Nature reviews. Drug discovery · 2024
The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap.
СтатьяОбзорПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод в очереди
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Jonker Anneliene H, Tataru Elena-Alexandra, Graessner Holm, Dimmock David, Jaffe Adam, Baynam Gareth, Davies James, Mitkus Shruti, Iliach Oxana, Horgan Rich, Augustine Erika F, Bateman-House Alison, Pasmooij Anna Maria Gerdina, Yu Tim, Synofzik Matthis, Douville Julie, Lapteva Larissa, Brooks Philip John, O'Connor Daniel, Aartsma-Rus Annemieke, N-of-1 Task Force of the International Rare Diseases Research Consortium (IRDiRC)
Аннотация
In recent years, a small number of people with rare diseases caused by unique genetic variants have been treated with therapies developed specifically for them. This pioneering field of genetic N-of-1 therapies is evolving rapidly, giving hope for the individualized treatment of people living with very rare diseases. In this Review, we outline the concept of N-of-1 individualized therapies, focusing on genetic therapies, and illustrate advances and challenges in the field using cases for which therapies have been successfully developed. We discuss why the traditional drug development and reimbursement pathway is not fit for purpose in this field, and outline the pragmatic, regulatory and ethical challenges this poses for future access to N-of-1 therapies. Finally, we provide a roadmap for N-of-1 individualized therapy development.