EMA’s CHMP recommends five new products

CHMP recommended approval of the first gene therapy for Wiskott-Aldrich syndrome, a rare genetic disease affecting blood and immune cells, at its November meeting.

Waskyra etuvetidigene autotemcel from Italy’s Fondazione Telethon is to be used in patients eligible for hematopoietic stem cell transplant but without a suitable donor. It uses autologous CD34+ stem cells transduced ex vivo with a lentiviral vector to express functional WAS protein...