Givosiran

Givosiran (API)

Isicelo soPhando:
I-Givosiran API yi-RNA encinci ephazamisayo (i-siRNA) efundela unyango lwe-acute hepatic porphyria (AHP). Ijolise ngokukodwa kwiI-ALAS1gene (i-aminolevulinic acid synthase 1), ebandakanyeka kwi-heme biosynthesis pathway. Abaphandi basebenzisa i-Givosiran ukuphanda ukungenelela kwe-RNA (RNAi)-based therapies, ukuthulisa i-gene ejoliswe kwisibindi, kunye nokuguqulwa kweendlela ze-metabolic ezibandakanyekayo kwi-porphyria kunye nokuphazamiseka kwemfuza okuhambelanayo.

Umsebenzi:
Givosiran imisebenzi ngokunciphisa intetho yeI-ALAS1kwi-hepatocytes, ngaloo ndlela kunciphisa ukuqokelelwa kwe-heme intermediates enetyhefu efana ne-ALA (aminolevulinic acid) kunye ne-PBG (porphobilinogen). Oku kunceda ukuthintela uhlaselo lwe-neurovisceral oluhambelana ne-acute hepatic porphyria. Njenge-API, i-Givosiran iyona nxalenye yeyeza esebenzayo kwi-RNAi-based therapeutics eyenzelwe ukubonelela ngolawulo lwexesha elide lwe-AHP kunye nokulawulwa kwe-subcutaneous.