{"id":"https://openalex.org/W7220905784","doi":"https://doi.org/10.1016/j.omta.2026.201862","title":"AAV serotype IVDs for patient selection in gene therapy development: Technical and regulatory considerations by ARDAT consortium","display_name":"AAV serotype IVDs for patient selection in gene therapy development: Technical and regulatory considerations by ARDAT consortium","publication_year":2026,"publication_date":"2026-10-08","ids":{"openalex":"https://openalex.org/W7220905784","doi":"https://doi.org/10.1016/j.omta.2026.201862","pmid":"https://pubmed.ncbi.nlm.nih.gov/42990908","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/13734834"},"language":"en","primary_location":{"id":"doi:10.1016/j.omta.2026.201862","doi":"https://doi.org/10.1016/j.omta.2026.201862","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.omta.2026.201862","pdf_url":null,"source":{"id":"https://openalex.org/S5407055139","display_name":"Molecular Therapy Advances","issn_l":"3117-387X","issn":["3117-387X"],"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":["jufo-1","oarl"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy Advances","raw_type":"journal-article"},"volume":null,"type":"review","is_traditional":true,"indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"https://doi.org/10.1016/j.omta.2026.201862","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5044087072","display_name":"Manuela Braun","orcid":"https://orcid.org/0000-0001-5837-5444","observed_orcids":["https://orcid.org/0000-0001-5837-5444"]},"institutions":[{"id":"https://openalex.org/I67348948","display_name":"Bayer (Germany)","ror":"https://ror.org/04hmn8g73","country_code":"DE","type":"company","lineage":["https://openalex.org/I67348948"]}],"countries":["DE"],"is_corresponding":true,"raw_author_name":"Manuela Braun","raw_affiliation_strings":["Bayer AG, Pharmaceuticals R&D, 13353 Berlin, Germany"],"raw_orcid":"https://orcid.org/0000-0001-5837-5444","affiliations":[{"raw_affiliation_string":"Bayer AG, Pharmaceuticals R&D, 13353 Berlin, Germany","institution_ids":["https://openalex.org/I67348948"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5157944913","display_name":"Christel Ravesteijn-Verrijt","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210157866","display_name":"Astellas Pharma (Netherlands)","ror":"https://ror.org/04kyfd050","country_code":"NL","type":"company","lineage":["https://openalex.org/I142395110","https://openalex.org/I4210157866"]}],"countries":["NL"],"is_corresponding":false,"raw_author_name":"Christel Ravesteijn-Verrijt","raw_affiliation_strings":["Astellas Pharma Europe B.V., 2333 BE Leiden, the Netherlands"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Astellas Pharma Europe B.V., 2333 BE Leiden, the Netherlands","institution_ids":["https://openalex.org/I4210157866"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5113556079","display_name":"Philipp Schatz","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I67348948","display_name":"Bayer (Germany)","ror":"https://ror.org/04hmn8g73","country_code":"DE","type":"company","lineage":["https://openalex.org/I67348948"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Philipp Schatz","raw_affiliation_strings":["Bayer AG, Pharmaceuticals R&D, 13353 Berlin, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Bayer AG, Pharmaceuticals R&D, 13353 Berlin, Germany","institution_ids":["https://openalex.org/I67348948"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5157964119","display_name":"Yanmei Lu","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210140998","display_name":"Sangamo BioSciences (United States)","ror":"https://ror.org/046dwx283","country_code":"US","type":"company","lineage":["https://openalex.org/I4210140998"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Yanmei Lu","raw_affiliation_strings":["Sangamo Therapeutics, Inc., Richmond, CA 94804, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Sangamo Therapeutics, Inc., Richmond, CA 94804, USA","institution_ids":["https://openalex.org/I4210140998"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5032004026","display_name":"Sarra Seninet","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210107271","display_name":"GenoSafe","ror":"https://ror.org/01wpxe954","country_code":"FR","type":"company","lineage":["https://openalex.org/I4210107271"]}],"countries":["FR"],"is_corresponding":false,"raw_author_name":"Sarra Seninet","raw_affiliation_strings":["GenoSafe, 91002 Evry, France"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"GenoSafe, 91002 Evry, France","institution_ids":["https://openalex.org/I4210107271"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5082348909","display_name":"Jon Giblin","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Jon Giblin","raw_affiliation_strings":["Asphalion S.L., 08014 Barcelona, Spain"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Asphalion S.L., 08014 Barcelona, Spain","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5066141980","display_name":"John M. Millholland","orcid":"https://orcid.org/0000-0003-2129-3298","observed_orcids":["https://orcid.org/0000-0003-2129-3298"]},"institutions":[{"id":"https://openalex.org/I4210091812","display_name":"Bristol-Myers Squibb (United States)","ror":"https://ror.org/00gtmwv55","country_code":"US","type":"company","lineage":["https://openalex.org/I4210091812"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"John Millholland","raw_affiliation_strings":["Bristol Myers Squibb, Princeton, NJ 08543, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Bristol Myers Squibb, Princeton, NJ 08543, USA","institution_ids":["https://openalex.org/I4210091812"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5065317972","display_name":"Gurunadh Reddy Chichili","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210165171","display_name":"Astellas Pharma (United States)","ror":"https://ror.org/05pw69n24","country_code":"US","type":"company","lineage":["https://openalex.org/I142395110","https://openalex.org/I4210165171"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Gurunadh Chichili","raw_affiliation_strings":["Astellas Pharma Global Development, Northbrook, IL 60062, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Astellas Pharma Global Development, Northbrook, IL 60062, USA","institution_ids":["https://openalex.org/I4210165171"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5090670617","display_name":"Fraser McBlane","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1283582996","display_name":"Novartis (Switzerland)","ror":"https://ror.org/02f9zrr09","country_code":"CH","type":"company","lineage":["https://openalex.org/I1283582996"]}],"countries":["CH"],"is_corresponding":false,"raw_author_name":"Fraser McBlane","raw_affiliation_strings":["Novartis, 4056 Basel, Switzerland"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Novartis, 4056 Basel, Switzerland","institution_ids":["https://openalex.org/I1283582996"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5023643447","display_name":"Johannes L. Stanta","orcid":"https://orcid.org/0000-0001-6015-515X","observed_orcids":["https://orcid.org/0000-0001-6015-515X"]},"institutions":[{"id":"https://openalex.org/I4210140431","display_name":"Celerion (United Kingdom)","ror":"https://ror.org/03paq3x39","country_code":"GB","type":"company","lineage":["https://openalex.org/I4210103107","https://openalex.org/I4210140431"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Johannes Stanta","raw_affiliation_strings":["Celerion (GB) Ltd., BT9 6AD Belfast, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Celerion (GB) Ltd., BT9 6AD Belfast, UK","institution_ids":["https://openalex.org/I4210140431"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5041127003","display_name":"Mimoun Azzouz","orcid":"https://orcid.org/0000-0001-6564-5967","observed_orcids":["https://orcid.org/0000-0001-6564-5967"]},"institutions":[{"id":"https://openalex.org/I91136226","display_name":"University of Sheffield","ror":"https://ror.org/05krs5044","country_code":"GB","type":"education","lineage":["https://openalex.org/I91136226"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Mimoun Azzouz","raw_affiliation_strings":["University of Sheffield, S102TN Sheffield, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of Sheffield, S102TN Sheffield, UK","institution_ids":["https://openalex.org/I91136226"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5103451476","display_name":"Christopher J. Mann","orcid":"https://orcid.org/0009-0005-4913-5349","observed_orcids":["https://orcid.org/0009-0005-4913-5349"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Christopher Mann","raw_affiliation_strings":["Asphalion SL, 08014 Barcelona, Spain"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Asphalion SL, 08014 Barcelona, Spain","institution_ids":[]}]}],"institutions":[],"countries_distinct_count":6,"institutions_distinct_count":9,"corresponding_author_ids":["https://openalex.org/A5044087072"],"corresponding_institution_ids":["https://openalex.org/I67348948"],"apc_list":null,"apc_paid":null,"fwci":0.0,"has_fulltext":true,"cited_by_count":0,"citation_normalized_percentile":{"value":0.54297792,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":null,"biblio":{"volume":"34","issue":"4","first_page":"201862","last_page":"201862"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9955999851226807,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},"topics":[{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9955999851226807,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10725","display_name":"RNA Interference and Gene Delivery","score":0.0005000000237487257,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":0.0003000000142492354,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/in-vitro-diagnostic-assays","display_name":"in vitro diagnostic assays","score":0.6899999976158142},{"id":"https://openalex.org/keywords/gene-therapy-development","display_name":"gene therapy development","score":0.7739999890327454},{"id":"https://openalex.org/keywords/patient-selection","display_name":"patient selection","score":0.7670000195503235},{"id":"https://openalex.org/keywords/adaptive-clinical-trials","display_name":"adaptive clinical trials","score":0.8130000233650208},{"id":"https://openalex.org/keywords/analytical-method-validation","display_name":"analytical method validation","score":0.6859999895095825},{"id":"https://openalex.org/keywords/regulatory-aspects","display_name":"regulatory considerations","score":0.8140000104904175}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.6707000136375427},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.531000018119812},{"id":"https://openalex.org/C81917197","wikidata":"https://www.wikidata.org/wiki/Q628760","display_name":"Selection (genetic algorithm)","level":2,"score":0.5273000001907349},{"id":"https://openalex.org/C10389963","wikidata":"https://www.wikidata.org/wiki/Q848328","display_name":"Serotype","level":2,"score":0.5206999778747559},{"id":"https://openalex.org/C159047783","wikidata":"https://www.wikidata.org/wiki/Q7215","display_name":"Virology","level":1,"score":0.37369999289512634},{"id":"https://openalex.org/C70721500","wikidata":"https://www.wikidata.org/wiki/Q177005","display_name":"Computational biology","level":1,"score":0.3418000042438507},{"id":"https://openalex.org/C60644358","wikidata":"https://www.wikidata.org/wiki/Q128570","display_name":"Bioinformatics","level":1,"score":0.3240000009536743},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.32170000672340393},{"id":"https://openalex.org/C177713679","wikidata":"https://www.wikidata.org/wiki/Q679690","display_name":"Intensive care medicine","level":1,"score":0.31769999861717224},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.3091000020503998},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.3068999946117401},{"id":"https://openalex.org/C2779473830","wikidata":"https://www.wikidata.org/wiki/Q1540899","display_name":"MEDLINE","level":2,"score":0.2777000069618225},{"id":"https://openalex.org/C8891405","wikidata":"https://www.wikidata.org/wiki/Q1059","display_name":"Immune system","level":2,"score":0.2524000108242035}],"mesh":[],"locations_count":3,"locations":[{"id":"doi:10.1016/j.omta.2026.201862","doi":"https://doi.org/10.1016/j.omta.2026.201862","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.omta.2026.201862","pdf_url":null,"source":{"id":"https://openalex.org/S5407055139","display_name":"Molecular Therapy Advances","issn_l":"3117-387X","issn":["3117-387X"],"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":["jufo-1","oarl"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy Advances","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:13734834","doi":null,"is_oa":true,"landing_page_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC13734834/","pdf_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC13734834/pdf/main.pdf","source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Mol Ther Adv","raw_type":"Text"},{"id":"pmid:42990908","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/42990908","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular therapy. Advances","raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.1016/j.omta.2026.201862","doi":"https://doi.org/10.1016/j.omta.2026.201862","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.omta.2026.201862","pdf_url":null,"source":{"id":"https://openalex.org/S5407055139","display_name":"Molecular Therapy Advances","issn_l":"3117-387X","issn":["3117-387X"],"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":["jufo-1","oarl"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy Advances","raw_type":"journal-article"},"sustainable_development_goals":[{"display_name":"Good health and well-being","score":0.8048999905586243,"id":"https://metadata.un.org/sdg/3"}],"study_designs":[],"awards":[],"funders":[{"id":"https://openalex.org/F4320307765","display_name":"Pfizer","ror":"https://ror.org/01xdqrp08"},{"id":"https://openalex.org/F4320314178","display_name":"European Federation of Pharmaceutical Industries and Associations","ror":"https://ror.org/00g1x4v36"},{"id":"https://openalex.org/F4320319491","display_name":"Innovative Health Initiative","ror":"https://ror.org/04afqts81"},{"id":"https://openalex.org/F4320320300","display_name":"European Commission","ror":"https://ror.org/00k4n6c32"}],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W7220905784.pdf","grobid_xml":"https://content.openalex.org/works/W7220905784.grobid-xml"},"referenced_works_count":21,"referenced_works":["https://openalex.org/W2032868562","https://openalex.org/W2112876769","https://openalex.org/W2144536093","https://openalex.org/W2935758806","https://openalex.org/W2947983278","https://openalex.org/W2988055556","https://openalex.org/W4200551987","https://openalex.org/W4285097740","https://openalex.org/W4291156529","https://openalex.org/W4320485513","https://openalex.org/W4321480301","https://openalex.org/W4384924887","https://openalex.org/W4387475882","https://openalex.org/W4388014839","https://openalex.org/W4388860142","https://openalex.org/W4390727025","https://openalex.org/W4391973026","https://openalex.org/W4394005744","https://openalex.org/W4405187673","https://openalex.org/W4409628286","https://openalex.org/W4414708259"],"related_works":["https://openalex.org/W4237598210","https://openalex.org/W4249136423","https://openalex.org/W3146935801","https://openalex.org/W4243236292","https://openalex.org/W2471129711","https://openalex.org/W4234952811","https://openalex.org/W4322770338","https://openalex.org/W3025546795","https://openalex.org/W4247793080","https://openalex.org/W4298010004"],"abstract_inverted_index":{"diagnostic":[0],"(IVD)":[1],"assay,":[2],"an":[3],"\"AAV":[4],"serotype":[5],"IVD,\"":[6],"with":[7,33,58],"pragmatic":[8],"analytical":[9],"validations":[10],"for":[11,56],"single":[12],"AAV":[13,36,50,61],"serotypes.":[14],"This":[15],"approach":[16],"would":[17],"allow":[18],"sponsors":[19],"to":[20],"establish":[21],"clinically":[22],"meaningful":[23],"cutoffs":[24,42],"through":[25],"adaptive":[26],"clinical":[27,44],"trials":[28],"that":[29],"progressively":[30],"include":[31],"patients":[32,57],"escalating":[34],"pre-existing":[35,60],"Ab":[37,62],"levels":[38],"by":[39],"implementing":[40],"multiple":[41],"during":[43],"development.":[45],"These":[46],"measures":[47],"could":[48],"expedite":[49],"GT":[51],"development":[52],"while":[53],"expanding":[54],"access":[55],"elevated":[59],"levels.":[63]},"counts_by_year":[],"updated_date":"2026-11-09T13:22:03.289127","created_date":"2026-10-09T00:00:00"}
