Givosiran

Givosiran (API)

Ntchito Yofufuza:
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.

Ntchito:
Givosiran ntchito ndi kuchepetsa mawu aALAS1mu hepatocytes, potero kuchepetsa kudzikundikira kwa poizoni heme intermediates monga ALA (aminolevulinic acid) ndi PBG (porphobilinogen). Izi zimathandiza kupewa kuukira kwa neurovisceral komwe kumakhudzana ndi pachimake hepatic porphyria. Monga API, Givosiran ndi gawo lothandizira lamankhwala mu RNAi-based therapeutics yopangidwa kuti ipereke kulamulira kwa nthawi yayitali kwa AHP ndi subcutaneous administration.