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Authors
Cesana, Daniela https://orcid.org/0000-0001-6366-4224
Cicalese, Maria Pia
Calabria, Andrea https://orcid.org/0000-0003-3515-3384
Merli, Pietro
Caruso, Roberta
Volpin, Monica https://orcid.org/0000-0002-0794-9900
Rudilosso, Laura
Migliavacca, Maddalena
Barzaghi, Federica
Fossati, Claudia
Gazzo, Francesco
Pizzi, Simone
Ciolfi, Andrea https://orcid.org/0000-0002-6191-0978
Bruselles, Alessandro
Tucci, Francesca
Spinozzi, Giulio https://orcid.org/0000-0002-4220-2474
Pais, Giulia https://orcid.org/0009-0005-5621-4803
Benedicenti, Fabrizio
Barcella, Matteo
Merelli, Ivan
Gallina, Pierangela
Giannelli, Stefania
Dionisio, Francesca
Scala, Serena
Casiraghi, Miriam
Strocchio, Luisa
Vinti, Luciana
Pacillo, Lucia https://orcid.org/0000-0002-7950-8848
Draghi, Eleonora
Cesana, Marcella
Riccardo, Sara
Colantuono, Chiara https://orcid.org/0000-0003-3014-6978
Six, Emmanuelle https://orcid.org/0000-0001-7806-0968
Cavazzana, Marina
Carlucci, Filippo
Schmidt, Manfred
Cancrini, Caterina
Ciceri, Fabio https://orcid.org/0000-0003-0873-0123
Vago, Luca https://orcid.org/0000-0003-4247-3175
Cacchiarelli, Davide https://orcid.org/0000-0002-9621-6716
Gentner, Bernhard https://orcid.org/0000-0001-6024-4718
Naldini, Luigi https://orcid.org/0000-0002-7835-527X
Tartaglia, Marco https://orcid.org/0000-0001-7736-9672
Montini, Eugenio https://orcid.org/0000-0003-1771-6067
Locatelli, Franco https://orcid.org/0000-0002-7976-3654
Aiuti, Alessandro https://orcid.org/0000-0002-5398-1717
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Funding
Funding for this research was provided by:
Fondazione Telethon
Ministry of Health, Italy | Agenzia Italiana del Farmaco, Ministero della Salute (GR-2018-12367860)
Fondazione Italiana per la Ricerca sul Cancro (IG #22197, 9962, IG #21614)
Ministero dell'Istruzione, dell'Università e della Ricerca (PRIN 2017WC8499_004, FOE 2019)
Ministero dell'Istruzione, dell'Università e della Ricerca
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Article History
Received: 13 October 2023
Accepted: 10 April 2024
First Online: 30 April 2024
Competing interests
: The San Raffaele Telethon Institute for GT (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 Named Patient Program and Hospital Exemption 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. Following disinvestment by Orchard, Fondazione Telethon became the Strimvelis Marketing Authorization Holder in July 2023. The product is also currently licensed in Iceland, Norway, Liechtenstein, and UK. A.A: was the PI of pilot and pivotal SR-TIGET clinical trial of GT for ADA SCID. A.A. is a member of the Committee for Advanced Therapies (CAT) and his views are personal and may not be understood or quoted as being made on behalf of the European Medicine Agency (EMA). M.P. Cicalese is the PI of the Strimvelis Registry, RIS and RMMs studies. Davide Cacchiarelli is founder, shareholder, and consultant of NEGEDIA srl Sara Riccardo, Chiara Colantuono are employees of NEGEDIA srl. All other authors declare no other competing interests.
