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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.

ION253

Generation 2.5 antisense drug

ION253, formerly known as IONIS-JBI2-2.5Rx.

IONIS-HBV-L

Generation 2+ LICA antisense drug

IONIS-HBV-LRx, formerly known as IONIS-GSK6-LRx, is a Generation 2+ ligand-conjugated antisense (LICA) drug designed to reduce the production of viral proteins associated with hepatitis B virus (HBV) infection and replication, including hepatitis B surface antigen, which is present in both acute and chronic infections and is associated with a poor prognosis in patients with chronic HBV infection. IONIS-HBV-LRx is the first anti-infective drug in development that incorporates our LICA technology, which is designed to increase drug potency by enhancing drug delivery to target tissue.

About Hepatitis B virus infection

Hepatitis B virus infection is a serious health problem that can lead to significant and potentially fatal health conditions, including cirrhosis, liver failure and liver cancer. Chronic HBV infection is one of the most common persistent viral infections in the world. Currently available therapies, although effective in reducing circulating HBV in the blood, do not efficiently inhibit HBV antigen production and secretion, which are associated with poor prognosis and increased risk of liver cancer.

IONIS-HBV

Generation 2+ antisense drug

IONIS-HBVRx is an antisense drug designed to reduce the production of viral proteins associated with hepatitis B virus (HBV) infection and replication, including hepatitis B surface antigen, which is present in both acute and chronic infections and is associated with a poor prognosis in patients with chronic HBV infection. HBV infection is a serious health problem that can lead to significant and potentially fatal health conditions. Chronic HBV infection is one of the most common persistent viral infections in the world.

About Hepatitis B virus infection

Hepatitis B virus infection is a serious health problem that can lead to significant and potentially fatal health conditions, including cirrhosis, liver failure and liver cancer. Chronic HBV infection is one of the most common persistent viral infections in the world. Currently available therapies, although effective in reducing circulating HBV in the blood, do not efficiently inhibit HBV antigen production and secretion, which are associated with poor prognosis and increased risk of liver cancer.

 

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 dry age-related macular degeneration (AMD). FB, a key regulatory protein, is produced predominately in the liver and circulates at high levels throughout the vascular system, including in capillaries in the eye. 1-5

About AMD

AMD is the leading cause of central vision loss in developed countries. It is estimated that more than three million people in the United States will be affected by the disease by 2020.6. AMD is believed to be a systemic disease with local disease manifestation at the aging retinal macula. AMD gradually destroys vision in the center of the visual field due to progressive damage of the retina.7