Antisense Inhibition of Apolipoprotein C-III in Patients with Hypertriglyceridemia.

@article{Gaudet2015AntisenseIO,
  title={Antisense Inhibition of Apolipoprotein C-III in Patients with Hypertriglyceridemia.},
  author={Daniel Gaudet and Veronica J Alexander and Brenda F. Baker and Diane Brisson and Karine Tremblay and Walter Singleton and Richard S. Geary and Steven G. Hughes and Nicholas J. Viney and Mark J. Graham and Rosanne M. Crooke and Joseph L. Witztum and John D. Brunzell and John J P Kastelein},
  journal={The New England journal of medicine},
  year={2015},
  volume={373 5},
  pages={
          438-47
        }
}
BACKGROUND Apolipoprotein C-III (APOC3) is a key regulator of plasma triglyceride levels. Elevated triglyceride levels are associated with a risk of adverse cardiovascular events and pancreatitis. ISIS 304801 is a second-generation antisense inhibitor of APOC3 synthesis. METHODS We conducted a randomized, double-blind, placebo-controlled, dose-ranging, phase 2 study to evaluate ISIS 304801 in untreated patients with fasting triglyceride levels between 350 mg per deciliter (4.0 mmol per liter… 

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Gene therapy: Inhibiting apoC-III synthesis in patients with hypertriglyceridaemia

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Results support the continued development of ISIS 304801 for the treatment of patients who remain at risk for cardiovascular events and pancreatitis because of very high triglyceride levels and further support the relationship between apoC‐III and triglycerides.

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Antisense Oligonucleotides for the Treatment of Hypertriglyceridemia and Hyperlipoproteinemia(a)

Two new ASOs, volanesorsen targeting ApoC-III and IONIS-APO(a)LRx targeting apo(a), are promising new agents for the treatment of hypertriglyceridemia and hyperlipoproteinemia (a), respectively.

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Current and developing therapies for reducing apoC-III levels and function to lower TGs and cardiovascular risk include existing options including statins, fibrates, thiazolidinediones, omega-3-fatty acids, and niacin, as well as an antisense oligonucleotide targeting APOC3 currently in clinical development.

Experimental therapies targeting apolipoprotein C-III for the treatment of hyperlipidemia – spotlight on volanesorsen

APOC3-targeted experimental treatments for dyslipidemia, a key regulator of TG metabolism and its targeting may reduce TG levels and cardiovascular risk, and M580, a retinoic acid receptor-α specific agonist, an agent in early-stage development are discussed.

Targeting apoC-III and ANGPTL3 in the treatment of hypertriglyceridemia

The purpose of this review is to discuss the currently available therapies and to elaborate the most promising novel therapeutics for TG lowering to become players in the treatment for (especially) severe HTG in the near future.
...

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