Triglyceride and apolipoprotein-targeted disorders
Arrowhead Pharmaceuticals Inc. is active in clinical research for hypertriglyceridemia, severe hypertriglyceridemia, mixed dyslipidemia, and familial chylomicronemia syndrome, with a strong emphasis on therapies aimed at lowering circulating triglycerides and addressing acute pancreatitis risk.
- Hypertriglyceridemia and severe hypertriglyceridemia
- Familial chylomicronemia syndrome
- Mixed dyslipidemia
- Acute pancreatitis prevention
The sponsor also studies apolipoprotein C-III-directed and related lipid-lowering approaches in adults with inherited and multifactorial lipid metabolism disorders.
LDL-C reduction in inherited cholesterol disorders
Its research portfolio includes homozygous familial hypercholesterolemia, focusing on therapies intended to reduce low-density lipoprotein cholesterol in people with severe inherited cholesterol elevation.
- Homozygous familial hypercholesterolemia
- Low-density lipoprotein cholesterol lowering
- Inherited dyslipidemia
This area reflects sustained interest in cardiometabolic disease linked to genetic lipid abnormalities.
Complement-mediated and renal disease
The sponsor funds studies in complement-mediated renal disease, with attention to therapies that modulate complement pathway activity in kidney disorders.
- Complement-mediated renal disease
- Complement system modulation
- Kidney disease
These investigations place nephrology and immune-mediated renal injury within its clinical research portfolio.
Respiratory and fibrotic lung disease
Arrowhead Pharmaceuticals Inc. also supports research in asthma, chronic obstructive pulmonary disease, inflammatory lung disease, and idiopathic pulmonary fibrosis, alongside muco-obstructive lung disease.
- Asthma and chronic obstructive pulmonary disease
- Idiopathic pulmonary fibrosis
- Inflammatory lung disease
- Muco-obstructive lung disease
Its respiratory programs include interest in mucus-related airway disease and fibrotic pulmonary conditions.
Neurologic and gene-silencing research
The sponsor’s portfolio extends to spinocerebellar ataxia type 2, indicating involvement in neurologic disease research with a focus on inherited movement disorders.
- Spinocerebellar ataxia type 2
- Inherited neurologic disorders
This area broadens the company’s therapeutic scope beyond metabolic, renal, and pulmonary disease.




