Patient organizations take on gene therapy’s redosing problem

Patient groups are taking matters into their own hands to address one of the largest challenges the gene therapy field is facing: the inability to redose AAV-based gene therapies. 

On Thursday, CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD) announced a collaboration to fund a clinical trial that will test marketed autoimmune disease drug Vyvgart efgartigimod alfa-fcab from argenx S.E. (Euronext:ARGX; NASDAQ:ARGX) as a way to reduce levels of anti-AAV antibodies enough to safely allow gene therapy dosing, or redosing. ...