Access to gene therapy for rare diseases when commercialization is not fit for purpose

Thomas A. Fox, Juan A. Bueren, Fabio Candotti, Alain Fischer, Alessandro Aiuti, Arjan C. Lankester, The AGORA Initiative, Michael H. Albert, Maria Ester Bernardo, Marina Cavazzana, Stephan Ehl, Bénédicte Neven, J Prévot, Axel Schambach, Julián Sevilla, Adrian J. Thrasher, Robert Wynn, Claire Booth