Article History
Accepted: 13 October 2020
First Online: 10 December 2020
Competing interests
: The San Raffaele Telethon Institute for Gene Therapy is a joint venture between Fondazione Telethon and Ospedale San Raffaele. Gene therapies for adenosine deaminase-deficient severe combined immunodeficiency, Wiskott–Aldrich syndrome, metachromatic leukodystrophy, β-thalassaemia and mucopolysaccharidosis type I developed at the San Raffaele Telethon Institute for Gene Therapy were licensed to Orchard Therapeutics in 2018 and 2019. A.A. is the principal investigator in the above-mentioned clinical trials. A.J.T has equity in and is on the scientific advisory board for Orchard Therapeutics and receives consultancy payments from Rocket Pharmaceuticals.