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ARO-AAT (Alpha-1 Liver Disease)Alpha-1 Liver DiseasePartnered with Takeda
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ARO-APOC3 (Hypertriglyceridemia)Hypertriglyceridemia
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ARO-ANG3 (Dyslipidemia)Dyslipidemia
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ARO-PNPLA3 (NASH)NASH
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GSK4532990 (NASH)NASHLicensed to GlaxoSmithKline
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ARO-C3 (Complement Mediated Disease)Complement Mediated Disease
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ARO-ENaC2 (Cystic Fibrosis)Cystic Fibrosis
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ARO-MUC5AC (Muco-obstructive)Muco-obstructive
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ARO-RAGE (Inflammatory)Inflammatory
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ARO-MMP7 (Idiopathic Pulmonary Fibrosis)Idiopathic Pulmonary Fibrosis
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ARO-COV (COVID-19 )COVID-19
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ARO-DUX4 (FSHD)FSHD
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ARO-SOD1 (ALS)ALS
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HZN-457 (Gout)GoutPartnered with Horizon
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JNJ-3989 (Hepatitis B)Hepatitis BLicensed to Janssen
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Olpasiran (Cardiovascular Disease)Cardiovascular DiseaseLicensed to Amgen
- Liver
- Lung
- Muscle
- CNS
“20 in 25” Pipeline Development Strategy
There is no shortage of need that we can endeavor to serve and no shortage of lives that can be touched by the versatility of our RNAi technologies. We are acutely aware of the urgent need to develop solutions for the many diseases that have genetic targets that are not otherwise druggable by small molecules or biologics. That is why we have embraced the bold goal of having 20 individual drugs, either partnered or wholly owned, in clinical trial or on the market in 2025. Below are the drugs currently in development with more to come shortly from our prolific R&D organization.

ARO-AAT
ARO-AAT is being developed to treat the liver disease associated with alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder that severely damages the liver and lungs of affected individuals.
ARO-AAT is designed to knock down the hepatic production of the mutant alpha-1 antitrypsin (Z-AAT) protein, the cause of progressive liver disease in AATD patients. Reducing production of the inflammatory Z-AAT protein is expected to halt the progression of liver disease and potentially allow it to regenerate and repair.
Read More »ARO-APOC3
ARO-APOC3 is designed to reduce production of Apolipoprotein C-III (apoC-III), a component of triglyceride rich lipoproteins (TRLs) including VLDL and chylomicrons and is a key regulator of triglyceride metabolism. The company believes that knocking down the hepatic production of apoC-III may result in reduced VLDL synthesis and assembly, enhanced breakdown of TRLs, and better clearance of VLDL and chylomicron remnants.
Read More »ARO-ANG3
ARO-ANG3 is designed to reduce production of angiopoietin-like protein 3 (ANGPTL3), a liver synthesized inhibitor of lipoprotein lipase and endothelial lipase. ANGPTL3 inhibition has been shown to lower serum LDL, serum and liver triglyceride and has genetic validation as a novel target for cardiovascular disease.
Read More »ARO-PNPLA3
ARO-PNPLA3, formerly called JNJ-75220795, is an investigational RNAi therapeutic designed to reduce liver expression of patatin-like phospholipase domain containing 3 (PNPLA3) as a potential treatment for patients with non-alcoholic steatohepatitis (NASH). PNPLA3 has strong genetic and preclinical validation as a driver of fat accumulation and damage in the livers of patients who carry the common I148M mutation.
Read More »GSK4532990
GSK4532990, formerly ARO-HSD, targets HSD17B13, a member of the hydroxysteroid dehydrogenase family involved in the metabolism of hormones, fatty acids, and bile acids. Published human genetic data indicate that a loss of function mutation in HSD17B13 provides strong protection against alcoholic hepatitis, cirrhosis, and NASH, with approximately 30-50% risk reduction compared to non-carriers. GSK4532990 is being developed under the November 2021 exclusive license agreement between Arrowhead and GSK.
Read More »ARO-C3
ARO-C3 is designed to reduce production of complement component 3 as a potential treatment for various complement mediated diseases.
Read More »ARO-ENaC2
ARO-ENaC2 is designed to reduce production of the epithelial sodium channel alpha subunit (αENaC) in the airways of the lung. In cystic fibrosis patients, increased ENaC activity contributes to airway dehydration and reduced mucociliary transport.
Read More »ARO-MUC5AC
ARO-MUC5AC is designed to reduce production of mucin 5AC (MUC5AC) as a potential treatment for various muco-obstructive and inflammatory pulmonary diseases.
ARO-RAGE
ARO-RAGE is designed to reduce production of the Receptor for Advanced Glycation End products (RAGE) as a potential treatment for various muco-obstructive and inflammatory pulmonary diseases.
ARO-MMP7
ARO-MMP7 is designed to the reduce expression of matrix metalloproteinase 7 (MMP7) as a potential treatment for idiopathic pulmonary fibrosis (IPF).
ARO-COV
ARO-COV is being developed to address the current novel coronavirus that causes COVID-19 and other possible future pulmonary-borne pathogens.
ARO-DUX4
ARO-DUX4 is designed to target the gene that encodes human double homeobox 4 (DUX4) protein as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD). FSHD is an autosomal dominant disease associated with the failure to maintain complete epigenetic suppression of DUX4 expression in differentiated skeletal muscle, leading to overexpression of DUX4, which is myotoxic and can lead to muscle degeneration.
ARO-SOD1
ARO-SOD1 is designed to reduce expression of superoxide dismutase 1 (SOD1) in the CNS as a potential treatment for patients with amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations.
HZN-457
HZN-457 is a small interfering RNA (siRNA) medicine candidate conjugated to N-acetylgalactosamine (GalNAc) that selectively targets and silences xanthine dehydrogenase expression in the liver, which produces uric acid. The development of HZN-457 is through an exclusive collaboration between Arrowhead and Horizon Therapeutics plc.
JNJ-3989
JNJ-3989, formerly ARO-HBV, is being developed in collaboration with Janssen Pharmaceuticals, Inc. to be a potentially curative therapy for patients with chronic hepatitis B infection, when used in combination with other direct antivirals. JNJ-3989 is a sub-cutaneous, ribonucleic acid interference (RNAi) therapy candidate which is designed to silence all HBV gene products and intervenes upstream of the reverse transcription process where current standard-of-care nucleotide and nucleoside analogues act. The company believes this may allow the body’s natural immune defenses to clear the virus and potentially lead to a functional cure.
Read More »Olpasiran
Olpasiran (formerly AMG 890 and ARO-LPA) is designed to reduce production of apolipoprotein A, a key component of lipoprotein(a), which has been genetically linked with increased risk of cardiovascular diseases, independent of cholesterol and LDL levels. Amgen acquired a worldwide, exclusive license in September 2016, to develop and commercialize olpasiran.
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