etuvetidigene autotemcel

Etuvetidigene autotemcel is an autologous ex vivo hematopoietic stem and progenitor cell gene therapy for Wiskott-Aldrich syndrome. The product consists of CD34-positive hematopoietic stem and progenitor cells collected from the patient and transduced with a self-inactivating lentiviral vector encoding human WAS cDNA under control of a reconstituted endogenous WAS promoter, with the aim of restoring WAS protein expression after reinfusion following reduced-intensity conditioning. Clinically, it has shown durable multilineage engraftment, restoration of WASP expression, reduced severe infections and bleeding events, improvement in platelet counts, and resolution or improvement of eczema and immune dysregulation. The program originated from Telethon and San Raffaele, was previously developed under Orchard Therapeutics as OTL-103, and later returned to Fondazione Telethon; the product has been described as approved in the US and Europe for eligible patients with Wiskott-Aldrich syndrome who lack a suitable matched related donor for HSCT.

Brand names

WASKYRA

Other names

etu-celetuvetidigene autotemcel