I-Oligonucleotide APIs
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I-Inclisiran sodium
I-Inclisiran sodium API (I-Active Pharmaceutical Ingredient) ifundwa ngokuyinhloko emkhakheni we-RNA interference (RNAi) kanye nokwelapha kwenhliziyo nemithambo yegazi. Njenge-siRNA enemicu ephindwe kabili eqondise isakhi sofuzo se-PCSK9, isetshenziswa ocwaningweni lwangaphambi kwezempilo nolwezempilo ukuze kuhlolwe amasu asebenza isikhathi eside okuthulisa ufuzo okwehlisa i-LDL-C (low-density lipoprotein cholesterol). Iphinde isebenze njengenhlanganisela yemodeli yokuphenya izinhlelo zokulethwa kwe-siRNA, ukuzinza, kanye nemithi yokwelapha ye-RNA eqondiswe esibindini.
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I-Donidalorsen
I-Donilorsen API iyi-antisense oligonucleotide (ASO) ephenywayo yokwelashwa kwe-angioedema yofuzo (HAE) kanye nezimo zokuvuvukala ezihlobene. Kufundwa kumongo wezindlela zokwelapha eziqondiswe ku-RNA, okuhloswe ngazo ukunciphisa ukubonakaliswa kwei-plasma prekallikrein(KLKB1 mRNA). Abacwaningi basebenzisa i-Donidalorsen ukuhlola izindlela zokuthulisa izakhi zofuzo, i-pharmacokinetics encike kumthamo, kanye nokulawulwa kwesikhathi eside kokuvuvukala okuhambisana ne-bradykinin.
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I-Fitusiran
I-Fitusiran API iyi-RNA encane ephazamisayo (siRNA) ephenywa ngokuyinhloko emkhakheni we-hemophilia kanye nokuphazamiseka kwe-coagulation. Ihlose ii-antithrombin (AT noma i-SERPINC1)isakhi sofuzo esibindini ukunciphisa ukukhiqizwa kwe-antithrombin. Abacwaningi basebenzisa i-Fitusiran ukuhlola izindlela zokuphazanyiswa kwe-RNA (i-RNAi), ukuthulisa izakhi zofuzo ezithize esibindini, kanye namasu anoveli okwelapha okubuyisela ukulinganisa ukuhlangana ezigulini ze-hemophilia A no-B, ezinama-inhibitors noma ngaphandle kwazo.
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Givosiran
I-Givosiran API iyi-RNA encane ephazamisayo yokwenziwa (siRNA) efundelwe ukwelashwa kwe-acute hepatic porphyria (AHP). Iqondise ngokuqondile i-I-ALAS1isakhi sofuzo (i-aminolevulinic acid synthase 1), ehilelekile endleleni ye-heme biosynthesis. Abacwaningi basebenzisa i-Givosiran ukuze baphenye izindlela zokwelapha ezisekelwe ku-RNA (RNAi), ukuthulisa izakhi zofuzo eziqondiswe esibindini, kanye nokuguqulwa kwemizila ye-metabolic ehilelekile ku-porphyria kanye nokuphazamiseka kofuzo okuhlobene.
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I-Plozasiran
I-Plozasiran API i-RNA encane ephazamisayo yokwenziwa (i-siRNA) eyenzelwe ukwelapha i-hypertriglyceridemia kanye nokuphazamiseka kwenhliziyo nemithambo yegazi okuhlobene. Ihlose iI-APOC3I-gene, ehlanganisa i-apolipoprotein C-III, isilawuli esiyinhloko se-triglyceride metabolism. Ocwaningweni, i-Plozasiran isetshenziselwa ukutadisha amasu okwehlisa i-lipid asuselwa ku-RNAi, ukucaciswa kokuthulisa izakhi zofuzo, kanye nokwelashwa okuthatha isikhathi eside ezimeni ezifana ne-familia chylomicronemia syndrome (FCS) kanye ne-dyslipidemia exubile.
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Zilebesiran
I-Zilebesiran API iyi-RNA encane ephazamisayo yophenyo (siRNA) eyenzelwe ukwelapha umfutho wegazi ophakeme. Ihlose iI-AGTisakhi sofuzo, esihlanganisa i-angiotensinogen—ingxenye eyinhloko yesistimu ye-renin-angiotensin-aldosterone (RAAS). Ocwaningweni, i-Zilebesiran isetshenziselwa ukutadisha izindlela zokuthulisa izakhi zofuzo zokulawula umfutho wegazi wesikhathi eside, ubuchwepheshe bokulethwa kwe-RNAi, kanye nendima ebanzi yomzila we-RAAS ezifweni zenhliziyo nemithambo yegazi.