Oligonucleotide APIs
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Soodhiyamka Inclisiran
Inclisiran sodium API (Waxyaabaha Farmaceutical Firmaceutical Active) waxaa lagu bartaa ugu horrayn dhanka faragelinta RNA (RNAi) iyo daaweynta wadnaha iyo xididdada dhiigga. Sida siRNA laba-jibbaaran oo lagu beegsanayo hidda-wadaha PCSK9, waxa loo adeegsadaa cilmi-baadhisyo hore iyo kuwo bukaan-socod si loo qiimeeyo xeeladaha aamusinta hidda-wadaha muddada-dheer ee hoos u dhigista LDL-C (kolestaroolka lipoprotein-ka-cufnaanta hoose). Waxa kale oo ay u adeegtaa sidii qaab dhismeed oo loogu talagalay baadhista hababka gudbinta siRNA, xasilloonida, iyo daaweynta RNA ee beerka-la beegsado.
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Donidalorsen
Donidalorsen API waa oligonucleotide antisense (ASO) oo lagu baarayo daaweynta xanuunka anjioedema ee la iska dhaxlo (HAE) iyo xaaladaha bararka ee la xiriira. Waxaa lagu bartaa macnaha guud ee daaweynta RNA-bartilmaameedka, ujeedadeedu tahay in la yareeyo muujintaplasma prekallikrein(KLKB1 mRNA). Cilmi-baadhayaashu waxay isticmaalaan Donidalorsen si ay u sahamiyaan hababka aamusinta hidda-wadaha, daawooyinka farmashiyaha ku-tiirsan ee qiyaasta, iyo xakamaynta muddada-dheer ee caabuqa dhexdhexaadka ah ee bradykinin.
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Fitusiran
Fitusiran API waa RNA faragelin yar oo synthetic ah (siRNA) oo ugu horrayn lagu baaro haemophilia iyo xanuunada xinjirowga. Waxay beegsanaysaaantithrombin (AT ama SERPINC1)hidde-sideyaasha beerka si loo yareeyo wax soo saarka antithrombin. Cilmi baadhayaashu waxay u isticmaalaan Fitusiran si ay u sahamiyaan hababka faragelinta RNA (RNAi), aamusinta hidda-socodka beerka-gaarka ah, iyo xeelado daweyn oo cusub oo loogu talagalay dib-u-dheellitirka xinjirowga dhiigga ee bukaannada hemophilia A iyo B, oo leh ama aan lahayn xannibayaal.
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Givosiran
Givosiran API waa RNA (siRNA) faragelin yar oo synthetic ah oo loo bartay daaweynta cagaarshow ba'an (AHP). Waxay si gaar ah u beegsanaysaaALAS1hiddo-wadaha (aminolevulinic acid synthase 1), kaas oo ku lug leh dariiqa biosynthesis ee heme. Cilmi-baadhayaashu waxay isticmaalaan Givosiran si ay u baaraan faragelinta RNA (RNAi) daaweynta ku salaysan, aamusnaanta hiddo-wadaha beerka, iyo habeynta hababka dheef-shiid kiimikaadka ee ku lug leh porphyria iyo cilladaha hiddaha ee la xiriira.
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Plozasiran
Plozasiran API waa RNA faragelin yar oo synthetic ah (siRNA) oo loo sameeyay daaweynta hypertriglyceridemia iyo xanuunada wadnaha iyo xididdada dhiigga ee la xiriira. Waxay beegsanaysaaAPOC3hidda-wadaha, kaas oo qeexaya apolipoprotein C-III, oo ah maamulaha muhiimka ah ee dheef-shiid kiimikaadka triglyceride. Cilmi-baarista, Plozasiran waxaa loo isticmaalaa in lagu barto xeeladaha hoos u dhigista dufanka-ku-saleysan ee RNAi, gaarka ah ee aamusnaanta hidda-wadaha, iyo daaweynta muddada-dheer ee daaweynta xaaladaha sida qoyska chylomicronemia syndrome (FCS) iyo dyslipidemia isku dhafan.
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Zilebesiran
Zilebesiran API waa baaritaan yar oo faragelinaya RNA (siRNA) oo loo sameeyay daawaynta hypertension. Waxay beegsanaysaaAGTHidde-side, kaas oo qeexaya angiotensinogen-waa qayb muhiim ah oo ka mid ah nidaamka renin-angiotensin-aldosterone (RAAS). Cilmi-baarista, Zilebesiran waxaa loo isticmaalaa in lagu barto hababka aamusinta hidda-socodka ee xakamaynta cadaadiska dhiigga ee muddada-dheer, tignoolajiyada gaarsiinta RNAi, iyo doorka ballaaran ee dariiqa RAAS ee cudurrada wadnaha iyo xididdada kelyaha.