Migliavacca, Maddalena
Barzaghi, Federica
Fossati, Claudia
Rancoita, Paola M. V.
Gabaldo, Michela
Dionisio, Francesca
Giannelli, Stefania
Salerio, Federica Andrea
Ferrua, Francesca
Tucci, Francesca
Calbi, Valeria http://orcid.org/0000-0003-3514-666X
Gallo, Vera
Recupero, Salvatore
Consiglieri, Giulia
Pajno, Roberta
Sambuco, Maria
Priolo, Alessio http://orcid.org/0000-0001-9394-7292
Ferri, Chiara
Garella, Vittoria
Monti, Ilaria
Silvani, Paolo
Darin, Silvia
Casiraghi, Miriam
Corti, Ambra
Zancan, Stefano
Levi, Margherita
Cesana, Daniela http://orcid.org/0000-0001-6366-4224
Carlucci, Filippo
Pituch-Noworolska, Anna
AbdElaziz, Dalia http://orcid.org/0000-0002-7830-7008
Baumann, Ulrich http://orcid.org/0000-0002-0162-0266
Finocchi, Andrea
Cancrini, Caterina
Ladogana, Saverio
Meinhardt, Andrea http://orcid.org/0000-0002-7464-0751
Meyts, Isabelle http://orcid.org/0000-0003-1214-0302
Montin, Davide
Notarangelo, Lucia Dora
Porta, Fulvio
Pasquet, Marlène
Speckmann, Carsten
Stepensky, Polina
Tommasini, Alberto
Rabusin, Marco
Karakas, Zeynep
Galicchio, Miguel
Leonardi, Lucia
Duse, Marzia http://orcid.org/0000-0001-9958-2570
Guner, Sukru Nail http://orcid.org/0000-0002-8860-6132
Di Serio, Clelia
Ciceri, Fabio http://orcid.org/0000-0003-0873-0123
Bernardo, Maria Ester
Aiuti, Alessandro http://orcid.org/0000-0002-5398-1717
Cicalese, Maria Pia
Funding for this research was provided by:
Fondazione Telethon
Else Kröner-Fresenius-Stiftung
Article History
Received: 31 August 2022
Accepted: 21 December 2023
First Online: 14 February 2024
Competing interests
: The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Telethon Foundation and Ospedale San Raffaele (OSR). GT for ADA-SCID was developed at SR-Tiget and licensed to GlaxoSmithKline (GSK) in 2010. The treatments under NPP and HE were provided free of charge by GSK. Strimvelis Marketing Authorization in Europe occurred in 2016 (under GSK holding) and then transferred to Orchard Therapeutics (Netherlands) B.V. in 2018, which divested the program and transferred the authorization to Fondazione Telethon that became the holder in July 2023. The product, apart from the EU, is also currently approved in Iceland, Norway, Liechtenstein and the United Kingdom. The funders had no role in study design, data collection and analysis, decision to publish or preparation of the manuscript. A.A. receives funding from Fondazione Telethon for other research projects. A.A. was the PI of pilot, pivotal and long-term F-U study of SR-Tiget clinical trials of gene therapy for ADA-SCID. M.P.C. and M.M. are PI and deputy PI, respectively, of the Strimvelis Registry, RIS and RMMs studies. The other authors declare no competing interests.