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
gene therapy
FDA Approves First Gene Therapy for Adults with Severe Hemophilia A
U.S. Food and Drug Administration
Roctavian recipients should be closely monitored for infusion-related reactions and liver enzyme elevation
FDA Approves First Gene Therapy Treatment for Duchenne Muscular Dystrophy
U.S. Food and Drug Administration
About one in every 3,300 boys is affected by DMD and the current treatments don’t address the genetic cause
FDA Approves First Topical Gene Therapy for Dystrophic Epidermolysis Bullosa Wounds
U.S. Food and Drug Administration
Around 65 percent of the wounds treated with Vyjuvek showed complete wound closure in DEB patients
Structure and Function of Newly Discovered CRISPR Immune System
Utah State University
In tandem papers in Nature, researchers report effects of Cas12a2
Tips for Transitioning Cell & Gene Therapies to the Clinic
Dominic Clarke, PhD
How to circumvent common barriers to bringing cell and gene therapies to the clinic
FDA Approves First Gene Therapy to Treat Adults with Hemophilia B
U.S. Food and Drug Administration
The one-time gene therapy, Hemgenix, was evaluated in two studies and was found to decrease annualized bleeding rate
Removing Roadblocks in Cell and Gene Therapy Quality Control
Marwan Alsarraj, MS
Droplet digital PCR (ddPCR) technology provides unmatched quality control for cell and gene therapies
FDA-Approved Gene Therapy for Transfusion-Dependent Beta-Thalassemia Offers Potential Cure
María Carla Rosales Gerpe, MSc, PhD
Patients with transfusion-dependent beta-thal will soon be treated with lentiviral vector gene therapy Zynteglo to restore hemoglobin levels
A Look Ahead: Clinical Diagnostics and Cell & Gene Therapies
Marwan Alsarraj, MS
The pandemic has fueled new market growth and will continue to shape the future of diagnostics