Oligonucleotide APIs
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Inclisiran sodium
Inclisiran sodium API (Active Pharmaceutical Ingredient) inodzidzwa kunyanya mumunda weRNA kupindira (RNAi) uye kurapa kwemwoyo. SeSiRNA ine tambo mbiri yakanangana nePCSK9 gene, inoshandiswa mu preclinical uye kiriniki yekutsvagisa kuongorora maitiro-akareba-anoita gene-kunyarara nzira dzekudzikisa LDL-C (yakaderera-density lipoprotein cholesterol). Iyo inoshandawo semuenzaniso wekomboni yekuongorora siRNA yekuendesa masisitimu, kugadzikana, uye chiropa-yakanangwa RNA therapeutics.
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Donilorsen
Donidalorsen API is an antisense oligonucleotide (ASO) iri pasi pekuferefeta kurapwa kwenhaka angioedema (HAE) uye ine hukama hwekuzvimba mamiriro. Inodzidzwa mumamiriro ezvinhu eRNA-inotarirwa marapirwo, nechinangwa chekudzikisa kutaura kweplasma prekallikrein(KLKB1 mRNA). Vatsvagiri vanoshandisa Donidalorsen kuti vatarise maitiro ekunyararidza majini, dosi-inotsamira pharmacokinetics, uye kudzora kwenguva refu kwebradykinin-mediated kuzvimba.
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Fitusiran
Fitusiran API ndeyekugadzira diki inopindira RNA (siRNA) yakaongororwa zvakanyanya mumunda wehemophilia uye coagulation kusagadzikana. Inonangana neantithrombin (AT kana SEPINC1)gene muchiropa kuderedza kugadzirwa kwe antithrombin. Vatsvakurudzi vanoshandisa Fitusiran kuongorora RNA interference (RNAi) nzira, chiropa-chaiyo gene kunyarara, uye novel nzira dzekurapa dzekugadzirisazve coagulation muhemophilia A uye B varwere, vane kana vasina inhibitors.
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Givosiran
Givosiran API is a synthetic small interfering RNA (siRNA) yakadzidzira kurapwa kweacute hepatic porphyria (AHP). Inonyanya kunanga iyoALAS1gene (aminolevulinic acid synthase 1), iyo inobatanidzwa muheme biosynthesis nzira. Vatsvakurudzi vanoshandisa Givosiran kuongorora RNA interference (RNAi) -based therapies, chiropa-chinonangwa gene kunyarara, uye modulation ye metabolic nzira dzinobatanidzwa mu porphyria uye ane hukama hwemajini kusagadzikana.
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Plozasiran
Plozasiran API is a synthetic diki inopindira RNA (siRNA) yakagadzirirwa kurapwa kwe hypertriglyceridemia uye ine hukama hwemoyo uye metabolic kusagadzikana. Inonangana neAPOC3gene, iyo inokodha apolipoprotein C-III, mutongi akakosha we triglyceride metabolism. Mukutsvagisa, Plozasiran inoshandiswa kudzidza RNAi-based lipid-kudzikisa marongero, gene-kunyarara, uye kwenguva refu-kuita marapirwo emamiriro ezvinhu akadai semhuri chylomicronemia syndrome (FCS) uye yakasanganiswa dyslipidemia.
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Zilebesiran
Zilebesiran API ndeyekuferefeta diki inopindira RNA (siRNA) yakagadzirirwa kurapwa kwehypertension. Inonangana neAGTgene, iro rinoisa angiotensinogen - chikamu chakakosha che renin-angiotensin-aldosterone system (RAAS). Mukutsvaga, Zilebesiran inoshandiswa kudzidza nzira dzekunyarara kwemajini kwenguva refu yekudzivirira kweropa, RNAi kuburitsa matekinoroji, uye basa rakakura reiyo RAAS nzira muzvirwere zvemoyo uye renal.