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Givosiran API is a synthetic small interfering RNA (siRNA) studied for the treatment of acute hepatic porphyria (AHP). It specifically targets the ALAS1 gene (aminolevulinic acid synthase 1), which is involved in the heme biosynthesis pathway. Researchers use Givosiran to investigate RNA interference (RNAi)-based therapies, liver-targeted gene silencing, and the modulation of metabolic pathways involved in porphyria and related genetic disorders.
Givosiran functions by reducing the expression of ALAS1 in hepatocytes, thereby decreasing the accumulation of toxic heme intermediates such as ALA (aminolevulinic acid) and PBG (porphobilinogen). This helps prevent the neurovisceral attacks associated with acute hepatic porphyria. As an API, Givosiran is the active pharmaceutical component in RNAi-based therapeutics designed to provide long-term control of AHP with subcutaneous administration.
Givosiran API is a synthetic small interfering RNA (siRNA) studied for the treatment of acute hepatic porphyria (AHP).
Givosiran specifically targets the ALAS1 gene (aminolevulinic acid synthase 1), which plays a role in the heme biosynthesis pathway.
Researchers utilize Givosiran to study RNA interference (RNAi)-based therapies, liver-targeted gene silencing, and the modulation of metabolic pathways related to porphyria and associated genetic disorders.
It functions by reducing the expression of ALAS1 in hepatocytes, which decreases the accumulation of toxic heme intermediates including ALA (aminolevulinic acid) and PBG (porphobilinogen).
By reducing toxic heme intermediates, Givosiran helps prevent neurovisceral attacks associated with acute hepatic porphyria.
As an active pharmaceutical ingredient (API) in RNAi-based therapeutics, Givosiran is designed for subcutaneous administration to provide long-term control of AHP.
