OTL-103: An autologous gene therapy for Wiskott Aldrich syndrome

Simon Ng — Thu, 03 Mar 2022 17:46:27 +0000

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.

Novel gene therapies for metachromatic leukodystrophy

Simon Ng — Thu, 03 Mar 2022 17:46:25 +0000

Kent Christopherson, PhD, Orchard Therapeutics, Boston, MA, describes the development and mechanism of action of atidarsagene autotemcel, a gene therapy consisting of an autologous CD34⁺ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced using a lentiviral vector. The gene therapy, which has been approved in the EU, aims to treat metachromatic leukodystrophy (MLD) and the vector encodes the human arylsulfatase-A (ARSA) gene, which is faulty in patients with MLD. HSPC cells with the functional gene can subsequently produce the correct protein, which can be trafficked and permanently integrated in the central nervous system (CNS). This interview took place at Advanced Therapies Week 2022.

Kent Christopherson – VJRegenMed

OTL-103: An autologous gene therapy for Wiskott Aldrich syndrome

Novel gene therapies for metachromatic leukodystrophy