Nexiguran Ziclumeran Gene Editing in Hereditary ATTR with Polyneuropathy

Julian D. Gillmore; Ed Gane; Jörg Täubel; Björn Pilebro; Andoni Echaniz-Laguna; Justin Kao; William Litchy; Safi Shahda; Alexandra Haagensen; Liron Walsh; Derek Smith; Jessica Kachadourian; Jonathan H. Ward; David Lebwohl; Peijuan Zhu; Yuanxin Xu; Adia Leung; Alison Sonderfan; David E. Gutstein; Garen Manvelian; David Adams

doi:10.1056/nejmoa2510209

Nexiguran Ziclumeran Gene Editing in Hereditary ATTR with Polyneuropathy

J Julian D. Gillmore (University College London, National Amyloidosis Centre, Royal Free Hospital, London) E Ed Gane (University of Auckland, Auckland, New Zealand) J Jörg Täubel (Richmond Pharmacology, London) B Björn Pilebro (Umeå University, Umeå, Sweden) A Andoni Echaniz-Laguna (Bicêtre University Hospital, Assistance Publique–Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France) J Justin Kao (Auckland City Hospital, Auckland, New Zealand) W William Litchy (Mayo Clinic, Rochester, MN) S Safi Shahda (Intellia Therapeutics, Cambridge, MA) A Alexandra Haagensen (Intellia Therapeutics, Cambridge, MA) L Liron Walsh (Intellia Therapeutics, Cambridge, MA) D Derek Smith (Intellia Therapeutics, Cambridge, MA) J Jessica Kachadourian (Intellia Therapeutics, Cambridge, MA) J Jonathan H. Ward (Intellia Therapeutics, Cambridge, MA) D David Lebwohl (Intellia Therapeutics, Cambridge, MA) P Peijuan Zhu (Intellia Therapeutics, Cambridge, MA) Y Yuanxin Xu (Intellia Therapeutics, Cambridge, MA) A Adia Leung (Intellia Therapeutics, Cambridge, MA) A Alison Sonderfan (Intellia Therapeutics, Cambridge, MA) D David E. Gutstein (Regeneron Pharmaceuticals, Tarrytown, NY) G Garen Manvelian (Regeneron Pharmaceuticals, Tarrytown, NY) D David Adams (Bicêtre University Hospital, Assistance Publique–Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France)

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Article Details

Journal New England Journal of Medicine

Volume / Issue Vol. 393, Issue 14

Published October 09, 2025

Pages 1375-1386

DOI 10.1056/nejmoa2510209

ISSN 0028-4793

Publisher Massachusetts Medical Society

Journal Info

New England Journal of Medicine

Massachusetts Medical Society

ISSN: 0028-4793 Social Sciences

Authors (21)

J

Julian D. Gillmore

University College London, National Amyloidosis Centre, Royal Free Hospital, London

E

Ed Gane

University of Auckland, Auckland, New Zealand

J

Jörg Täubel

Richmond Pharmacology, London

B

Björn Pilebro

Umeå University, Umeå, Sweden

A

Andoni Echaniz-Laguna

Bicêtre University Hospital, Assistance Publique–Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France

J

Justin Kao

Auckland City Hospital, Auckland, New Zealand

W

William Litchy

Mayo Clinic, Rochester, MN

S

Safi Shahda