{"id":"https://openalex.org/W3092190815","doi":"https://doi.org/10.1007/s40262-020-00940-9","title":"Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria","display_name":"Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria","publication_year":2020,"publication_date":"2020-10-13","ids":{"openalex":"https://openalex.org/W3092190815","doi":"https://doi.org/10.1007/s40262-020-00940-9","mag":"3092190815","pmid":"https://pubmed.ncbi.nlm.nih.gov/33047216","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/9203406"},"language":"en","primary_location":{"id":"doi:10.1007/s40262-020-00940-9","doi":"https://doi.org/10.1007/s40262-020-00940-9","is_oa":true,"landing_page_url":"https://doi.org/10.1007/s40262-020-00940-9","pdf_url":"https://link.springer.com/content/pdf/10.1007/s40262-020-00940-9.pdf","source":{"id":"https://openalex.org/S69290020","display_name":"Clinical Pharmacokinetics","issn_l":"0312-5963","issn":["0312-5963","1179-1926"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-b","doyens","jufo-2","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320330","host_organization_name":"Adis, Springer Healthcare","host_organization_lineage":["https://openalex.org/P4310320330","https://openalex.org/P4310319965"],"host_organization_lineage_names":["Adis, Springer Healthcare","Springer Nature"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical Pharmacokinetics","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"https://link.springer.com/content/pdf/10.1007/s40262-020-00940-9.pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5026326021","display_name":"Prajakta S. Badri","orcid":"https://orcid.org/0000-0003-1248-616X","observed_orcids":["https://orcid.org/0000-0003-1248-616X"]},"institutions":[{"id":"https://openalex.org/I145311141","display_name":"Alnylam Pharmaceuticals (United States)","ror":"https://ror.org/00thr3w71","country_code":"US","type":"company","lineage":["https://openalex.org/I145311141"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Prajakta Badri","raw_affiliation_strings":["Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA. pbadri@alnylam.com","Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA"],"raw_orcid":"https://orcid.org/0000-0003-1248-616X","affiliations":[{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA. pbadri@alnylam.com","institution_ids":["https://openalex.org/I145311141"]},{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA","institution_ids":["https://openalex.org/I145311141"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5113819936","display_name":"Xuemin Jiang","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210092565","display_name":"Agios Pharmaceuticals (United States)","ror":"https://ror.org/002x06r10","country_code":"US","type":"company","lineage":["https://openalex.org/I4210092565"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Xuemin Jiang","raw_affiliation_strings":["Agios Pharmaceuticals, Cambridge, MA, USA","Agios Pharmaceuticals, Cambridge, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Agios Pharmaceuticals, Cambridge, MA, USA","institution_ids":["https://openalex.org/I4210092565"]},{"raw_affiliation_string":"Agios Pharmaceuticals, Cambridge, USA","institution_ids":["https://openalex.org/I4210092565"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5044854100","display_name":"Anna Borodovsky","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I145311141","display_name":"Alnylam Pharmaceuticals (United States)","ror":"https://ror.org/00thr3w71","country_code":"US","type":"company","lineage":["https://openalex.org/I145311141"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Anna Borodovsky","raw_affiliation_strings":["Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","institution_ids":["https://openalex.org/I145311141"]},{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA","institution_ids":["https://openalex.org/I145311141"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5037972566","display_name":"Nader Najafian","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210125492","display_name":"Alexion Pharmaceuticals (United States)","ror":"https://ror.org/031ywxc85","country_code":"US","type":"company","lineage":["https://openalex.org/I4210125492"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Nader Najafian","raw_affiliation_strings":["Alexion Pharmaceuticals, Boston, MA, USA","Alexion Pharmaceuticals, Boston, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Alexion Pharmaceuticals, Boston, MA, USA","institution_ids":["https://openalex.org/I4210125492"]},{"raw_affiliation_string":"Alexion Pharmaceuticals, Boston, USA","institution_ids":["https://openalex.org/I4210125492"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5101950148","display_name":"Jae Bum Kim","orcid":"https://orcid.org/0000-0001-6942-4037","observed_orcids":["https://orcid.org/0000-0001-6942-4037"]},"institutions":[{"id":"https://openalex.org/I145311141","display_name":"Alnylam Pharmaceuticals (United States)","ror":"https://ror.org/00thr3w71","country_code":"US","type":"company","lineage":["https://openalex.org/I145311141"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jae Kim","raw_affiliation_strings":["Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","institution_ids":["https://openalex.org/I145311141"]},{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA","institution_ids":["https://openalex.org/I145311141"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5057249319","display_name":"Valerie A. Clausen","orcid":"https://orcid.org/0009-0006-2901-9540","observed_orcids":["https://orcid.org/0009-0006-2901-9540"]},"institutions":[{"id":"https://openalex.org/I145311141","display_name":"Alnylam Pharmaceuticals (United States)","ror":"https://ror.org/00thr3w71","country_code":"US","type":"company","lineage":["https://openalex.org/I145311141"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Valerie A. Clausen","raw_affiliation_strings":["Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","institution_ids":["https://openalex.org/I145311141"]},{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA","institution_ids":["https://openalex.org/I145311141"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Varun Goel","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I145311141","display_name":"Alnylam Pharmaceuticals (United States)","ror":"https://ror.org/00thr3w71","country_code":"US","type":"company","lineage":["https://openalex.org/I145311141"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Varun Goel","raw_affiliation_strings":["Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","institution_ids":["https://openalex.org/I145311141"]},{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA","institution_ids":["https://openalex.org/I145311141"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5086099648","display_name":"Bahru A. Habtemariam","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I145311141","display_name":"Alnylam Pharmaceuticals (United States)","ror":"https://ror.org/00thr3w71","country_code":"US","type":"company","lineage":["https://openalex.org/I145311141"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Bahru Habtemariam","raw_affiliation_strings":["Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","institution_ids":["https://openalex.org/I145311141"]},{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA","institution_ids":["https://openalex.org/I145311141"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5083919448","display_name":"Gabriel J. Robbie","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I145311141","display_name":"Alnylam Pharmaceuticals (United States)","ror":"https://ror.org/00thr3w71","country_code":"US","type":"company","lineage":["https://openalex.org/I145311141"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Gabriel J. Robbie","raw_affiliation_strings":["Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, 101 Main Street, Cambridge, MA, 02142, USA","institution_ids":["https://openalex.org/I145311141"]},{"raw_affiliation_string":"Clinical Pharmacology and Pharmacometrics, Alnylam Pharmaceuticals, Cambridge, USA","institution_ids":["https://openalex.org/I145311141"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":3,"corresponding_author_ids":["https://openalex.org/A5026326021"],"corresponding_institution_ids":["https://openalex.org/I145311141"],"apc_list":{"value":3860,"currency":"USD","value_usd":3860},"apc_paid":{"value":3860,"currency":"USD","value_usd":3860},"fwci":3.6716,"has_fulltext":true,"cited_by_count":79,"citation_normalized_percentile":{"value":0.94917706,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":98,"max":100},"biblio":{"volume":"60","issue":"3","first_page":"365","last_page":"378"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10993","display_name":"Complement system in diseases","score":0.9961000084877014,"subfield":{"id":"https://openalex.org/subfields/2403","display_name":"Immunology"},"field":{"id":"https://openalex.org/fields/24","display_name":"Immunology and Microbiology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},"topics":[{"id":"https://openalex.org/T10993","display_name":"Complement system in diseases","score":0.9961000084877014,"subfield":{"id":"https://openalex.org/subfields/2403","display_name":"Immunology"},"field":{"id":"https://openalex.org/fields/24","display_name":"Immunology and Microbiology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10725","display_name":"RNA Interference and Gene Delivery","score":0.00139999995008111,"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/T11280","display_name":"Hemophilia Treatment and Research","score":9.999999747378752e-05,"subfield":{"id":"https://openalex.org/subfields/2720","display_name":"Hematology"},"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/paroxysmal-nocturnal-hemoglobinuria","display_name":"paroxysmal nocturnal hemoglobinuria","score":0.9369999766349792},{"id":"https://openalex.org/keywords/complement-component-5","display_name":"complement component 5","score":0.8920000195503235},{"id":"https://openalex.org/keywords/rna-interference","display_name":"RNA interference","score":0.49799999594688416},{"id":"https://openalex.org/keywords/galnac-conjugation","display_name":"GalNAc conjugation","score":0.7260000109672546},{"id":"https://openalex.org/keywords/pharmacokinetics","display_name":"pharmacokinetics","score":0.8970000147819519},{"id":"https://openalex.org/keywords/pharmacodynamics","display_name":"pharmacodynamics","score":0.996999979019165},{"id":"https://openalex.org/keywords/eculizumab","display_name":"eculizumab","score":0.8270000219345093},{"id":"https://openalex.org/keywords/dose-selection","display_name":"dose selection","score":0.5979999899864197},{"id":"https://openalex.org/keywords/dose-proportionality","display_name":"dose proportionality","score":0.43799999356269836},{"id":"https://openalex.org/keywords/sigmoid-emax-model","display_name":"sigmoid Emax model","score":0.6050000190734863},{"id":"https://openalex.org/keywords/complement-mediated-disease","display_name":"complement-mediated disease","score":0.42800000309944153},{"id":"https://openalex.org/keywords/healthy-volunteers","display_name":"healthy volunteers","score":0.5609999895095825},{"id":"https://openalex.org/keywords/japanese-subjects","display_name":"Japanese subjects","score":0.5289999842643738},{"id":"https://openalex.org/keywords/safety-and-tolerability","display_name":"safety and tolerability","score":0.6859999895095825}],"concepts":[{"id":"https://openalex.org/C2777991916","wikidata":"https://www.wikidata.org/wiki/Q421617","display_name":"Eculizumab","level":4,"score":0.8122949600219727},{"id":"https://openalex.org/C2777346216","wikidata":"https://www.wikidata.org/wiki/Q1479494","display_name":"Paroxysmal nocturnal hemoglobinuria","level":2,"score":0.8102328181266785},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.8002763986587524},{"id":"https://openalex.org/C112705442","wikidata":"https://www.wikidata.org/wiki/Q323936","display_name":"Pharmacokinetics","level":2,"score":0.6817474365234375},{"id":"https://openalex.org/C111113717","wikidata":"https://www.wikidata.org/wiki/Q725307","display_name":"Pharmacodynamics","level":3,"score":0.6436685919761658},{"id":"https://openalex.org/C2778375690","wikidata":"https://www.wikidata.org/wiki/Q7814214","display_name":"Tolerability","level":3,"score":0.5922744274139404},{"id":"https://openalex.org/C98274493","wikidata":"https://www.wikidata.org/wiki/Q128406","display_name":"Pharmacology","level":1,"score":0.529022216796875},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.49108242988586426},{"id":"https://openalex.org/C27081682","wikidata":"https://www.wikidata.org/wiki/Q269829","display_name":"Placebo","level":3,"score":0.4831799566745758},{"id":"https://openalex.org/C90924648","wikidata":"https://www.wikidata.org/wiki/Q120569","display_name":"Gastroenterology","level":1,"score":0.41575756669044495},{"id":"https://openalex.org/C111684460","wikidata":"https://www.wikidata.org/wiki/Q334848","display_name":"Complement system","level":3,"score":0.3014715015888214},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.22938483953475952},{"id":"https://openalex.org/C197934379","wikidata":"https://www.wikidata.org/wiki/Q2047938","display_name":"Adverse effect","level":2,"score":0.19212469458580017},{"id":"https://openalex.org/C159654299","wikidata":"https://www.wikidata.org/wiki/Q79460","display_name":"Antibody","level":2,"score":0.1279909312725067},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.07683435082435608},{"id":"https://openalex.org/C204787440","wikidata":"https://www.wikidata.org/wiki/Q188504","display_name":"Alternative medicine","level":2,"score":0.0}],"mesh":[{"descriptor_ui":"D000067492","descriptor_name":"RNAi Therapeutics","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D003182","descriptor_name":"Complement C5","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":true},{"descriptor_ui":"D006457","descriptor_name":"Hemoglobinuria, Paroxysmal","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D034622","descriptor_name":"RNA Interference","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D064368","descriptor_name":"Healthy Volunteers","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":3,"locations":[{"id":"doi:10.1007/s40262-020-00940-9","doi":"https://doi.org/10.1007/s40262-020-00940-9","is_oa":true,"landing_page_url":"https://doi.org/10.1007/s40262-020-00940-9","pdf_url":"https://link.springer.com/content/pdf/10.1007/s40262-020-00940-9.pdf","source":{"id":"https://openalex.org/S69290020","display_name":"Clinical Pharmacokinetics","issn_l":"0312-5963","issn":["0312-5963","1179-1926"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-b","doyens","jufo-2","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320330","host_organization_name":"Adis, Springer Healthcare","host_organization_lineage":["https://openalex.org/P4310320330","https://openalex.org/P4310319965"],"host_organization_lineage_names":["Adis, Springer Healthcare","Springer Nature"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical Pharmacokinetics","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:9203406","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/9203406","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-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clin Pharmacokinet","raw_type":"Text"},{"id":"pmid:33047216","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/33047216","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":"Clinical pharmacokinetics","raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.1007/s40262-020-00940-9","doi":"https://doi.org/10.1007/s40262-020-00940-9","is_oa":true,"landing_page_url":"https://doi.org/10.1007/s40262-020-00940-9","pdf_url":"https://link.springer.com/content/pdf/10.1007/s40262-020-00940-9.pdf","source":{"id":"https://openalex.org/S69290020","display_name":"Clinical Pharmacokinetics","issn_l":"0312-5963","issn":["0312-5963","1179-1926"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-b","doyens","jufo-2","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320330","host_organization_name":"Adis, Springer Healthcare","host_organization_lineage":["https://openalex.org/P4310320330","https://openalex.org/P4310319965"],"host_organization_lineage_names":["Adis, Springer Healthcare","Springer Nature"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical Pharmacokinetics","raw_type":"journal-article"},"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being","score":1.0}],"study_designs":[{"id":"https://openalex.org/study-designs/clinical-trial","display_name":"Clinical Trial"}],"awards":[],"funders":[{"id":"https://openalex.org/F4320309056","display_name":"Alnylam Pharmaceuticals","ror":"https://ror.org/00thr3w71"}],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W3092190815.pdf","grobid_xml":"https://content.openalex.org/works/W3092190815.grobid-xml"},"referenced_works_count":26,"referenced_works":["https://openalex.org/W1963924977","https://openalex.org/W1967317528","https://openalex.org/W1973771269","https://openalex.org/W1974915370","https://openalex.org/W1992365936","https://openalex.org/W1995181352","https://openalex.org/W2004265876","https://openalex.org/W2012668509","https://openalex.org/W2021905258","https://openalex.org/W2023377972","https://openalex.org/W2066580298","https://openalex.org/W2100451532","https://openalex.org/W2130696459","https://openalex.org/W2142051176","https://openalex.org/W2152757679","https://openalex.org/W2195444372","https://openalex.org/W2409156875","https://openalex.org/W2568306867","https://openalex.org/W2595668953","https://openalex.org/W2753865234","https://openalex.org/W2756425540","https://openalex.org/W2781906140","https://openalex.org/W2802881274","https://openalex.org/W2914314726","https://openalex.org/W2944262545","https://openalex.org/W3026834368"],"related_works":["https://openalex.org/W4382139431","https://openalex.org/W4310161045","https://openalex.org/W4282960502","https://openalex.org/W4389231779","https://openalex.org/W3213100696","https://openalex.org/W4386071011","https://openalex.org/W4386052327","https://openalex.org/W4310114298","https://openalex.org/W4417012988","https://openalex.org/W4405044256"],"abstract_inverted_index":{"Cemdisiran,":[0],"an":[1,124,262],"N":[2],"-acetylgalactosamine":[3],"(GalNAc)":[4],"conjugated":[5],"RNA":[6],"interference":[7],"(RNAi)":[8],"therapeutic,":[9],"is":[10],"currently":[11],"under":[12],"development":[13],"for":[14,62,105,127,174,246,314,352],"the":[15,35,160,163,166,181,205,356,363],"treatment":[16],"of":[17,24,43,83,93,152,162,169,208,237,254,279,286,308,337,347,359,366,400,408,427],"complement-mediated":[18,430],"diseases":[19,431],"by":[20,261,332],"suppressing":[21],"liver":[22,367],"production":[23],"complement":[25],"5":[26],"(C5)":[27],"protein.":[28],"This":[29],"study":[30],"was":[31,120,193,350],"designed":[32],"to":[33,58,79,196,387],"evaluate":[34],"safety,":[36],"tolerability,":[37],"pharmacokinetics":[38],"(PK),":[39],"and":[40,48,176,216,244,251,282,293,299,302,305,372,381,392,414,423],"pharmacodynamics":[41],"(PD)":[42],"cemdisiran":[44,85,95,192,333,371,401,428],"in":[45,49,56,123,132,138,142,211,410,429,435],"healthy":[46],"subjects":[47,310],"patients":[50,136,145,171,304],"with":[51,140,180,233,317,320,341,362,437],"paroxysmal":[52],"nocturnal":[53],"hemoglobinuria":[54],"(PNH)":[55],"order":[57],"support":[59,424],"dose":[60,168,275,343,358],"selection":[61],"late-stage":[63],"clinical":[64],"trials.":[65],"Healthy":[66],"volunteers":[67],"(HVs;":[68],"n":[69,112],"=":[70,113],"32,":[71],"including":[72],"12":[73],"Japanese":[74,298],"subjects)":[75],"were":[76,147,172,231,295],"randomized":[77],"(3:1)":[78],"receive":[80],"single":[81,250,391],"doses":[82,92,151,253],"subcutaneous":[84,94],"(50\u2013900":[86],"mg)":[87,97],"or":[88,90,98,103,108,117,137,155,185,325,434],"placebo,":[89],"repeat":[91],"(100\u2013600":[96],"placebo":[99],"weekly,":[100],"biweekly,":[101],"weekly/biweekly,":[102],"weekly/monthly":[104],"5,":[106],"8,":[107],"13":[109,388],"weeks":[110],"(":[111,267],"24).":[114],"Cemdisiran":[115],"200":[116],"400":[118],"mg":[119,157,187,281],"administered":[121],"weekly":[122],"open-label":[125],"manner,":[126],"varying":[128],"durations,":[129],"as":[130,432],"monotherapy":[131,433],"three":[133,143],"eculizumab-na\u00efve":[134],"PNH":[135,144,303,328],"combination":[139,436],"eculizumab":[141,153,182],"who":[146],"receiving":[148],"stable":[149],"label":[150],"(900":[154],"1200":[156],"biweekly)":[158],"before":[159],"start":[161],"study.":[164],"After":[165],"last":[167,357],"cemdisiran,":[170],"followed":[173],"safety":[175,416],"ongoing":[177],"pharmacologic":[178],"effects":[179],"regimen":[183],"(600":[184],"900":[186],"every":[188],"month).":[189],"In":[190,327],"HVs,":[191],"rapidly":[194,377],"converted":[195],"a":[197,438],"major":[198],"active":[199],"metabolite,":[200],"AS(N-2)3\u2032-cemdisiran,":[201],"both":[202],"declining":[203],"below":[204],"lower":[206,342],"limit":[207],"quantification":[209],"(LLOQ)":[210],"plasma":[212,374],"within":[213,402],"48":[214,309],"h,":[215],"showing":[217,379],"minimal":[218],"renal":[219],"excretion.":[220],"AS(N-2)3\u2032-cemdisiran":[221,373],"exhibited":[222],"more":[223],"than":[224],"dose-proportional":[225],"PK.":[226],"The":[227,257,291,404],"C5":[228,238,284,330,339,383,439],"protein":[229,239],"reductions":[230],"dose-dependent,":[232],">":[234],"90%":[235],"reduction":[236,285],"beginning":[240],"on":[241,323],"days":[242],"21\u201328":[243],"maintained":[245,351,385],"10\u201313":[247],"months":[248,354,389],"following":[249,390],"biweekly":[252],"600":[255,289],"mg.":[256,290],"dose\u2013response":[258],"relationship,":[259],"described":[260],"inhibitory":[263],"sigmoid":[264],"maximum":[265,283],"effect":[266],"E":[268],"max":[269],")":[270,278],"model,":[271],"estimated":[272],"half-maximal":[273],"effective":[274,335],"(ED":[276],"50":[277],"14.0":[280],"99%":[287],"at":[288],"PK":[292,324],"PD":[294,406],"similar":[296],"between":[297],"non-Japanese":[300],"subjects,":[301],"HVs.":[306],"One":[307],"tested":[311],"transiently":[312],"positive":[313],"antidrug":[315],"antibody":[316],"low":[318,412],"titer,":[319],"no":[321],"impact":[322],"PD.":[326],"patients,":[329],"suppression":[331,384],"enabled":[334],"inhibition":[336],"residual":[338],"levels":[340],"and/or":[344],"dosing":[345,422],"frequency":[346],"eculizumab,":[348],"which":[349,395],"6\u201310":[353],"after":[355],"cemdisiran.":[360],"Consistent":[361],"PK/PD":[364],"properties":[365],"targeting":[368],"GalNac":[369],"conjugates,":[370],"concentrations":[375],"declined":[376],"while":[378],"rapid":[380],"robust":[382],"up":[386],"multiple":[393],"doses,":[394],"indicates":[396],"long":[397,405],"residence":[398],"times":[399],"hepatocytes.":[403],"duration":[407],"action":[409],"liver,":[411],"immunogenicity":[413],"acceptable":[415],"profiles":[417],"enables":[418],"low,":[419],"infrequent":[420],"SC":[421],"further":[425],"evaluation":[426],"inhibitor":[440],"antibody.":[441],"NCT02352493.":[442]},"counts_by_year":[{"year":2026,"cited_by_count":8},{"year":2025,"cited_by_count":12},{"year":2024,"cited_by_count":17},{"year":2023,"cited_by_count":18},{"year":2022,"cited_by_count":18},{"year":2021,"cited_by_count":6}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2020-10-15T00:00:00"}
