Sickle cell disease (SCD) is a common inherited blood disorder, affecting 70,000 to 80,000 Americans, with approximately 300,000 babies born each year worldwide with the disease.
Dr. Christopher Vakulskas is a senior staff scientist in the molecular genetics research group at Integrated DNA Technologies (IDT), and in a recent article in Drug Discovery, from Technology Networks, he explains how research using CRISPR gene editing can correct the mistake in the genes responsible for SCD.
Part of that development includes an improved enzyme for gene editing using CRISPR/Cas9. Aldevron is partnering with IDT to provide a GMP form of the enzyme for clinical trials, hopefully developing an SCD therapy based on ex vivo gene editing of HSCs for autologous transplants.
Dr Matthew Porteus, from Stanford Medicine, is leading a proposed Phase I SCD clinical trial to evaluate a gene editing therapy that corrects the actual disease-causing HBB mutation.
Dr. Christopher Vakulskas
Dr. Matthew Porteus