{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T04:23:10Z","timestamp":1789014190734,"version":"build-2803163510"},"reference-count":13,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2025,12,1]],"date-time":"2025-12-01T00:00:00Z","timestamp":1764547200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,12,1]],"date-time":"2025-12-01T00:00:00Z","timestamp":1764547200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T00:00:00Z","timestamp":1762387200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Methods &amp; Clinical Development"],"published-print":{"date-parts":[[2025,12]]},"DOI":"10.1016\/j.omtm.2025.101628","type":"journal-article","created":{"date-parts":[[2025,11,11]],"date-time":"2025-11-11T23:30:25Z","timestamp":1762903825000},"page":"101628","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":3,"title":["Nonclinical toxicity study duration in AAV gene therapy development: Evidence from industry survey supports adequacy of short-term assessments"],"prefix":"10.1016","volume":"33","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2286-0303","authenticated-orcid":false,"given":"Basel T.","family":"Assaf","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Edwards","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shawn Jay","family":"Berens","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian","family":"Lauritzen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christelle","family":"Pierrot","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francis Fonyuy","family":"Tukov","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina","family":"Bertinetti-Lapatki","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manuela","family":"Braun","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kai","family":"Schaefer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica L.","family":"Lynch","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2025.101628_bib1","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1016\/j.omtm.2020.08.017","article-title":"Nonclinical Studies that Support Viral Vector-Delivered Gene Therapies: An EFPIA Gene Therapy Working Group Perspective","volume":"19","author":"Bolt","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2025.101628_bib5","doi-asserted-by":"crossref","DOI":"10.1016\/j.taap.2024.117087","article-title":"Do longer duration nonclinical toxicology studies provide predictive clinical safety value? The IQ consortium longer duration nonclinical to clinical translational database","volume":"492","author":"Monticello","year":"2024","journal-title":"Toxicol. Appl. Pharmacol."},{"key":"10.1016\/j.omtm.2025.101628_bib6","doi-asserted-by":"crossref","DOI":"10.1016\/j.yrtph.2022.105329","article-title":"Re-evaluating the need for chronic toxicity studies with therapeutic monoclonal antibodies, using a weight of evidence approach","volume":"138","author":"Chien","year":"2023","journal-title":"Regul. Toxicol. Pharmacol."},{"key":"10.1016\/j.omtm.2025.101628_bib7","doi-asserted-by":"crossref","first-page":"2","DOI":"10.1016\/j.yrtph.2007.08.001","article-title":"Duration of chronic toxicity studies for biotechnology-derived pharmaceuticals: is 6 months still appropriate?","volume":"50","author":"Clarke","year":"2008","journal-title":"Regul. Toxicol. Pharmacol."},{"key":"10.1016\/j.omtm.2025.101628_bib8","doi-asserted-by":"crossref","first-page":"118","DOI":"10.1177\/01926233211041962","article-title":"Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors","volume":"50","author":"Hutt","year":"2022","journal-title":"Toxicol. Pathol."},{"key":"10.1016\/j.omtm.2025.101628_bib15","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1208\/s12248-021-00678-7","article-title":"The Perspective of DMPK on Recombinant Adeno-Associated Virus-Based Gene Therapy: Past Learning, Current Support, and Future Contribution","volume":"24","author":"Chen","year":"2022","journal-title":"AAPS J."},{"key":"10.1016\/j.omtm.2025.101628_bib17","doi-asserted-by":"crossref","first-page":"523","DOI":"10.1177\/01926233241298892","article-title":"Systemic Toxicity of Recombinant Adeno-Associated Virus Gene Therapy Vectors","volume":"52","author":"Assaf","year":"2024","journal-title":"Toxicol. Pathol."},{"key":"10.1016\/j.omtm.2025.101628_bib18","series-title":"Haschek and Rousseaux' S Handbook of Toxicologic Pathology","first-page":"269","article-title":"Chapter 8 - Gene Therapy and Gene Editing","author":"Assaf","year":"2023"},{"key":"10.1016\/j.omtm.2025.101628_bib19","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1089\/hum.2018.015","article-title":"Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN","volume":"29","author":"Hinderer","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2025.101628_bib20","doi-asserted-by":"crossref","first-page":"808","DOI":"10.1089\/hum.2020.167","article-title":"Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology","volume":"31","author":"Hordeaux","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2025.101628_bib21","doi-asserted-by":"crossref","first-page":"60","DOI":"10.1038\/s41434-023-00405-1","article-title":"Adeno-associated virus vectors and neurotoxicity-lessons from preclinical and human studies","volume":"32","author":"Stone","year":"2025","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2025.101628_bib36","doi-asserted-by":"crossref","first-page":"2479","DOI":"10.1016\/j.ymthe.2025.04.045","article-title":"Current clinical applications of AAV-mediated gene therapy","volume":"33","author":"Byrne","year":"2025","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2025.101628_bib37","doi-asserted-by":"crossref","first-page":"884","DOI":"10.1016\/j.nmd.2023.09.008","article-title":"261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands","volume":"33","author":"Servais","year":"2023","journal-title":"Neuromuscul. Disord."}],"container-title":["Molecular Therapy Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050125002232?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050125002232?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,4,14]],"date-time":"2026-04-14T20:31:31Z","timestamp":1776198691000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050125002232"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,12]]},"references-count":13,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2025,12]]}},"alternative-id":["S2329050125002232"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2025.101628","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2025,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Nonclinical toxicity study duration in AAV gene therapy development: Evidence from industry survey supports adequacy of short-term assessments","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2025.101628","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Authors. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"101628"}}