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Article History

First Online: 10 March 2025

Competing interests

: D.J.O.C. is employed by a pharmaceutical industry trade association. M.W. is a co-founder of and consultant for Pepgen Limited. M.M. is founder and chair of Rosa Genomic Medicines, non-executive director of Autolus Therapeutics PLC, chair of investment committee L&G UK Universities Ventures LP and non-executive director of Synairgen PLC. N.B. is chair, Genomics England; chair, Oxford University Innovation; director, supervisory board, BioNTech SE; and senior independent non-executive director, RTW Biotech Opportunities Ltd (a full list of her interests can be accessed here: ). M.A.B. is a consultant for Altis Medicines, Grey Wolf Therapeutics, Incyte and Ipsen and has research funding from UCB. D.L. is a trustee of Myeloma UK. T.Y. has received research support for an N=1 trial in ataxia telangiectasia from EveryONE Medicines and is named as an inventor on three patents pertaining to antisense oligonucleotide treatments: for transposon-associated diseases (WO2019055460A1), ataxia telangiectasia (US20230174979A1) and progranulin (US11359199B2); he has also received scientific consulting fees as pertains to ASO therapies from SynaptixBio, RegUp, Servier Pharmaceuticals and Lilly, serves as an unpaid board member for the N=1 Collaborative, the Oligonucleotide Therapeutics Society and the Society for RNA Therapeutics, and is an unpaid scientific advisor to several rare disease foundations. J.V. is a co-founder and unpaid board member of the N=1 Collaborative, a co-founder and consultant for EveryONE Medicines and an unpaid board member for several rare disease foundations.