Givosiran

I-Givosiran (API)

Isicelo socwaningo:
I-Givosiran API iyi-RNA encane ephazamisayo yokwenziwa (siRNA) efundelwe ukwelashwa kwe-acute hepatic porphyria (AHP). Iqondise ngokuqondile i-I-ALAS1isakhi sofuzo (i-aminolevulinic acid synthase 1), ehilelekile endleleni ye-heme biosynthesis. Abacwaningi basebenzisa i-Givosiran ukuze baphenye izindlela zokwelapha ezisekelwe ku-RNA (RNAi), ukuthulisa izakhi zofuzo eziqondiswe esibindini, kanye nokuguqulwa kwemizila ye-metabolic ehilelekile ku-porphyria kanye nokuphazamiseka kofuzo okuhlobene.

Umsebenzi:
Imisebenzi ye-Givosiran ngokunciphisa ukubonakaliswa kweI-ALAS1kuma-hepatocytes, ngaleyo ndlela kunciphisa ukuqoqwa kwe-heme enobuthi ephakathi njenge-ALA (aminolevulinic acid) ne-PBG (porphobilinogen). Lokhu kusiza ukuvimbela ukuhlaselwa kwe-neurovisceral okuhambisana ne-acute hepatic porphyria. Njenge-API, i-Givosiran iyisakhi semithi esebenzayo ku-RNAi-based therapeutics eklanyelwe ukunikeza ukulawula kwesikhathi eside kwe-AHP ngokuphathwa okungaphansi kwesikhumba.