Oligonucleotide APIs
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Kuphatikizika kwa sodium
Inclisiran sodium API (Active Pharmaceutical Ingredient) imaphunziridwa makamaka m'munda wa RNA interference (RNAi) ndi mankhwala a mtima. Monga siRNA yokhala ndi mizere iwiri yolunjika ku jini ya PCSK9, imagwiritsidwa ntchito pofufuza zachipatala komanso zachipatala kuti awunike njira zochepetsera jini zochepetsera LDL-C (low-density lipoprotein cholesterol). Imagwiranso ntchito ngati gawo lachitsanzo pakufufuza njira zoperekera siRNA, kukhazikika, komanso njira zochizira za RNA zomwe zimayang'aniridwa ndi chiwindi.
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Donilorsen
Donilorsen API ndi antisense oligonucleotide (ASO) yomwe ikufufuzidwa pochiza angioedema ya cholowa (HAE) ndi zina zotupa. Amaphunziridwa mu nkhani ya RNA-zochizira, cholinga kuchepetsa kufotokozaplasma prekallikrein(KLKB1 mRNA). Ofufuza amagwiritsa ntchito Donidalorsen kuti afufuze njira zochepetsera ma jini, ma pharmacokinetics omwe amadalira mlingo, komanso kuwongolera kwanthawi yayitali kwa kutupa kwa bradykinin.
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Fitusiran
Fitusiran API ndi kaphatikizidwe kakang'ono kosokoneza RNA (siRNA) komwe kumafufuzidwa makamaka pankhani ya hemophilia ndi matenda a coagulation. Imalimbana ndiantithrombin (AT kapena SEPINC1)jini m'chiwindi kuti achepetse kupanga kwa antithrombin. Ofufuza amagwiritsa ntchito Fitusiran kuti afufuze njira za RNA interference (RNAi), kuletsa jini yeniyeni ya chiwindi, ndi njira zatsopano zochiritsira zochepetsera coagulation mu hemophilia A ndi B odwala, kapena opanda zoletsa.
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Givosiran
Givosiran API ndi kaphatikizidwe kakang'ono kosokoneza RNA (siRNA) yophunzirira pochiza pachimake kwa chiwindi porphyria (AHP). Imalimbana makamaka ndiALAS1jini (aminolevulinic acid synthase 1), yomwe imakhudzidwa ndi njira ya heme biosynthesis. Ofufuza amagwiritsa ntchito Givosiran kuti afufuze njira zochiritsira za RNA interference (RNAi), kuletsa jini yolimbana ndi chiwindi, komanso kusintha kwa kagayidwe kachakudya kamene kamayambitsa porphyria ndi matenda okhudzana ndi majini.
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Plozasiran
Plozasiran API ndi kaphatikizidwe kakang'ono kosokoneza RNA (siRNA) komwe kamapangidwira kuchiza hypertriglyceridemia ndi matenda okhudzana ndi mtima ndi kagayidwe kachakudya. Imalimbana ndiAPOC3jini, yomwe imayika apolipoprotein C-III, wowongolera kwambiri wa triglyceride metabolism. Pofufuza, Plozasiran amagwiritsidwa ntchito pophunzira njira zochepetsera lipids za RNAi, gene-sencing specificity, ndi mankhwala okhalitsa kwa nthawi yaitali monga matenda a chylomicronemia syndrome (FCS) ndi dyslipidemia yosakanikirana.
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Zilebesiran
Zilebesiran API ndi kafukufuku waung'ono wosokoneza RNA (siRNA) wopangidwa kuti azichiza matenda oopsa. Imalimbana ndiAGTjini, yomwe imayika angiotensinogen - chigawo chachikulu cha renin-angiotensin-aldosterone system (RAAS). Pofufuza, Zilebesiran amagwiritsidwa ntchito pophunzira njira zochepetsera ma jini pakuwongolera kuthamanga kwa magazi kwa nthawi yayitali, matekinoloje operekera RNAi, komanso gawo lalikulu la njira ya RAAS pamtima ndi aimpso.