{"id":"https://openalex.org/W7119984027","doi":"https://doi.org/10.1038/s41433-025-04202-5","title":"A novel modifier gene therapy to treat Stargardt disease: Phase 1 GARDian1 Trial Insights","display_name":"A novel modifier gene therapy to treat Stargardt disease: Phase 1 GARDian1 Trial Insights","publication_year":2026,"publication_date":"2026-01-10","ids":{"openalex":"https://openalex.org/W7119984027","doi":"https://doi.org/10.1038/s41433-025-04202-5","pmid":"https://pubmed.ncbi.nlm.nih.gov/41520062","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/12881397"},"language":"en","primary_location":{"id":"doi:10.1038/s41433-025-04202-5","doi":"https://doi.org/10.1038/s41433-025-04202-5","is_oa":true,"landing_page_url":"https://doi.org/10.1038/s41433-025-04202-5","pdf_url":"https://www.nature.com/articles/s41433-025-04202-5.pdf","source":{"id":"https://openalex.org/S167047176","display_name":"Eye","issn_l":"0078-5334","issn":["0078-5334","0950-222X","1476-5454"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310319965","host_organization_name":"Springer Nature","host_organization_lineage":["https://openalex.org/P4310319965"],"host_organization_lineage_names":["Springer Nature"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Eye","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"https://www.nature.com/articles/s41433-025-04202-5.pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5035499225","display_name":"Arshad M. Khanani","orcid":"https://orcid.org/0000-0001-8928-5599","observed_orcids":["https://orcid.org/0000-0001-8928-5599"]},"institutions":[{"id":"https://openalex.org/I134113660","display_name":"University of Nevada, Reno","ror":"https://ror.org/01keh0577","country_code":"US","type":"education","lineage":["https://openalex.org/I134113660"]},{"id":"https://openalex.org/I4210161514","display_name":"Sierra Eye Associates","ror":"https://ror.org/04v77c541","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210161514"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Arshad M. Khanani","raw_affiliation_strings":["Sierra Eye Associates, Reno, NV, USA. arshad.khanani@gmail.com","University of Nevada, Reno School of Medicine, Reno, NV, USA. arshad.khanani@gmail.com"],"raw_orcid":"https://orcid.org/0000-0001-8928-5599","affiliations":[{"raw_affiliation_string":"Sierra Eye Associates, Reno, NV, USA. arshad.khanani@gmail.com","institution_ids":["https://openalex.org/I4210161514"]},{"raw_affiliation_string":"University of Nevada, Reno School of Medicine, Reno, NV, USA. arshad.khanani@gmail.com","institution_ids":["https://openalex.org/I134113660"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5078534469","display_name":"Lejla Mutapcic Vajzovic","orcid":"https://orcid.org/0000-0002-4469-6834","observed_orcids":["https://orcid.org/0000-0002-4469-6834"]},"institutions":[{"id":"https://openalex.org/I170897317","display_name":"Duke University","ror":"https://ror.org/00py81415","country_code":"US","type":"education","lineage":["https://openalex.org/I170897317"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Lejla Vajzovic","raw_affiliation_strings":["Duke University, Durham, NC, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Duke University, Durham, NC, USA","institution_ids":["https://openalex.org/I170897317"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5012564184","display_name":"Benjamin Bakall","orcid":"https://orcid.org/0000-0003-1143-3007","observed_orcids":["https://orcid.org/0000-0003-1143-3007"]},"institutions":[{"id":"https://openalex.org/I4405259572","display_name":"University of Arizona College of Medicine- Phoenix","ror":"https://ror.org/02drhvq25","country_code":"US","type":"education","lineage":["https://openalex.org/I138006243","https://openalex.org/I4405259572"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Benjamin Bakall","raw_affiliation_strings":["Associated Retina Consultants, Phoenix, AZ, USA","University of Arizona, College of Medicine-Phoenix, Phoenix, AZ, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Associated Retina Consultants, Phoenix, AZ, USA","institution_ids":[]},{"raw_affiliation_string":"University of Arizona, College of Medicine-Phoenix, Phoenix, AZ, USA","institution_ids":["https://openalex.org/I4405259572"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5012716024","display_name":"Venkata Ramana Murthy Chavali","orcid":"https://orcid.org/0000-0003-3439-3490","observed_orcids":["https://orcid.org/0000-0003-3439-3490"]},"institutions":[{"id":"https://openalex.org/I4405267114","display_name":"Ocugen, Inc. (United States)","ror":"https://ror.org/0194jyj31","country_code":"US","type":"company","lineage":["https://openalex.org/I4405267114"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Venkata Ramana Murthy Chavali","raw_affiliation_strings":["Ocugen, Inc., Malvern, PA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Ocugen, Inc., Malvern, PA, USA","institution_ids":["https://openalex.org/I4405267114"]}]},{"author_position":"last","author":{"id":null,"display_name":"Huma Qamar","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4405267114","display_name":"Ocugen, Inc. (United States)","ror":"https://ror.org/0194jyj31","country_code":"US","type":"company","lineage":["https://openalex.org/I4405267114"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Huma Qamar","raw_affiliation_strings":["Ocugen, Inc., Malvern, PA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Ocugen, Inc., Malvern, PA, USA","institution_ids":["https://openalex.org/I4405267114"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":5,"corresponding_author_ids":["https://openalex.org/A5035499225"],"corresponding_institution_ids":["https://openalex.org/I134113660","https://openalex.org/I4210161514"],"apc_list":{"value":3590,"currency":"USD","value_usd":3590},"apc_paid":{"value":3590,"currency":"USD","value_usd":3590},"fwci":13.8697,"has_fulltext":true,"cited_by_count":3,"citation_normalized_percentile":{"value":0.98110545,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":96,"max":97},"biblio":{"volume":"40","issue":"3","first_page":"429","last_page":"430"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10691","display_name":"Retinal Development and Disorders","score":0.9854000210762024,"subfield":{"id":"https://openalex.org/subfields/2731","display_name":"Ophthalmology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},"topics":[{"id":"https://openalex.org/T10691","display_name":"Retinal Development and Disorders","score":0.9854000210762024,"subfield":{"id":"https://openalex.org/subfields/2731","display_name":"Ophthalmology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T10170","display_name":"Retinal Diseases and Treatments","score":0.010499999858438969,"subfield":{"id":"https://openalex.org/subfields/2731","display_name":"Ophthalmology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.000699999975040555,"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"}}],"keywords":[{"id":"https://openalex.org/keywords/abca4-mutations","display_name":"ABCA4 mutations","score":0.8190000057220459},{"id":"https://openalex.org/keywords/retinal-pigment-epithelium","display_name":"retinal pigment epithelium","score":0.8700000047683716},{"id":"https://openalex.org/keywords/macular-dystrophy","display_name":"macular dystrophy","score":0.7829999923706055},{"id":"https://openalex.org/keywords/subretinal-gene-therapy","display_name":"subretinal gene therapy","score":0.7379999756813049},{"id":"https://openalex.org/keywords/safety-and-tolerability","display_name":"safety and tolerability","score":0.7390000224113464}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.6430000066757202},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.6305999755859375},{"id":"https://openalex.org/C535046627","wikidata":"https://www.wikidata.org/wiki/Q30612","display_name":"Clinical trial","level":2,"score":0.4124000072479248},{"id":"https://openalex.org/C44280652","wikidata":"https://www.wikidata.org/wiki/Q104837","display_name":"Phase (matter)","level":2,"score":0.4025999903678894},{"id":"https://openalex.org/C2780604041","wikidata":"https://www.wikidata.org/wiki/Q1317319","display_name":"Stargardt disease","level":3,"score":0.3433000147342682},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.32989999651908875},{"id":"https://openalex.org/C31760486","wikidata":"https://www.wikidata.org/wiki/Q7180990","display_name":"Phases of clinical research","level":3,"score":0.3167000114917755},{"id":"https://openalex.org/C502942594","wikidata":"https://www.wikidata.org/wiki/Q3421914","display_name":"Cancer research","level":1,"score":0.3165000081062317},{"id":"https://openalex.org/C2779473830","wikidata":"https://www.wikidata.org/wiki/Q1540899","display_name":"MEDLINE","level":2,"score":0.2919999957084656},{"id":"https://openalex.org/C60644358","wikidata":"https://www.wikidata.org/wiki/Q128570","display_name":"Bioinformatics","level":1,"score":0.28600001335144043}],"mesh":[],"locations_count":4,"locations":[{"id":"doi:10.1038/s41433-025-04202-5","doi":"https://doi.org/10.1038/s41433-025-04202-5","is_oa":true,"landing_page_url":"https://doi.org/10.1038/s41433-025-04202-5","pdf_url":"https://www.nature.com/articles/s41433-025-04202-5.pdf","source":{"id":"https://openalex.org/S167047176","display_name":"Eye","issn_l":"0078-5334","issn":["0078-5334","0950-222X","1476-5454"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310319965","host_organization_name":"Springer Nature","host_organization_lineage":["https://openalex.org/P4310319965"],"host_organization_lineage_names":["Springer Nature"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Eye","raw_type":"journal-article"},{"id":"pmh:oai:europepmc.org:11689020","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/12881397","pdf_url":null,"source":{"id":"https://openalex.org/S4306400806","display_name":"Europe PMC (PubMed Central)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1303153112","host_organization_name":"European Bioinformatics Institute","host_organization_lineage":["https://openalex.org/I1303153112"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":null,"raw_type":"Text"},{"id":"pmid:41520062","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/41520062","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":"Eye (London, England)","raw_type":"Journal Article"},{"id":"pmh:oai:pubmedcentral.nih.gov:12881397","doi":null,"is_oa":true,"landing_page_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC12881397/","pdf_url":null,"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","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Eye (Lond)","raw_type":"Text"}],"best_oa_location":{"id":"doi:10.1038/s41433-025-04202-5","doi":"https://doi.org/10.1038/s41433-025-04202-5","is_oa":true,"landing_page_url":"https://doi.org/10.1038/s41433-025-04202-5","pdf_url":"https://www.nature.com/articles/s41433-025-04202-5.pdf","source":{"id":"https://openalex.org/S167047176","display_name":"Eye","issn_l":"0078-5334","issn":["0078-5334","0950-222X","1476-5454"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310319965","host_organization_name":"Springer Nature","host_organization_lineage":["https://openalex.org/P4310319965"],"host_organization_lineage_names":["Springer Nature"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Eye","raw_type":"journal-article"},"sustainable_development_goals":[{"score":0.98580002784729,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[{"id":"https://openalex.org/study-designs/clinical-trial","display_name":"Clinical Trial"}],"awards":[],"funders":[],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W7119984027.pdf","grobid_xml":"https://content.openalex.org/works/W7119984027.grobid-xml"},"referenced_works_count":5,"referenced_works":["https://openalex.org/W2617433841","https://openalex.org/W2808419704","https://openalex.org/W2808848006","https://openalex.org/W4416087492","https://openalex.org/W4416701854"],"related_works":["https://openalex.org/W2086542140","https://openalex.org/W6939447072","https://openalex.org/W4387119462","https://openalex.org/W3091775442","https://openalex.org/W4388488432","https://openalex.org/W6944810876","https://openalex.org/W254705429","https://openalex.org/W4412527005","https://openalex.org/W4401011647","https://openalex.org/W7221781823"],"abstract_inverted_index":{"Stargardt":[0],"disease":[1],"type":[2],"1":[3,27,83],"(STGD1)":[4],"is":[5,64],"an":[6],"orphan":[7],"inherited":[8],"macular":[9],"dystrophy":[10],"affecting":[11],"over":[12],"100,000":[13],"individuals":[14],"in":[15,22,99],"the":[16],"US":[17],"and":[18,43,69,89],"EU,":[19],"commonly":[20],"presenting":[21],"childhood":[23],"or":[24],"adolescence":[25],"[":[26,48],"].":[28,50],"Biallelic":[29],"ABCA4":[30,78],"mutations":[31],"lead":[32],"to":[33,66],"toxic":[34],"bisretinoid":[35],"lipofuscin":[36],"accumulation,":[37],"retinal":[38,74],"pigment":[39],"epithelium":[40],"(RPE)":[41],"atrophy,":[42],"progressive":[44],"central":[45],"vision":[46],"loss":[47],"2":[49],"With":[51],"no":[52],"approved":[53],"treatments,":[54],"STGD1":[55],"remains":[56],"a":[57,93],"significant":[58],"unmet":[59],"medical":[60],"need.":[61],"OCU410ST":[62,98],"(AAV5-hRORA)":[63],"designed":[65],"modulate":[67],"RPE":[68],"photoreceptor":[70],"homeostasis,":[71],"thereby":[72],"preserving":[73],"integrity":[75],"independent":[76],"of":[77,92,97],"genotype.":[79],"This":[80],"first-in-human":[81],"phase":[82],"trial":[84],"(NCT05956626)":[85],"evaluated":[86],"safety,":[87],"tolerability,":[88],"exploratory":[90],"efficacy":[91],"single":[94],"subretinal":[95],"dose":[96],"adults":[100],"with":[101],"advanced":[102],"STGD1.":[103]},"counts_by_year":[{"year":2026,"cited_by_count":3}],"updated_date":"2026-10-28T10:33:24.094632","created_date":"2026-01-10T00:00:00"}