food and Drug Administration
The FDA granted approval of Lenmeldy™ to Orchard Therapeutics
FDA Approves First Gene Therapies for Sickle Cell Disease
U.S. Food and Drug Administration
The FDA granted the approval of Casgevy to Vertex Pharmaceuticals Inc., and Lyfgenia to Bluebird Bio Inc.
First Two Children Receive New Gene Therapy for Rett Syndrome
Neurogene Inc.
Phase 1/2 trial showed the safety and therapeutic potential of administering a novel transgene
Gene Therapy to Treat Rare and Hereditary Diseases
Norwegian University of Science And Technology
Researchers are finding clues that may help treat rare and deadly diseases using mini-organs and advanced technology
Gene Therapy for Glioblastoma Shows Promise in Phase 1 Trial
Brigham and Women's Hospital
In a first-in-human trial in patients with recurrent glioblastoma, an oncolytic virus treatment safely extended survival
Cell and Gene Therapies Demand a Robust System
Miltenyi Biotec
Standardizing your CGT platform to ensure consistency during experiments is essential for improving future clinical outcomes