ARWR
- Arrowhead Pharmaceuticals, Inc.
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Overview
Company Summary
Arrowhead Pharmaceuticals, Inc. is a biopharmaceutical company that primarily focuses on the development and commercialization of RNA interference (RNAi) therapeutics. RNAi is a natural biological process that regulates gene expression and plays a crucial role in various diseases.
Arrowhead Pharmaceuticals utilizes their proprietary Targeted RNAi Molecule (TRiM) platform to design and develop therapies that selectively silence disease-causing genes. By targeting and silencing specific genes, Arrowhead aims to address a wide range of diseases that had limited treatment options in the past.
The company's pipeline consists of both clinical and pre-clinical programs across multiple therapeutic areas, including liver diseases, cardio-metabolic diseases, and pulmonary diseases. The liver-focused programs primarily target conditions such as chronic hepatitis B virus (HBV) infection, alpha-1 antitrypsin deficiency (AATD), and hypertriglyceridemia.
Arrowhead Pharmaceuticals' leading clinical candidate, ARO-AAT, is being developed for the treatment of AATD-related liver disease, which leads to liver fibrosis, cirrhosis, and ultimately, liver failure. ARO-AAT works by reducing the production of mutant alpha-1 antitrypsin protein, which causes the disease.
In addition to liver-related programs, Arrowhead is also developing RNAi therapeutics for other diseases, including cardiovascular diseases, such as dyslipidemia and homozygous familial hypercholesterolemia (HoFH), and non-alcoholic steatohepatitis (NASH).
Overall, Arrowhead Pharmaceuticals, Inc. specializes in utilizing RNAi technology to develop targeted therapies that have the potential to address various diseases with unmet medical needs.