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Author

A. Aiuti

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Clinical trial Sep 2026

Etuvetidigene Autotemcel for the Treatment of Wiskott-Aldrich Syndrome.

BACKGROUND Wiskott-Aldrich syndrome is a rare, X-linked, life-threatening inborn error of immunity and platelet disorder caused by variants in the gene WAS. Etuvetidigene autotemcel (etu-cel) is an autologous gene therapy consisting of hematopoietic stem and progenitor cells that have been transduced ex vivo with a len...

F. Ferrua, S. Cenciarelli, S. Giannelli et al. · 0 citations
Open access Jul 2026

Engineering the future of advanced therapy medicinal products: a bioengineering call to action

This Perspective, grounded in clinical experience across hematological malignancies, monogenic diseases, and metabolic disorders, identifies five rate-limiting bottlenecks where bioengineering intervention is urgently needed and uniquely tractable: scalable and adaptive biomanufacturing.

Giulia Nieri, Anna Spiller, Stella Federico et al. · 0 citations

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