Oligonucleotide APIs
-
Inclisiran sodium
Inclisiran sodium API (Active Pharmaceutical Ingredient) yizwe cyane cyane mubijyanye no kwivanga kwa RNA (RNAi) hamwe nubuvuzi bwumutima. Nka siRNA ikubye kabiri yibasira gene PCSK9, ikoreshwa mubushakashatsi bwibanze nubuvuzi kugirango isuzume ingamba zimaze igihe zicecekesha gene yo kugabanya LDL-C (cholesterol ya lipoprotein nkeya). Ikora kandi nk'icyitegererezo cyo gukora iperereza kuri sisitemu yo gutanga siRNA, ituze, hamwe na RNA ivura umwijima.
-
Donidalorsen
Donidalorsen API ni antisense oligonucleotide (ASO) iri gukorwaho iperereza kugirango ivure umurage wa angioedema (HAE) hamwe nindwara ziterwa na inflammatory. Yizwe murwego rwa RNA igamije kuvura, igamije kugabanya imvugo yaplasma prekallikrein(KLKB1 mRNA). Abashakashatsi bifashisha Donidalorsen kugira ngo basuzume uburyo bwo gucecekesha gene, imiti iterwa na farumasi, no kugenzura igihe kirekire kurwanya indwara ya bradykinin.
-
Fitusiran
Fitusiran API ni sintetike ntoya yivanga RNA (siRNA) yakozweho iperereza cyane cyane mubijyanye na hemofilia n'indwara ya coagulation. Ireba iantithrombine (AT cyangwa SERPINC1)gene mu mwijima kugirango igabanye umusaruro wa antithrombine. Abashakashatsi bifashisha Fitusiran kugira ngo basuzume uburyo RNA yivanga (RNAi), gucecekesha gene yihariye y’umwijima, hamwe n’ingamba zo kuvura udushya zo kongera guhuza imitsi mu barwayi ba hémofilia A na B, hamwe na inhibitor.
-
Givosiran
Givosiran API ni sintetike ntoya yivanga RNA (siRNA) yize kugirango ivure poropiya ikaze ya hepatike (AHP). Irasa cyane cyane iALAS1gene (aminolevulinic acide synthase 1), igira uruhare munzira ya heme biosynthesis. Abashakashatsi bifashisha Givosiran kugira ngo bakore iperereza ku buvuzi bwa RNA (RNAi) bushingiye ku buvuzi, gucecekesha gene igamije umwijima, no guhindura inzira za metabolike zigira uruhare muri porphiriya n'indwara zishingiye ku ngirabuzima fatizo.
-
Plozasiran
Plozasiran API ni sintetike ntoya yivanga RNA (siRNA) yatejwe imbere yo kuvura hypertriglyceridemia hamwe nindwara zifata umutima nimiyoboro hamwe na metabolike. Ireba iAPOC3gene, igizwe na apolipoprotein C-III, igenga urufunguzo rwa metabolism ya triglyceride. Mu bushakashatsi, Plozasiran akoreshwa mu kwiga ingamba zo kugabanya lipide zishingiye kuri RNAi, gucecekesha gene, no kuvura igihe kirekire ku bihe nka syndrome de famille chylomicronemia (FCS) hamwe na dyslipidemiya ivanze.
-
Zilebesiran
Zilebesiran API ni iperereza rito ryivanga RNA (siRNA) ryakozwe kugirango rivure hypertension. Ireba iAGTgene, igizwe na angiotensinogen-igice cyingenzi cya sisitemu ya renin-angiotensin-aldosterone (RAAS). Mu bushakashatsi, Zilebesiran akoreshwa mu kwiga uburyo bwo gucecekesha gene mu gihe cyo kurwanya umuvuduko w’amaraso igihe kirekire, tekinoroji yo gutanga RNAi, n’uruhare runini rw’inzira ya RAAS mu ndwara zifata umutima ndetse n’impyiko.