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Authors
Mathews, Ella W. https://orcid.org/0000-0003-1183-1988
Coffey, Sydney R. https://orcid.org/0000-0002-1477-2197
Gärtner, Annette https://orcid.org/0000-0001-6189-9679
Belgrad, Jillian https://orcid.org/0000-0002-2577-2336
Bragg, Robert M. https://orcid.org/0000-0003-0619-8954
O’Reilly, Daniel
Cantle, Jeffrey P. https://orcid.org/0000-0001-6051-0465
McHugh, Cassandra https://orcid.org/0009-0002-6184-3258
Summers, Ashley https://orcid.org/0000-0002-5425-0457
Fentz, Joachim https://orcid.org/0000-0002-9334-1298
Schwagarus, Tom
Cornelius, Antje
Lingos, Ioannis https://orcid.org/0009-0008-5846-957X
Burch, Zoe
Kovalenko, Marina
Andrew, Marissa A.
Bennett, C. Frank
Kordasiewicz, Holly B.
Marchionini, Deanna M. https://orcid.org/0000-0003-2071-1064
Wilkinson, Hilary https://orcid.org/0000-0002-0401-9329
Vogt, Thomas F.
Beuzer, Paolo
Pinto, Ricardo M. https://orcid.org/0000-0001-6744-2805
Khvorova, Anastasia https://orcid.org/0000-0001-6928-8071
Howland, David
Wheeler, Vanessa C. https://orcid.org/0009-0004-8259-5796
Carroll, Jeffrey B. https://orcid.org/0000-0003-1711-8868
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Funding
Funding for this research was provided by:
CHDI Foundation (A-18222)
CHDI Foundation (A-5038)
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Article History
Received: 6 November 2024
Accepted: 26 September 2025
First Online: 14 November 2025
Competing interests
: A.G., J.F., T.S., A.C., I.L. are employees of Evotec, and may have stock options. C.F.B. and H.K. Are full time employees at, and hold shares in, Ionis Pharmaceuticals. V.C.W. was a founding scientific advisory board member with a financial interest in Triplet Therapeutics Inc., her financial interests were reviewed and are managed by Massachusetts General Hospital (MGH) and Mass General Brigham (MGB) in accordance with their conflict of interest policies. V.C.W. is a scientific advisory board member of LoQus23 Therapeutics Ltd. and has provided paid consulting services to Acadia Pharmaceuticals Inc., Alnylam Inc., Biogen Inc., Passage Bio and Rgenta Therapeutics and has received research support from Pfizer Inc. J.B.C. has provided paid consulting and/or conducted sponsored research for Wave Life Sciences, Skyhawk Therapeutics, Cajal Neuroscience, Ionis Pharmaceuticals, Alnylam, and Guidepoint. D.H., D.M., P.B., and T.V.are full-time employees of CHDI Foundation. A.K. discloses ownership of stocks in RXi Pharmaceuticals and Advirna, and is a founder of Atalanta Therapeutics and Comanche Biopharma. R.B. received consulting fees from Takeda. The remaining authors declare no competing interests.
