Oligonucleotide APIs
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Inclisiran sodium
Inclisiran sodium API (Active Pharmaceutical Ingredient) is studied primarily in the field of RNA interference (RNAi) and cardiovascular therapeutics. As a double-stranded siRNA targeting the PCSK9 gene, it is used in preclinical and clinical research to evaluate long-acting gene-silencing strategies for lowering LDL-C (low-density lipoprotein cholesterol). It also serves as a model compound for investigating siRNA delivery systems, stability, and liver-targeted RNA therapeutics.
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Donidalorsen
Donidalorsen API is an antisense oligonucleotide (ASO) under investigation for the treatment of hereditary angioedema (HAE) and related inflammatory conditions. It is studied in the context of RNA-targeted therapies, aiming to reduce the expression of plasma prekallikrein (KLKB1 mRNA). Researchers use Donidalorsen to explore gene silencing mechanisms, dose-dependent pharmacokinetics, and long-term control of bradykinin-mediated inflammation.
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Fitusiran
Fitusiran API is a synthetic small interfering RNA (siRNA) investigated primarily in the field of hemophilia and coagulation disorders. It targets the antithrombin (AT or SERPINC1) gene in the liver to reduce antithrombin production. Researchers use Fitusiran to explore RNA interference (RNAi) mechanisms, liver-specific gene silencing, and novel therapeutic strategies for rebalancing coagulation in hemophilia A and B patients, with or without inhibitors.
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Givosiran
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.
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Plozasiran
Plozasiran API is a synthetic small interfering RNA (siRNA) developed for the treatment of hypertriglyceridemia and related cardiovascular and metabolic disorders. It targets the APOC3 gene, which encodes apolipoprotein C-III, a key regulator of triglyceride metabolism. In research, Plozasiran is used to study RNAi-based lipid-lowering strategies, gene-silencing specificity, and long-acting treatments for conditions such as familial chylomicronemia syndrome (FCS) and mixed dyslipidemia.
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Zilebesiran
Zilebesiran API is an investigational small interfering RNA (siRNA) developed for the treatment of hypertension. It targets the AGT gene, which encodes angiotensinogen—a key component of the renin-angiotensin-aldosterone system (RAAS). In research, Zilebesiran is used to study gene silencing approaches for long-term blood pressure control, RNAi delivery technologies, and the broader role of the RAAS pathway in cardiovascular and renal diseases.