ION717*

ION717 is an investigational antisense medicine designed to inhibit the production of prion protein (PrP) for the potential treatment of prion disease.

About Prion Disease
Prion disease is a rare, fatal neurodegenerative disease caused by misfolding of PrP which accumulates in the brain. People with prion disease often experience progressive memory impairment, personality changes, difficulties with movement and loss of independence. There are currently no effective disease-modifying treatments for prion disease. In most cases, a person succumbs to prion disease within a year following symptom onset.

Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.

Sapablursen

Sapablursen, formerly known as IONIS-TMPRSS6-LRx, is an investigational ligand-conjugated antisense (LICA) medicine designed to target the TMPRSS6 gene to modulate the production of hepcidin, which is the key regulator of iron homeostasis. By modulating hepcidin expression, sapablursen has the potential to positively impact diseases characterized by iron deficiency, such as polycythemia vera (PV). 

About Polycythemia Vera  

Polycythemia Vera (PV) is a rare, non-genetic and potentially fatal disease caused by overproduction of red blood cells. This overproduction leads to a thickening of the blood, which increases patients’ risk of life-threatening blood clots, including in the lungs, heart and brain. Patients with PV also experience severe iron deficiency due to hepcidin overexpression. There are no approved disease-modifying treatments for PV. 

Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.

ION582*

ION582 is an investigational antisense medicine designed to inhibit the expression of the UBE3A transcript (UBE3A-ATS) for the potential treatment of Angelman syndrome (AS). 

About Angelman Syndrome

AS is a rare, genetic neurological disease caused by the loss of function of the maternally inherited UBE3A gene. AS typically presents in infancy and is characterized by intellectual disability, balance issues, motor impairment, and debilitating seizures. Some patients are unable to walk or speak. Some symptoms can be managed with existing drugs; however, there are no approved disease modifying therapies. 

Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.

ION904

ION904 is an investigational next-generation ligand-conjugated antisense (LICA) medicine designed to inhibit the production of angiotensinogen to decrease blood pressure in people with uncontrolled hypertension.

About Treatment-Resistant Hypertension

Treatment-resistant hypertension (TRH) is defined as failure to achieve a blood pressure goal of 140/90 (systolic/diastolic) despite the use of three or more antihypertensive medications. People with TRH have been found to have a three-fold higher chance of having fatal and non-fatal cardiovascular events relative to those with controlled hypertension.

Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.

ION224

ION224 is an investigational ligand-conjugated antisense (LICA) medicine designed to reduce the production of diacylglycerol acyltransferase 2 (DGAT2) to treat patients with metabolic dysfunction-associated steatohepatitis (MASH), previously referred to as nonalcoholic steatohepatitis (NASH). DGAT2 is an enzyme that catalyzes the final step in triglyceride synthesis in the liver. Reducing the production of DGAT2 should therefore decrease triglyceride synthesis in the liver. The Phase 2 trial demonstrated clinical evidence that the reduction of hepatic fat after DGAT2 inhibition correlates with MASH histological endpoints.

About Metabolic Dysfunction-Associated Steatohepatitis (MASH)

MASH is the more severe form of metabolic dysfunction-associated fatty liver disease (MASLD). It is related to the epidemic of obesity, pre-diabetes and diabetes. Unlike liver disease caused by alcohol consumption, MASH is the result of an accumulation of fat in the liver, which can lead to inflammation, cirrhosis and liver-related death.

Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.

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