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Genethon’s Lentiviral Vector-Based Gene Therapy Demonstrates Long-Term Safety and Efficacy for Wiskott-Aldrich Syndrome

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Anne Gally in her laboratory

Genethon announced that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.

The long-term results of the WAS clinical trial, sponsored by Genethon and conducted by colleagues in France and England, were published in Nature Medicine, in a paper titled “Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome.”  The vector used in the study was designed, developed and manufactured by Genethon.

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