Oligonucleotide APIs
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I-Inclisiran sodium
I-Inclisiran sodium API (i-Active Pharmaceutical Ingredient) ifundwa ngokuyinhloko kwintsimi ye-RNA interference (RNAi) kunye ne-cardiovascular therapeutics. Njenge-siRNA ephindwe kabini ejolise kwi-PCSK9 gene, isetyenziselwa uphando lwangaphambili kunye nophando lweklinikhi ukuvavanya izicwangciso zexesha elide zokuthulisa imfuza yokunciphisa i-LDL-C (i-low-density lipoprotein cholesterol). Ikwasebenza njengekhompawundi yemodeli yokuphanda ngeenkqubo zokuhanjiswa kwe-siRNA, uzinzo, kunye nonyango lwe-RNA olujoliswe esibindini.
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I-Donidalorsen
I-Donidalorsen API yi-oligonucleotide ye-antisense (ASO) ephantsi kophando lonyango lwe-angioedema ye-hereditary (HAE) kunye neemeko ezinxulumene nokuvuvukala. Kufundwa kumxholo wonyango olujoliswe kwi-RNA, ejolise ekunciphiseni ukubonakaliswa kweplasma prekallikrein(KLKB1 mRNA). Abaphandi basebenzisa i-Donidalorsen ukuphonononga iindlela zokuthulisa imfuza, i-pharmacokinetics exhomekeke kwidosi, kunye nolawulo lwexesha elide lokudumba kwe-bradykinin-mediated.
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IFitusiran
I-Fitusiran API yi-RNA encinci ephazamisayo (i-siRNA) ephandwe ngokukodwa kwintsimi ye-hemophilia kunye nokuphazamiseka kwe-coagulation. Ijolise kwii-antithrombin (AT okanye i-SERPINC1)gene esibindini ukunciphisa imveliso ye-antithrombin. Abaphandi basebenzisa i-Fitusiran ukuphonononga iindlela zokuphazamiseka kwe-RNA (i-RNAi), ukuthuliswa kofuzo oluthile lwesibindi, kunye nezicwangciso zonyango ezinoveli zokubuyisela i-coagulation kwi-hemophilia A kunye ne-B izigulane, kunye okanye ngaphandle kwe-inhibitors.
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Givosiran
I-Givosiran API yi-RNA encinci ephazamisayo (i-siRNA) efundela unyango lwe-acute hepatic porphyria (AHP). Ijolise ngokukodwa kwiI-ALAS1gene (i-aminolevulinic acid synthase 1), ebandakanyeka kwi-heme biosynthesis pathway. Abaphandi basebenzisa i-Givosiran ukuphanda ukungenelela kwe-RNA (RNAi)-based therapies, ukuthulisa i-gene ejoliswe kwisibindi, kunye nokuguqulwa kweendlela ze-metabolic ezibandakanyekayo kwi-porphyria kunye nokuphazamiseka kwemfuza okuhambelanayo.
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Plozasiran
I-Plozasiran API yi-RNA encinci ephazamisayo (i-siRNA) ephuhliselwe unyango lwe-hypertriglyceridemia kunye ne-cardiovascular and metabolic disorders. Ijolise kwiAPOC3gene, efaka i-apolipoprotein C-III, umlawuli oyintloko we-triglyceride metabolism. Kuphando, i-Plozasiran isetyenziselwa ukufunda izicwangciso ze-RNAi-based based lipid-downing, i-gene-sincing specificity, kunye nonyango oluthatha ixesha elide kwiimeko ezifana ne-familial chylomicronemia syndrome (FCS) kunye ne-dyslipidemia edibeneyo.
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Zilebesiran
I-Zilebesiran API yi-RNA encinci ephazamisayo yophando (i-siRNA) ephuhliselwe unyango lwe-hypertension. Ijolise kwiAGTgene, efaka i-angiotensinogen-inxalenye ephambili yenkqubo ye-renin-angiotensin-aldosterone (RAAS). Kuphando, i-Zilebesiran isetyenziselwa ukufunda iindlela zokuthulisa i-gene yokulawula uxinzelelo lwegazi ixesha elide, iteknoloji yokuhambisa i-RNAi, kunye nendima ebanzi yendlela ye-RAAS kwi-cardiovascular and renal disease.