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ISIS-ANGPTL3

ISIS-ANGPTL3 is an antisense drug we designed to reduce angiopoietin-like 3 protein, or ANGPTL3, an independent risk factor for cardiovascular disease. ANGPTL3 is produced in the liver and regulates lipid, glucose and energy metabolism.

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ISIS-APOA

ISIS-APOA is an antisense drug we designed to reduce apolipoprotein(a) in the liver to offer a direct approach for reducing Lp(a), an independent risk factor for cardiovascular disease.

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ISIS-APOCIII

ISIS-APOCIII is an antisense drug we designed to reduce apolipoprotein C-III, or apoC-III, protein production and lower triglycerides. ApoC-III regulates triglyceride metabolism in the blood and is an independent cardiovascular risk factor. Humans who do not produce apoC-III have lower levels of triglycerides and lower instances of cardiovascular disease.

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ISIS-CRP

ISIS-CRP is an antisense drug that targets CRP, a protein produced in the liver. CRP levels increase dramatically during inflammatory disorders, and scientists have linked excessive amounts of CRP to coronary artery disease. Furthermore, a growing body of evidence from clinical trials implicates CRP in cardiovascular disease progression.

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ISIS-FXI

ISIS-FXI is an antisense drug we designed to treat clotting disorders. It targets Factor XI, a clotting factor produced in the liver that is an important component of the coagulation pathway.

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ISIS-GCCR

ISIS-GCCR is an antisense drug that targets the glucocorticoid receptor, or GCCR. Glucocorticoid hormones affect a variety of processes throughout the body, and excessive levels of glucocorticoid hormones can have a detrimental effect on many of the tissues and organs in the body.

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ISIS-PKK

ISIS-PKK is an antisense drug designed to prevent hereditary angioedema, or HAE, attacks. It inhibits the production of prekallikren, or PKK, a protein produced in the liver that plays an important role in the activation of inflammatory mediators associated with acute attacks of HAE.

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ISIS-SMN

ISIS-SMN is an antisense drug we designed to treat spinal muscular atrophy, or SMA, a severe motor-neuron disease that is the leading genetic cause of infant mortality.

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ISIS-TTR

ISIS-TTR is an antisense drug we designed to treat transthyretin amyloidosis, or TTR amyloidosis, a severe and rare genetic disease in which the patient inherits a mutant gene that produces a misfolded form of TTR, which progressively accumulates in tissues.

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IU1

IU1 is a reversible, small molecule inhibitor of deubiquitination by USP14 that demonstrates an IC50 value of 4-5 μM. IU1 selectively stimulates ubiquitin-dependent protein degradation in vitro at 34 μM and in MEF cells at 50 μM.

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