ARWR

Arrowhead Pharmaceuticals develops RNA interference therapeutics using proprietary delivery technologies to silence disease-causing genes across diverse therapeutic areas including liver diseases, pulmonary conditions, and rare genetic disorders. Headquartered in Pasadena, California, Arrowhead's platform enables targeted delivery of small interfering RNA (siRNA) molecules that selectively reduce production of specific disease-associated proteins, potentially treating conditions where reducing rather than blocking protein function offers therapeutic benefit. The company's lead commercial asset, PLOZASIRAN, recently received FDA approval for treating severe hypertriglyceridemia and familial chylomicronemia syndrome, conditions causing dangerously elevated blood triglyceride levels. Arrowhead's clinical pipeline includes therapies for cardiovascular disease (targeting ANGPTL3 and Lp(a)), chronic hepatitis B virus infection using multi-pronged approach silencing multiple viral and host targets, and alpha-1 antitrypsin deficiency-associated liver disease. The company's Targeted RNAi Molecule (TRiM) platform incorporates targeting ligands enabling selective delivery to affected tissues while minimizing off-target effects, addressing a key challenge limiting earlier RNAi therapeutic approaches. Arrowhead established collaborations with Takeda Pharmaceutical, Amgen, and Horizon Therapeutics providing significant upfront payments, development funding, and milestone payments supporting pipeline advancement. The company employs approximately 400 people and invests heavily in manufacturing capabilities producing clinical-grade siRNA therapeutics supporting advancing trials and potential commercial launches.