Oligonucleotide APIs
-
Inclisiran sodium
Inclisiran sodium API (Active Pharmaceutical Ingredient) yog kawm feem ntau hauv kev cuam tshuam ntawm RNA (RNAi) thiab kev kho mob plawv. Raws li ob-stranded siRNA lub hom phiaj ntawm PCSK9 noob, nws yog siv nyob rau hauv preclinical thiab soj ntsuam kev tshawb fawb los soj ntsuam cov gene-silencing lub tswv yim ntev rau txo LDL-C (tsawg-density lipoprotein cholesterol). Nws kuj tseem ua haujlwm ua qauv sib xyaw rau kev tshawb xyuas siRNA kev xa khoom, kev ruaj ntseg, thiab lub siab-targeted RNA kho.
-
Donidalorsen
Donidalorsen API yog ib qho antisense oligonucleotide (ASO) nyob rau hauv kev tshawb nrhiav rau kev kho mob ntawm hereditary angioedema (HAE) thiab lwm yam mob. Nws yog kawm nyob rau hauv cov ntsiab lus ntawm RNA-targeted kev kho mob, aiming kom txo tau cov kev qhia ntawmntshav plasma prekallikrein(KLKB1 mRNA). Cov kws tshawb fawb siv Donidalorsen los tshawb nrhiav cov tshuaj gene silencing mechanisms, koob tshuaj raws li pharmacokinetics, thiab kev tswj mus sij hawm ntev ntawm bradykinin-mediated o.
-
Fitusiran
Fitusiran API yog ib qho hluavtaws me me cuam tshuam RNA (siRNA) tau tshawb xyuas feem ntau hauv thaj tsam ntawm hemophilia thiab coagulation disorders. Nws aims lubAntithrombin (AT lossis SERPINC1)gene nyob rau hauv lub siab kom txo tau cov tshuaj antithrombin. Cov kws tshawb fawb siv Fitusiran los tshawb txog RNA cuam tshuam (RNAi) cov txheej txheem, lub siab tshwj xeeb ntawm cov gene silencing, thiab cov tswv yim kho tshiab rau rebalancing coagulation hauv cov neeg mob hemophilia A thiab B, nrog lossis tsis muaj inhibitors.
-
Givosiran
Givosiran API yog ib qho hluavtaws me me cuam tshuam RNA (siRNA) tau kawm rau kev kho mob ntawm tus mob hepatic porphyria (AHP). Nws tshwj xeeb tsom rau covALAS 1noob (aminolevulinic acid synthase 1), uas yog koom nrog hauv heme biosynthesis txoj kev. Cov kws tshawb fawb siv Givosiran los tshawb xyuas RNA cuam tshuam (RNAi)-raws li kev kho mob, lub siab-targeted gene silencing, thiab kev hloov pauv ntawm cov txheej txheem metabolic koom nrog porphyria thiab cuam tshuam txog caj ces.
-
Plozasiran
Plozasiran API yog ib qho hluavtaws me me cuam tshuam RNA (siRNA) tsim los rau kev kho mob hypertriglyceridemia thiab cuam tshuam txog cov hlab plawv thiab cov kab mob metabolic. Nws aims lubAPOC 3noob, uas encodes apolipoprotein C-III, ib tug tseem ceeb regulator ntawm triglyceride metabolism. Hauv kev tshawb fawb, Plozasiran yog siv los kawm RNAi-raws li lipid-txo cov tswv yim, gene-silencing tshwj xeeb, thiab kev kho mob ntev rau cov xwm txheej xws li tsev neeg chylomicronemia syndrome (FCS) thiab sib xyaw dyslipidemia.
-
Zilebesiran
Zilebesiran API yog ib qho kev tshawb nrhiav me me cuam tshuam RNA (siRNA) tsim rau kev kho mob ntshav siab. Nws aims lubAGTgene, uas encodes angiotensinogen-ib qho tseem ceeb ntawm renin-angiotensin-aldosterone system (RAAS). Hauv kev tshawb fawb, Zilebesiran yog siv los kawm gene silencing txoj hauv kev rau kev tswj ntshav siab mus ntev, RNAi kev xa khoom thev naus laus zis, thiab lub luag haujlwm dav dav ntawm RAAS txoj hauv kev hauv cov kab mob plawv thiab lub raum.