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AKCEA-APOCIII-L

Generation 2+ LICA antisense drug

AKCEA-APOCIII-LRx is a Generation 2+ ligand-conjugated antisense (LICA) drug designed to inhibit the production of apoC-III, for patients who are at risk of disease due to elevated triglyceride levels. ApoC-III is a protein produced in the liver that regulates triglyceride metabolism in the blood. People with severely elevated triglycerides, such as people with familial chylomicronemia syndrome (FCS), are at high risk for acute pancreatitis and other serious conditions. ApoC-III is also the target of WAYLIVRA, the first approved medicine for patients with FCS.

About familial chylomicronemia syndrome (FCS)

FCS is a rare, genetic disease characterized by extremely elevated triglyceride levels that is estimated to affect 3,000 to 5,000 people worldwide. People with FCS are at high risk of unpredictable and potentially fatal acute pancreatitis. In addition to pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage, including chronic pancreatitis and pancreatogenic (type 3c) diabetes. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognition that affect their ability to work. People with FCS also report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog. Additional information on FCS is available at www.fcsfocus.com, through the LPLD Alliance at http://www.lpldalliance.org and through The FCS Foundation at http://www.livingwithfcs.org. For a full list of organizations supporting the FCS community worldwide, please click here.

AKCEA-TTR-L

Generation 2+ LICA antisense drug

AKCEA-TTR-LRx is is a Generation 2+ ligand-conjugated antisense (LICA) drug designed to reduce the production of transthyretin, or TTR protein, to treat all types of TTR amyloidosis (ATTR), a systemic, progressive and fatal disease. In patients with ATTR. Both the mutant and wild type (wt) TTR protein builds up as fibrils in tissues, such as the peripheral nerves, heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid and bone marrow. The presence of TTR fibrils interferes with the normal functions of these tissues. As the TTR protein fibrils enlarge, more tissue damage occurs and the disease worsens, resulting in poor quality of life and eventually death.

About TTR Amyloidosis (ATTR)

ATTR amyloidosis is a systemic, progressive and fatal disease in which patients experience multiple overlapping clinical manifestations caused by the inappropriate formation and aggregation of TTR amyloid deposits in various tissues and organs, including peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone marrow. The progressive accumulation of TTR amyloid deposits in these tissues and organs leads to organ failure and eventually death.

Polyneuropathy due to hATTR is caused by the accumulation of misfolded mutated TTR protein in the peripheral nerves. Patients with polyneuropathy due to hATTR experience ongoing debilitating nerve damage throughout their body resulting in the progressive loss of motor functions, such as walking. These patients also accumulate TTR in other major organs, which progressively compromise their function and eventually leading to death within five to fifteen years of disease onset. There are an estimated 10,000 patients with polyneuropathy due to hATTR worldwide.

ATTR cardiomyopathy is caused by the accumulation of misfolded TTR protein in the cardiac muscle. Patients experience ongoing debilitating heart damage resulting in progressive heart failure, which results in death within 3 to 5 years from disease onset. ATTR cardiomyopathy includes both the genetic and wild-type form of the disease. There are an estimated 240,000 patients with ATTR cardiomyopathy worldwide.

Often patients with the polyneuropathy form of TTR amyloidosis will also have TTR build up in the heart and also experience cardiomyopathy symptoms. Similarly, patients with the cardiomyopathy form of TTR amyloidosis may often have TTR build up in their peripheral nerves and can experience nerve damage and progressive difficulty with motor functions.

IONIS-FB-L

Generation 2+ LICA antisense drug
IONIS-FB-LRx is a Generation 2+ ligand-conjugated antisense (LICA) drug designed to reduce the production of complement factor B (FB). Genetic association studies have shown that overaction of this cascade has been associated with the development of several complement-mediated diseases, including IgA nephropathy (IgAN). FB, a key regulatory protein, is produced predominately in the liver and circulates at high levels throughout the vascular system, including in the kidney.

About IgA Nephropathy (IgAN)
IgA Nephropathy (IgAN) is the most prevalent primary chronic glomerulonephritis worldwide and is an important cause of chronic kidney disease and renal failure. Also known as Berger’s disease, IgAN is characterized by immunodeposits with dominant or codominant IgA in the glomerular mesangium of the kidneys, resulting in inflammation and tissue damage. Although IgAN may occur at any age, it generally presents in the second or third decade of life. The clinical presentation, disease progression and histologic findings are highly variable among affected individuals. Current therapies are aimed at reduction in proteinuria with administration of angiotensin inhibitors and control of blood pressure. Sometimes immunosuppressive therapies are given; however, this practice is not universally accepted.

ION839

Generation 2.5 LICA antisense drug

ION839, formerly known as IONIS-AZ6-2.5-LRx and AZD2693.

ION859

Generation 2+ antisense drug

ION859 (formerly IONIS-BIIB7Rx), also known as BIIB094, is an antisense oligonucleotide (ASO) targeting Leucine Rich Repeat Kinase 2 (LRRK2) messenger ribonucleic acid (mRNA). ION859 is designed to prevent the production of LRRK2 protein and is being developed as a potential therapy for Parkinson’s disease (PD). The most common genetic mutations in PD cases are found in the LRRK2 gene. Increased LRRK2 protein activity is hypothesized to be one of the key drivers of PD pathogenesis. It is hypothesized that reduction of LRRK2 mRNA and, subsequently, reduced synthesis of LRRK2 protein will ameliorate the toxic effects of gain-of-function mutations as well as the primary pathology in PD patients without the LRRK2 mutation.

About Parkinson’s Disease
Parkinson’s disease is a progressive neurodegenerative disease characterized by loss of neurons in the motor system. Patient’s with Parkinson’s disease can experience tremors, loss of balance and coordination, stiffness, slowing of movement, changes in speech and in some cases cognitive decline. Parkinson’s disease is ultimately fatal. There are treatments that can relieve symptoms, but there is no disease modifying therapy. The exact cause is unknown, but it is believed to be a combination of genetics and environmental factors. There are known hereditary mutations that cause Parkinson’s disease, including dominantly inherited gain-of-function mutations in the LRRK2 gene.