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Wiskott-Aldrich syndrome: Long-term results of gene therapy developed

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Wiskott-Aldrich syndrome: Long-term results of gene therapy developed

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A single infusion of genetically corrected autologous hematopoietic stem cells can provide lasting clinical benefits for patients with Wiskott-Aldrich syndrome, a rare genetic disorder that impairs immune and platelet function, exposing affected children from an early age to severe infections, bleeding episodes, eczema and autoimmune complications.

The findings are reported in a paper published in the New England Journal of Medicine and authored by Alessandro Aiuti, head of pediatric immunohematology at IRCCS Ospedale San Raffaele, deputy director of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), professor of pediatrics at Vita-Salute San Raffaele University, and the study's senior and corresponding author.

The publication presents the outcomes of 27 patients treated with etuvetidigene autotemcel (etu-cel), an autologous gene therapy developed to correct the genetic defect underlying the disease. Patients were followed for at least 5.7 years, with some monitored for more than 13 years. Overall survival was 96% at both 1 and 5 years, while severe infections and moderate-to-severe bleeding events were markedly reduced following treatment. None of the adverse events observed were attributable to the gene therapy product, supporting a favorable risk-benefit profile.

The program originated and was developed within SR-Tiget, established in 1996 through a partnership between IRCCS Ospedale San Raffaele and Fondazione Telethon to develop gene therapies for rare genetic diseases, together with the Pediatric Immunohematology and Bone Marrow Transplant Unit of IRCCS Ospedale San Raffaele.


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