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

SAFETY AND EFFICACY OF LENTIVIRAL HEMATOPOIETIC STEM AND PROGENITOR CELL GENE THERAPY WITH ETUVETIDIGENE AUTOTEMCEL FOR THE TREATMENT OF WISKOTT-ALDRICH SYNDROME (EBMT 2026) - P1/2, P3 | "Clinical Trial Registry: Phase I/II Study TIGET-WAS ClinicalTrials.gov ID: NCT01515462Phase III Study OTL-103-4 ClinicalTrials.gov ID: NCT03837483 Background: Wiskott-Aldrich Syndrome (WAS) is a life-threatening platelet disorder and inborn error of immunity caused by WAS gene mutations...Etu-cel was infused after rituximab and reduced-intensity conditioning... Results from this integrated safety and efficacy analysis show sustained long-term significant clinical benefit of one single infusion of etu-cel for the treatment of WAS, demonstrating its favorable benefit-risk profile with up to 13 years follow-up."

Clinical • Gene therapy • Atopic Dermatitis • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Immunology • Infectious Disease • Primary Immunodeficiency • CD34

Etuvetidigene Autotemcel: First Approval. (PubMed, Mol Diagn Ther) - "Etuvetidigene autotemcel (WASKYRA™) is an autologous haematopoietic stem cell (HSC)-based gene therapy being developed by Fondazione Telethon for the treatment of Wiskott-Aldrich syndrome (WAS)...Etuvetidigene autotemcel was approved in the EU for the same indication in January 2026. This article summarizes the milestones in the development of etuvetidigene autotemcel leading to this first approval for the treatment of WAS."

Journal • Bone Marrow Transplantation • Gene Therapies • Immunology • Pediatrics • Primary Immunodeficiency • Transplantation

OTL-103-4: 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

Enrollment closed • Gene Therapies • Primary Immunodeficiency

OTL-103-4: 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

Phase classification • Gene Therapies • Immunology • Primary Immunodeficiency

OTL-103-4: 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 | Recruiting | Sponsor: Orchard Therapeutics | Phase classification: P1/2 ➔ P2

Phase classification • Gene Therapies • Primary Immunodeficiency

OTL-103-4: 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

OTL-103-4: 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

Trial completion date • Trial primary completion date • Gene Therapies • Immunology • Primary Immunodeficiency

OTL-103-4: A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome (clinicaltrials.gov) - P1/2 | N=6 | Recruiting | Sponsor: Orchard Therapeutics Limited

New P1/2 trial • New P2 trial • Gene Therapies • Primary Immunodeficiency

OTL-103-4: 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

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

Fondazione Telethon receives european marketing authorisation for Waskyra (etuvetidigene autotemcel), a gene therapy for the treatment of Wiskott-Aldrich syndrome (Telethon Foundation) - "The European Commission’s decision follows the positive opinion issued by the EMA’s CHMP in November 2025."

EMA approval • FDA approval • Gene Therapies

The U.S. Food and Drug Administration today approved Waskyra (etuvetidigene autotemcel), the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS). (FDA) - "The safety and effectiveness of Waskyra was assessed based on two open-label, single-arm, multinational clinical studies and an expanded access program totaling 27 patients with severe WAS, which demonstrate substantial and sustained clinical benefit for patients with severe WAS, with significant reductions in the primary disease manifestations that drive morbidity and mortality."

FDA approval • Genetic Disorders

On 13 November 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Waskyra, intended for the treatment Wiskott-Aldrich Syndrome (WAS). (European Medicines Agency)

CHMP • Genetic Disorders

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

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

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