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Drug Target Partner PreclinicalPhase IPhase IIPhase IIIApprovedhalfandhalf
SEVERE & RARE
KYNAMRO® ApoB-100 Genzyme
 
Alicaforsen ICAM-1 Atlantic
 
ISIS-TTRRx TTR GSK
 
ISIS-SMNRx SMN2 Biogen Idec
 
ISIS-APOCIIIRx ApoCIII -
 
ATL1103 GHr ATL
 
ISIS-GCCRRx GCCR -
 
ISIS-PKKRx PKK -
 
ISIS-DMPKRx DMPK Biogen Idec
 
ISIS-HTTRx HTT Roche
 
ISIS-BIIB3Rx Undisclosed Biogen Idec
 
RG-012 miR-21 Regulus
 
CARDIOVASCULAR
KYNAMRO® ApoB-100 Genzyme
 
ISIS-APOCIIIRx ApoC-III -
 
ISIS-FXIRx Factor XI -
 
ISIS-CRPRx CRP -
 
ISIS-APO(a)Rx Apo(a) -
 
ISIS-ANGPTL3Rx ANGPTL3 -
 
ISIS-FVIIRx Factor VII -
 
METABOLIC
ISIS-GCGRRx GCGR -
 
ISIS-GCCRRx GCCR -
 
ISIS-PTP1BRx PTP-1B -
 
ISIS-FGFR4Rx FGFR4 -
 
ISIS-DGAT2Rx DGAT2 -
 
CANCER
Custirsen clusterin Teva/OncoGenex
 
ISIS-EIF4ERx eIF-4E -
 
Apatorsen Hsp27 OncoGenex
 
ISIS-STAT3Rx STAT3 AstraZeneca
 
ISIS-ARRx AR AstraZeneca
 
INFLAMMATION AND OTHER
Plazomicin Aminoglycoside Achaogen
 
EXC 001 CTGF Pfizer
 
iCo-007 C-raf kinase iCo
 
ATL1102 VLA-4 ATL
 
ISIS-HBVRx HBV GSK
 
RG-101 miR-122 Regulus
 
ISIS-GSK4Rx Undisclosed GSK
 
ISIS-GSK5Rx Undisclosed GSK
 

* Named Patient Supply

Drug:
KYNAMRO®
Indication:
Homozygous FH
KYNAMRO® has been approved by the U.S. Food and Drug Administration (FDA) as an adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (apoB), total cholesterol (TC), and non-high density lipoprotein-cholesterol (non HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH). Learn More >
Drug:
Alicaforsen
Indication:
Pouchitis

An antisense drug that inhibits the production of ICAM-1, an intracellular adhesion protein that is overexpressed in a wide variety of inflammatory disorders. Our partner, Atlantic Pharmaceuticals Limited, is developing alicaforsen for the treatment of ulcerative colitis and currently supplies alicaforsen in response to physicians’ requests under international Named Patient Supply regulations for patients with IBD.

Learn More >
Drug:
ISIS-TTRRx
Indication:
TTR Amyloidosis
An antisense drug that inhibits the production of TTR, a carrier protein that transports a thyroid hormone and retinol in the blood. In patients with TTR amyloidosis, both the mutant and normal forms of TTR can build up as fibrils in tissue. ISIS-TTRRx inhibits the production of all forms of TTR, offering a unique approach to treating TTR amyloidosis. Learn More >
Visit our TTR Study website >
Drug:
ISIS-SMNRx
Indication:
Spinal Muscular Atrophy
An antisense drug designed to treat spinal muscular atrophy. SMA occurs from a genetic deletion of a gene responsible for producing a protein critical for normal cellular function. ISIS-SMNRx increases the production of this protein by modulating the splicing of a closely related gene, thereby compensating for the underlying genetic defect. Learn More >
Drug:
ISIS-APOCIIIRx
Indication:
FCS
An antisense drug that inhibits apoC-III, a protein that is responsible for triglyceride transport in blood. High levels of apoC-III are linked to inflammation, high triglycerides, atherosclerosis and metabolic syndrome. ISIS-APOCIIIRx has been shown to significantly reduce triglycerides in patients with Familial Chylomicronemia Syndrome. Learn More >
Drug:
ATL1103
Indication:
Acromegaly
An antisense drug that inhibits the production of a receptor protein, GHr. GHr reduces the level of a circulating hormone that has been shown to contribute to various diseases. Our partner, Antisense Therapeutics Limited, is developing ATL1103 for the treatment of acromegaly, a disease characterized by abnormal growth of organs, face, hands and feet. Learn More >
Drug:
ISIS-GCCRRx
Indication:
Cushing's Syndrome
An antisense drug that targets the glucocorticoid receptor, or GCCR. Glucocorticoid hormones affect a variety of processes throughout the body, and excessive levels of glucocorticoid hormones can have a detrimental effect on many of the tissues and organs in the body. Learn More >
Drug:
ISIS-PKKRx
Indication:
Hereditary Angioedema
Antisense drug designed to prevent hereditary angioedema, or HAE, attacks. ISIS-PKKRx inhibits the production of prekallikrein, or PKK, a protein produced in the liver that plays an important role in the activation of inflammatory mediators associated with acute attacks of HAE. HAE is a rare genetic disease that is characterized by rapid and painful attacks of inflammation in the hands, feet, limbs, face, abdomen, larynx and trachea. Learn More >
Drug:
ISIS-DMPKRx
Indication:
Myotonic Dystrophy Type 1
An antisense drug designed to treat Myotonic Dystrophy Type 1 (DM1), a genetic neuromuscular disease characterized by progressive muscle atrophy, weakness and disabling muscle spasms. DM1 is unusual in that the disease is not caused by a missing or malfunctioning protein, but rather is due to a genetic defect that causes a toxic RNA to accumulate in the cell which prevents the production of a number of proteins needed for normal cellular function. ISIS-DMPKRx is designed to target the toxic RNA and reduce its accumulation thereby restoring normal cellular function. Learn More >
Drug:
ISIS-HTTRx
Indication:
Huntington’s Disease
An antisense drug designed to treat Huntington’s disease (HD), a genetic brain disorder that results in the progressive loss of mental faculties and physical control. There are currently no therapeutics approved that slow the progression of HD. ISIS-HTTRx is designed to inhibit the production of the disease causing protein and slow or stop progression of the disease. Learn More >
Drug:
ISIS-BIIB3Rx
Indication:
Neurodegenerative Disease
An antisense drug designed to inhibit an undisclosed target to treat patients with a neurodegenerative disease. ISIS-BIIB3Rx is being developed as part of our collaboration with Biogen Idec. Learn More >
Drug:
RG-012
Indication:
Alport Syndrome
RG-012 is an anti-miR, an antisense oligonucleotide microRNA inhibitor, of the microRNA, miR-21. Regulus Therapeutics is developing RG-012 to treat Alport Syndrome, a progressive disease that leads to loss of kidney function. Learn More >
Drug:
KYNAMRO®
Indication:
Severe HeFH
KYNAMRO® has been approved by the U.S. Food and Drug Administration (FDA) as an adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (apoB), total cholesterol (TC), and non-high density lipoprotein-cholesterol (non HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH). KYNAMRO is currently being evaluated in a Phase 3 study in patients wtih severe HeFH. Learn More >
Drug:
ISIS-APOCIIIRx
Indication:
Hypertriglyceridemia
An antisense drug that inhibits apoC-III, a protein that regulates triglyceride metabolism in blood. High levels of apoC-III are linked to inflammation, high triglycerides, atherosclerosis and metabolic syndrome. We are developing ISIS-APOCIIIRx to treat patients with familial chylomicronemia syndrome and severely high triglycerides either as a single agent or in combination with other triglyceride-lowering agents. Learn More >
Drug:
ISIS-FXIRx
Indication:
Clotting Disorders
An antisense drug designed to treat clotting disorders by inhibiting the production of Factor XI, a clotting factor that is produced in the liver. High levels of Factor XI are linked to heart attack, stroke and blood clots. Learn More >
Drug:
ISIS-CRPRx
Indication:
CAD
An antisense drug that inhibits the production of CRP, a protein that is dramatically elevated during inflammatory disorders. Excessive amounts of CRP have been linked to coronary artery disease. In a Phase 1 study, ISIS-CRPRx produced statistically significant reductions in CRP in subjects that entered the study with elevated levels of CRP. Learn More >
Drug:
ISIS-APO(a)Rx
Indication:
CAD
An antisense drug that inhibits the production of apo(a) in the liver and is designed to offer a direct approach to reducing Lp(a), an independent risk factor for cardiovascular disease. High levels of Lp(a) are associated with an increased risk of atherosclerosis, coronary heart disease, heart attack and stroke. Lp(a) promotes premature plaque buildup, or atherosclerosis, in arteries. Learn More >
Drug:
ISIS-ANGPTL3Rx
Indication:
Hyperlipidemia
An antisense drug that inhibits ANGPTL3, a protein that regulates lipid, glucose and energy metabolism. Humans with elevated levels of ANGPTL3 have hyperlipidemia associated with an increased risk of premature heart attacks, increased arterial wall thickness as well as multiple metabolic abnormalities, such as insulin resistance. In contrast, humans with lower levels of ANGPTL3 have lower LDL-C and triglyceride levels and a lower risk of cardiovascular disease. Learn More >
Drug:
ISIS-FVIIRx
Indication:
Clotting Disorders
An antisense drug that inhibits the production of Factor VII, a key component of the tissue factor coagulation pathway, for the treatment or prevention of thrombotic diseases without causing bleeding, which is a common side effect of currently available anti-thrombotic drugs. Learn More >
Drug:
ISIS-GCGRRx
Indication:
Diabetes
An antisense drug designed to inhibit GCGR, a receptor for the hormone, glucagon, that opposes the action of insulin and stimulates the liver to produce glucose. Learn More >
Drug:
ISIS-GCCRRx
Indication:
Diabetes
An antisense drug designed to inhibit GCCR, a receptor for the hormone, glucacorticoid, that has a variety of effects throughout the body, including promoting liver glucose production and fat storage. ISIS-GCCRRx may also be useful in patients with Cushing's Syndrome where there is an excess of glucocorticoid hormones. Learn More >
Drug:
ISIS-PTP1BRx
Indication:
Diabetes
An antisense drug that inhibits the production of PTP-1B, a protein that negatively regulates insulin receptor signaling. PTP-1B is responsible for turning off the activated insulin receptor, so reducing PTP-1B enhances insulin activity. We have reported positive Phase 2 data showing that antisense inhibition of PTP-1B improved glucose control in patients with type 2 diabetes. Learn More >
Drug:
ISIS-FGFR4Rx
Indication:
Obesity
An antisense drug designed to treat obesity by inhibiting the production of FGFR4, a protein that plays an important role in the regulation of fat burning and body weight. Learn More >
Drug:
ISIS-DGAT2Rx
Indication:
NASH
An antisense drug that inhibits the production of DGAT2 and is designed to reduce liver fat in patients with nonalcoholic steatohepatitis (NASH), a common and often asymptomatic liver disease that can cause irreversible damage to the liver, and lead to liver cirrhosis and cancer. Learn More >
Drug:
Custirsen
Indication:
Cancer
An antisense drug that inhibits the production of clusterin, a secreted protein that acts as a cell-survival protein and is overexpressed in response to cancer treatments, like chemotherapy, hormone ablation and radiation therapy. Teva and OncoGenex are developing Custirsen (OGX-011) for the treatment of cancer. Custirsin has shown promising results in combination with currently available chemotherapies in several tumor types. Learn More >
Drug:
ISIS-EIF4ERx
Indication:
Cancer
An antisense drug that inhibits the production of eIF-4E, a protein that is overexpressed in a variety of cancers. eIF-4E is involved in key growth and survival factors that contribute to tumor progression and the spread of cancer. Learn More >
Drug:
Apatorsen
Indication:
Cancer
An antisense drug that inhibits the production of Hsp27, a cell-survival protein that is overexpressed in response to many cancer treatments. Our partner, OncoGenex, is developing apatorsen for the treatment of a variety of cancers. Learn More >
Drug:
ISIS-STAT3Rx
Indication:
Cancer
An antisense drug that inhibits the production of STAT3. STAT3 is a protein involved in the translation of key factors that are critical for tumor cell growth and survival. STAT3 is overactive in a variety of cancers, including ovarian, brain, lung, breast, bone, liver and multiple myeloma. Learn More >
Drug:
ISIS-ARRx
Indication:
Prostate Cancer
An antisense drug that inhibits the production of androgen receptor for the treatment of prostate cancer. Androgen receptor (AR) function is involved in disease progression for all stages of prostate cancer, and prostate cancer growth, proliferation and progression are all androgen-dependent. As such, ISIS-ARRx has the potential to be an effective treatment for all stages of prostate cancer, including prostate cancer patients who are resistant to current therapies. Learn More >
Drug:
Plazomicin
Indication:
Severe Bacterial Infection
Plazomicin (formerly ACHN-490): An aminoglycoside drug discovered by Achaogen based on technology we licensed to Achaogen. Achaogen is developing plazomicin for the treatment of patients with serious multi-drug resistant gram-negative bacterial infections. Learn More >
Drug:
EXC 001
Indication:
Local Fibrosis
An antisense drug that inhibits the production of the growth factor CTGF. CTGF is overexpressed in damaged skin or tissue following a traumatic event. In 2011, our partner, Excaliard, was acquired by Pfizer. Pfizer is developing EXC 001 for the local treatment of fibrotic diseases, including scarring. In 2011, Excaliard reported data from three Phase 2 studies of EXC 001 demonstrating that treatment with EXC 001 produced statistically significant decreases in scar severity. Learn More >
Drug:
iCo-007
Indication:
Ocular Disease
An antisense drug that inhibits the production of c-raf kinase, a protein associated with the reduction and leakage of new blood vessels in the eye. Our partner, iCo Therapeutics, is developing iCo-007 for the treatment of both diabetic macular edema and diabetic retinopathy. Learn More >
Drug:
ATL1102
Indication:
Multiple Sclerosis
An antisense drug that inhibits the production of CD49d. CD49d is a subunit of VLA-4 and is involved in a number of inflammatory diseases. Our partner, Antisense Therapeutics Limited, is developing ATL1102 for the treatment of multiple sclerosis. Learn More >
Drug:
ISIS-HBVRx
Indication:
Hepatitis B Virus Infection
An antisense drug designed to reduce the production of viral proteins associated with hepatitis B virus (HBV) infection and replication. ISIS-HBVRx is part of Isis’ alliance with GSK and is currently being evaluated in a Phase 1 clinical study. Learn More >
Drug:
RG-101
Indication:
Chronic Hepatitis C
Virus Infection
RG-101 is an anti-miR, an antisense oligonucleotide microRNA inhibitor, of the microRNA, miR-122. Regulus Therapeutics is developing RG-101 to treat chronic hepatitis c virus. Learn More >
Drug:
ISIS-GSK4Rx
Indication:
Ocular Disease
An antisense drug to an undisclosed target designed to treat an ocular disease. ISIS-GSK4Rx is being developed as part of our collaboration with GSK. Learn More >
Drug:
ISIS-GSK5Rx
Indication:
Ocular Disease
An antisense drug designed to inhibit an undisclosed target to treat patients with an ocular disease. ISIS-GSK5Rx is being developed as part of our collaboration with GSK. Learn More >