etuvetidigene autotemcel (OTL-103) / GSK, IRCCS San Raffaele Hospital, Fondazione Telethon, Kyowa Kirin - LARVOL DELTA

Fondazione Telethon submits EU marketing authorization application for Etuvetidigene Autotemcel Gene Therapy for the treatment of Wiskott-Aldrich Syndrome (Telethon Foundation) - "Fondazione Telethon announced that it has submitted the Marketing Authorization Application (MAA) for the gene therapy - etuvetidigene autotemcel - for the treatment of patients with Wiskott-Aldrich Syndrome (WAS), a rare genetic disease of the immune system to the European Medicines Agency (EMA)."

EMA filing • Genetic Disorders

TIGET-WAS: Gene Therapy for Wiskott-Aldrich Syndrome (clinicaltrials.gov) - P1/2 | N=8 | Completed | Sponsor: Fondazione Telethon | Active, not recruiting ➔ Completed

Trial completion • Gene Therapies • Immunology • Primary Immunodeficiency • CD34

TIGET-WAS: Gene Therapy for Wiskott-Aldrich Syndrome (clinicaltrials.gov) - P1/2 | N=8 | Active, not recruiting | Sponsor: Orchard Therapeutics | Trial completion date: Sep 2025 ➔ Sep 2030

Gene therapy • Trial completion date • Gene Therapies • Immunology • Primary Immunodeficiency • CD34 • NCAM1

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome (clinicaltrials.gov) - P3 | N=10 | Active, not recruiting | Sponsor: Orchard Therapeutics | Trial completion date: Jan 2024 ➔ Sep 2027 | Trial primary completion date: Jul 2023 ➔ Sep 2025

Gene therapy • Trial completion date • Trial primary completion date • Viral vector • Gene Therapies • Immunology • Primary Immunodeficiency

LENTIVIRAL HEMATOPOIETIC STEM AND PROGENITOR CELL GENE THERAPY FOR WISKOTT-ALDRICH SYNDROME: SAFETY AND CLINICAL BENEFIT IN 23 PATIENTS UP TO 10.5 YEARS OF FOLLOW-UP (ESID 2022) - "Methods OTL-103 is an investigational autologous hematopoietic stem and progenitor cell (HSPC) GT composed of CD34 + HSPCs transduced ex vivo with a self-inactivating lentiviral vector encoding human WAS cDNA under endogenous promoter control...All received rituximab and reduced-intensity conditioning pre-GT...Eczema improved and clinical autoimmunity resolved. Conclusions Our data show that lentiviral GT for WAS is well tolerated and leads to sustained clinical benefit, highlighting its potential as an alternative treatment for WAS patients."

Clinical • Atopic Dermatitis • Immunology • Primary Immunodeficiency • CD34

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome (clinicaltrials.gov) - P3 | N=10 | Active, not recruiting | Sponsor: Orchard Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene Therapies • Immunology • Primary Immunodeficiency

Up to 10.5 Years of Follow-Up in 17 Subjects Treated with Hematopoietic Stem and Progenitor Cell Lentiviral Gene Therapy for Wiskott-Aldrich Syndrome (ASGCT 2022) - " All subjects received rituximab and reduced-intensity conditioning with busulfan and fludarabine pre-GT. This safety and efficacy analysis of subjects treated with OTL-103 demonstrates the potential of GT as an effective treatment for patients with WAS, including in those ≥5 years."

Clinical • Atopic Dermatitis • Bone Marrow Transplantation • Dermatology • Gene Therapies • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Oncology • Primary Immunodeficiency • Transplantation • CD34

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome (clinicaltrials.gov) - P3; N=9; Recruiting; Sponsor: Orchard Therapeutics; Active, not recruiting ➔ Recruiting; Trial completion date: Dec 2022 ➔ Jan 2024; Trial primary completion date: Feb 2022 ➔ Jul 2023

Clinical • Enrollment open • Trial completion date • Trial primary completion date • Gene Therapies • Immunology • Primary Immunodeficiency

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome (clinicaltrials.gov) - P3; N=6; Active, not recruiting; Sponsor: Orchard Therapeutics; Phase classification: P2 ➔ P3

Clinical • Phase classification • Gene Therapies • Immunology • Primary Immunodeficiency

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome (clinicaltrials.gov) - P2; N=6; Active, not recruiting; Sponsor: Orchard Therapeutics; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed