REGENXBIO has announced that the Phase I/II AFFINITY DUCHENNE™ trial of RGX-202, an investigational gene therapy product for the potential treatment of Duchenne, is now active and recruiting trial participants. RGX-202 utilizes a novel adeno-associated virus (AAV8) to transport a shortened version of the dystrophin gene (micro-dystrophin) that may provide benefit in place of the missing full-length dystrophin protein.
Recruitment for AFFINITY DUCHENNE is targeting patients aged 4-11 years old with Duchenne. Like many other gene therapy clinical trials there is a mutation restriction for AFFITINTY DUCHENNE, currently they are excluding patients with mutations in exons 1-17. Patients will need to be on a steady dose of glucocorticoids prior to screening for at least 12 weeks. Patients who have previously received either an investigational or approved gene therapy product are excluded from this study. Lastly, patients cannot have pre-existing antibodies to AAV8 the vector they are using to delivery their micro-dystrophin product.
REGENXBIO also shared a non-interventional observation study AFFINITY BEYOND. This means that no investigational product is being delivered in this study, the company is just trying to gather data to better understand the disease and inform future research. For this study, REGENXBIO will be conduct phone/video interviews and require bloodwork in order to better understand the prevalence of pre-existing antibodies to AAV8 and AAV9 in the Duchenne population. Only patients aged 0-11 years old with a diagnosis of Duchenne who have never received a gene therapy product are eligible.
Since launching our Gene Therapy Initiative in 2017, PPMD has been optimistic that gene therapy may be a potential treatment for Duchenne and we will continue to provide updates on the latest information and ensure that our community’s questions and concerns are shared. For more information about current gene therapy trials in Duchenne and results data so far, click here.