Advanced Therapies Week 2022 | OTL-103: An autologous gene therapy for Wiskott Aldrich syndrome

Kent Christopherson, PhD, Orchard Therapeutics, Boston, MA, provides an overview of OTL-103, an investigational autologous gene therapy utilizing a lentiviral gene construct for patients with Wiskott Aldrich syndrome (WAS). OTL-103 aims to insert the corrected version of the WAS gene in patients, who often result in non-functioning leucocytes and platelets. Administration of the functioning cells will result in the cells differentiating into immune cells as well as the progenitor cells to platelets and a clinical trial is currently underway to assess the efficacy of OTL-103. This interview took place at Advanced Therapies Week 2022.