ION839

ION839 (formerly IONIS-AZ6-2.5-LRx), also known as AZD2693, is an investigational ligand-conjugated antisense (LICA) medicine designed to inhibit the production of patatin-like phospholipase domain-containing 3 (PNPLA3) protein. PNPLA3 is a protein that is found on the surface of intracellular lipid droplets. Studies have shown that a common genetic mutation of PNPLA3 is strongly associated with an increased risk for metabolic dysfunction-associated steatohepatitis (MASH), previously known as non-alcoholic steatohepatitis (NASH), an accumulation of fat in the liver that causes liver damage. The mutant PNPLA3 protein is resistant to degradation, causing it to accumulate on the surface of lipid droplets, which disrupts the normal process for degrading lipid droplets, leading to increased liver fat accumulation, the underlying pathology of MASH. In a mouse model of MASH that results from the expression of mutant PNPLA3, intervention with an antisense drug targeting PNPLA3 reduced hepatic mutant protein expression. This inhibition of mutant protein expression reduced liver fat accumulation, inflammation, and fibrosis, all hallmarks of MASH pathology in the mutant mouse model.

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.

ION541*

ION541, also known as BIIB105, is an investigational antisense medicine designed to reduce the production of the ataxin-2 (ATXN2) protein for the potential treatment of amyotrophic lateral sclerosis (ALS). The reduction of ATXN2 has been shown to decrease aggregation of TDP-43, a toxic RNA binding protein found in most patients with ALS, including the approximately 90 percent of the ALS population with no known family history of ALS. 

About Amyotrophic Lateral Sclerosis

ALS is a rare, fatal neurodegenerative disorder characterized by progressive degeneration of motor neurons resulting in a declining quality of life due to muscle weakness, loss of movement and difficulty in breathing and swallowing. Currently, treatment options for patients with ALS are extremely limited with no drugs that significantly slow disease progression.

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

ION859*

ION859 (formerly IONIS-BIIB7Rx), also known as BIIB094, is an investigational antisense medicine designed to inhibit the production of the leucine-rich repeat kinase 2 (LRRK2) protein as a potential therapy for Parkinson’s disease (PD). The most common genetic mutations in PD are found in the LRRK2 protein. It is believed that increased LRRK2 protein activity could be one of the key drivers for developing PD. 

About Parkinson’s Disease

PD is a progressive neurodegenerative disease characterized by loss of neurons in the motor system. Patients with PD can experience tremors, loss of balance and coordination, stiffness, slowing of movement, changes in speech and in some cases cognitive decline. PD is ultimately fatal. There are treatments that can relieve symptoms, but there are no approved disease modifying therapies. 

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

ION464*

ION464, also known as BIIB101, is an investigational antisense medicine designed to inhibit the production of the alpha-synuclein protein as a potential therapy for Parkinson’s disease (PD), multiple system atrophy (MSA) and related synucleinopathies. 

About Multiple System Atrophy and Parkinson’s Disease 

Alpha-synuclein protein abnormally accumulates in the brains of PD and MSA patients and is thought to be one of the key drivers of these diseases. It is believed that decreasing the production of the alpha-synuclein protein will reduce the toxic effects of gain-of-function mutations. 

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

IONIS-MAPT

IONIS-MAPTRx, also known as BIIB080, is an investigational antisense medicine designed to selectively inhibit production of the microtubule-associated protein tau (MAPT), or tau protein in the brain. IONIS-MAPTRx is being developed to treat people with Alzheimer’s disease (AD) and potentially other neurodegenerative disorders characterized by the deposition of abnormal tau protein in the brain, such as certain forms of frontotemporal degeneration and progressive supranuclear palsy.

About Alzheimer’s Disease 

AD is characterized predominantly by memory impairment and behavioral changes, resulting in a person’s inability to independently perform daily activities. AD generally occurs late in life and may progress to death in five to 20 years after the onset of the disease.

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

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