APIs na Oligonucleotide
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Ciwon sodium
Inclisiran sodium API (Active Pharmaceutical Ingredient) ana nazarinsa da farko a fagen tsoma bakin RNA (RNAi) da kuma hanyoyin kwantar da hankali na zuciya. A matsayin siRNA mai madauri biyu da ke niyya ga kwayar PCSK9, ana amfani da shi a cikin bincike na gaskiya da na asibiti don kimanta dabarun yin shiru na dogon aiki don rage LDL-C (cholesterol mai ƙarancin yawa). Hakanan yana aiki azaman abin ƙira don bincika tsarin isar da siRNA, kwanciyar hankali, da magungunan RNA mai niyya da hanta.
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Donidalorsen
Donidalorsen API wani maganin antisense oligonucleotide (ASO) ne a ƙarƙashin bincike don maganin angioedema na gado (HAE) da kuma yanayin kumburi masu alaƙa. Ana nazarin shi a cikin mahallin hanyoyin kwantar da hankali na RNA, da nufin rage bayyanar cututtukaplasma prekallikrein(KLKB1 mRNA). Masu bincike suna amfani da Donidalorsen don bincika hanyoyin yin shiru na kwayoyin halitta, magunguna masu dogara da kashi, da kuma kulawa na dogon lokaci na kumburi mai shiga tsakani na bradykinin.
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Fitusiran
Fitusiran API wani ƙaramin RNA ne mai tsoma baki (siRNA) wanda aka bincika da farko a fagen cutar haemophilia da rikicewar coagulation. Yana kaiwa hariAntithrombin (AT ko SERPINC1)kwayoyin halitta a cikin hanta don rage samar da antithrombin. Masu bincike suna amfani da Fitusiran don bincika hanyoyin tsangwama na RNA (RNAi), takamaiman ƙwayoyin halittar hanta, da sabbin dabarun warkewa don sake daidaita coagulation a cikin marasa lafiya na hemophilia A da B, tare da ko ba tare da masu hanawa ba.
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Givosiran
Givosiran API wani ƙaramin RNA ne mai shiga tsakani (siRNA) wanda aka yi nazari don maganin cututtukan hanta mai tsanani (AHP). Yana musamman hari daALAS1gene (aminolevulinic acid synthase 1), wanda ke da hannu a cikin hanyar heme biosynthesis. Masu bincike suna amfani da Givosiran don yin bincike kan tsoma bakin RNA (RNAi), hanyoyin kwantar da hankali na hanta da ke da niyya, da daidaita hanyoyin hanyoyin rayuwa da ke cikin porphyria da cututtukan ƙwayoyin cuta masu alaƙa.
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Plozasiran
Plozasiran API shine ƙaramin RNA (siRNA) na roba wanda aka haɓaka don maganin hypertriglyceridemia da cututtukan zuciya da jijiyoyin jini da ke da alaƙa. Yana kaiwa hariAPOC3Halitta, wanda ke ɓoye apolipoprotein C-III, mai mahimmanci mai kula da metabolism na triglyceride. A cikin bincike, ana amfani da Plozasiran don yin nazarin dabarun rage yawan lipid na tushen RNAi, ƙayyadaddun ƙayyadaddun kwayoyin halitta, da kuma dogon aiki na jiyya don yanayi irin su cututtukan chylomicronemia na iyali (FCS) da gauraye dyslipidemia.
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Zilebesiran
Zilebesiran API ƙaramin bincike ne na RNA (siRNA) wanda aka haɓaka don maganin hauhawar jini. Yana kaiwa hariAGTgene, wanda ke ɓoye angiotensinogen-wani maɓalli mai mahimmanci na tsarin renin-angiotensin-aldosterone (RAAS). A cikin bincike, ana amfani da Zilebesiran don nazarin hanyoyin yin shiru na kwayoyin halitta don sarrafa hawan jini na dogon lokaci, fasahar isar da RNAi, da kuma faffadan rawar hanyar RAAS a cikin cututtukan zuciya da na koda.