A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

Clinical Publications

• Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study
• Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome
• B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome
• Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy
• (Abstract) Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010 - ASH 2019
• Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome
• Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome

News and Press Releases

• Fondazione Telethon submits US marketing authorization application for etuvetidigene autotemcel gene therapy for the treatment of Wiskott-Aldrich syndrome
• Inside the efforts to rescue a rare disease gene therapy

Preclinical Publications

• Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction
• Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome
• Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models

Related NCTID

• Phase 1/2: NCT01515462
• Phase 1/2: NCT01347242 (same vector, sponsored by Genethon)
• Phase 1/2: NCT01347346 (same vector, sponsored by Genethon)
• Phase 1/2: NCT01410825
• LTFU: NCT02333760 (sponsored by Genethon)