Givosiran

Givosiran (API)

Tsvagiridzo Yekushandisa:
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.

Basa:
Givosiran inoshanda nekuderedza kutaura kweALAS1muhepatocytes, nokudaro kuderedza kuunganidzwa kwechepfu heme intermediates yakadai seALA (aminolevulinic acid) uye PBG (porphobilinogen). Izvi zvinobatsira kudzivirira neurovisceral kurwisa kwakabatana neacute hepatic porphyria. Se API, Givosiran ndiyo inoshandiswa mishonga yemishonga muRNAi-based therapeutics yakagadzirirwa kupa kutonga kwenguva refu kweAHP ne subcutaneous administration.