Oligonucleotide APIs
-
E kenyelletsa sodium
Inclisiran sodium API (Active Pharmaceutical Ingredient) e ithutoa haholo-holo tšimong ea ho kena-kenana le RNA (RNAi) le therapeutics ea pelo. Joalo ka siRNA e nang le likarolo tse peli e shebileng mofuta oa PCSK9, e sebelisoa lipatlisisong tsa pele le tsa bongaka ho lekola maano a nako e telele a ho khutsisa liphatsa tsa lefutso bakeng sa ho theola LDL-C (low-density lipoprotein cholesterol). E boetse e sebetsa e le motsoako oa mohlala bakeng sa ho batlisisa mekhoa ea phepelo ea siRNA, botsitso, le phekolo ea RNA e lebisitsoeng sebeteng.
-
Donidalorsen
Donidalorsen API ke antisense oligonucleotide (ASO) e ntseng e etsoa lipatlisiso bakeng sa phekolo ea angioedema ea lefutso (HAE) le maemo a amanang le ho ruruha. E ithutoa moelelong oa liphekolo tse lebisitsoeng ho RNA, ka sepheo sa ho fokotsa polelo eaplasma prekallikrein(KLKB1 mRNA). Bafuputsi ba sebelisa Donidalorsen ho hlahloba mekhoa ea ho khutsisa liphatsa tsa lefutso, pharmacokinetics e itšetlehileng ka tekanyo, le taolo ea nako e telele ea ho ruruha ha bradykinin.
-
"Fitusiran"
Fitusiran API ke RNA e nyane e sitisang e nyane (siRNA) e batlisisitsoeng haholo-holo lebaleng la mathata a haemophilia le a coagulation. E lebisitse hoantithrombin (AT kapa SEPINC1)liphatsa tsa lefutso sebeteng ho fokotsa tlhahiso ea antithrombin. Bafuputsi ba sebelisa Fitusiran ho hlahloba mekhoa ea ho kena-kenana le RNA (RNAi), ho khutsisa liphatsa tsa lefutso ka sebete, le mekhoa e mecha ea phekolo ea ho leka-lekanya coagulation ho bakuli ba hemophilia A le B, ba nang le li-inhibitors kapa ba se na tsona.
-
Givosiran
Givosiran API ke RNA e nyane e sitisang e nyane (siRNA) e ithutoang bakeng sa kalafo ea acute hepatic porphyria (AHP). E shebane ka ho khetheha leALAS1gene (aminolevulinic acid synthase 1), e amehang tseleng ea heme biosynthesis. Bafuputsi ba sebelisa Givosiran ho batlisisa mekhoa ea phekolo e thehiloeng ho RNA (RNAi), ho khutsisa liphatsa tsa lefutso tse lebisitsoeng ho sebete, le ho feto-fetoha ha mekhoa ea metabolic e amehang ho porphyria le mathata a amanang le lefutso.
-
Plozasiran
Plozasiran API ke RNA e nyane e sitisang e nyane (siRNA) e ntlafalitsoeng bakeng sa kalafo ea hypertriglyceridemia le mathata a amanang le pelo le metabolic. E lebisitse hoAPOC3gene, e kenyelletsang apolipoprotein C-III, e laolang metabolism ea triglyceride. Lipatlisisong, Plozasiran e sebelisetsoa ho ithuta maqheka a theolang lipid a thehiloeng ho RNAi, ho khetholla liphatsa tsa lefutso, le phekolo ea nako e telele bakeng sa maemo a kang malapa a chylomicronemia syndrome (FCS) le dyslipidemia e tsoakiloeng.
-
Zilebesiran
Zilebesiran API ke lipatlisiso tse nyane tse sitisang RNA (siRNA) tse ntlafalitsoeng bakeng sa kalafo ea khatello ea mali. E lebisitse hoAGTgene, e kenyang angiotensinogen-e leng karolo ea bohlokoa ea renin-angiotensin-aldosterone system (RAAS). Lipatlisisong, Zilebesiran e sebelisetsoa ho ithuta mekhoa ea ho khutsisa liphatsa tsa lefutso bakeng sa taolo ea nako e telele ea khatello ea mali, theknoloji ea ho fana ka RNAi, le karolo e pharaletseng ea tsela ea RAAS ho mafu a pelo le a renal.