AIR-001

AIR-001 refers to two distinct investigational drugs in development by different companies: 1. **AIRNA Pharmaceuticals' AIR-001** is an RNA editing therapeutic designed to treat alpha-1 antitrypsin deficiency (AATD). It precisely repairs the most common and harmful SERPINA1 gene mutation (PiZ), restoring production of functional wild-type M-AAT protein. The drug is optimized for potent and durable M-AAT production, convenient subcutaneous dosing, and a favorable safety profile. It utilizes oligonucleotide-based RNA editing technology delivered via GalNAc conjugation for targeted delivery to hepatocytes[8][9][10]. The mechanism involves gene transference through precise RNA base editing. 2. **Aires/Mast Therapeutics' AIR-001** is an inhaled sodium nitrite solution developed for pulmonary arterial hypertension (PAH) and heart failure with preserved ejection fraction (HFpEF). Its mechanism relies on the conversion of nitrite to nitric oxide in vivo, independent of nitric oxide synthase pathways—especially under hypoxic or acidic conditions—leading to vasodilation and improved hemodynamics[7]. This formulation was investigated as a small molecule inhalant but has not been approved for any indication[1][7].

Other names

AIR001AIR-001AIR 001rAIR-100 (research candidate related to development)