Monaco Lucia, Faccio Lucia
Fondazione Telethon, Milan, Italy.
EMBO Mol Med. 2017 Mar;9(3):289-292. doi: 10.15252/emmm.201607293.
The recent approval of Strimvelis, the first gene therapy to gain marketing authorization (Schimmer & Breazzano, 2016), has drawn attention to Fondazione Telethon, the Italian charity that played a pivotal role in this effort. Although it is not uncommon that advanced therapies, such as Strimvelis, are developed by partnerships between academia and industry, direct involvement of a charity in key steps of this process is still unusual. Illustrating the strategies and operational model adopted by Fondazione Telethon to achieve its mission of supporting excellent research aimed at curing rare genetic diseases may elucidate some of the enabling factors behind the Strimvelis success story.
首个获得上市许可的基因疗法Strimvelis最近获批（施immer与布雷亚扎诺，2016年），这使得人们将目光投向了意大利慈善机构“Telethon基金会”，该机构在这一过程中发挥了关键作用。虽然像Strimvelis这样的先进疗法由学术界和产业界合作开发并不罕见，但慈善机构直接参与这一过程的关键步骤仍属不寻常。阐释Telethon基金会为实现其支持旨在治愈罕见遗传疾病的卓越研究的使命而采用的策略和运营模式，可能会揭示Strimvelis成功故事背后的一些促成因素。
