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Arrowhead Receives Orphan Drug Designation for ARC-AAT

PASADENA, Calif.–(BUSINESS WIRE)– Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced the United States Food and Drug Administration (FDA) has granted ARC-AAT orphan drug designation. ARC-AAT is Arrowhead’s RNAi-based therapeutic candidate being investigated for the treatment of liver disease associated with Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disease that severely damages the liver and lungs of affected children and adults. Arrowhead is currently conducting part B of a Phase 1 study of ARC-AAT in patients with PiZZ genotype AATD.

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