Shots:
The CHMP has recommended Waskyra, an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS)
Waskyra is a single-administration therapy that uses a patient’s CD34+ hematopoietic stem and progenitor cells, modified with a lentiviral vector carrying the WAS gene
The therapy will be available to pts at IRCCS Ospedale San Raffaele, where the clinical trial was conducted. The BLA for WAS…
