Gene Therapy Leads to Improved Quality of Life in Patients with Sickle Cell Disease and Beta Thalassemia
American Society of Hematology
Substantial improvements seen across ages in overall health and well-being
gene therapy
Gene Therapy Restored Hearing in Deaf Patients
Karolinska Institutet
New gene therapy well-tolerated and improves hearing in all 10 patients with congenital deafness or severe hearing impairment
Xcell Biosciences Announces Joint Research Collaboration with Thermo Fisher Scientific to Advance the Next Generation of Cell Therapies
Xcell Biosciences
Strategic collaboration to innovate Treg and TIL therapies, streamline cell therapy workflows, and enhance manufacturing efficiency
'The Critical Role of the Clinical Lab'
Miriam Bergeret, MSc
The Today's Clinical Lab 2025 Cancer & Precision Medicine print issue features cancer screening successes, emerging technologies, and advances in targeted treatments
Gene Therapies for Retinal Diseases Head to Clinical Trials
Jennifer Welsh
Inherited retinal diseases cause vision loss and blindness. New gene therapy trials have improved sight for some
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
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