Drug Profile

AIR 001 - AiRNA Pharmaceuticals

Alternative Names: AIR-001 - AiRNA Pharmaceuticals

Latest Information Update: 27 Mar 2026

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At a glance

Originator AiRNA Pharmaceuticals
Class Oligonucleotides
Mechanism of Action Alpha 1-antitrypsin expression modulators; Gene silencing

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes
Available For Licensing Yes

Highest Development Phases

Phase I Alpha 1-antitrypsin deficiency

Most Recent Events

27 Feb 2026 Phase-I clinical trials in Alpha 1-antitrypsin deficiency in USA, Australia (SC) (NCT07431112)
02 Oct 2025 AiRNA Pharmaceuticals plans a clinical trial in Alpha 1-antitrypsin deficiency (Parenteral)
13 May 2025 Pharmacodynamics, adverse events and pharmacokinetics data from preclinical studies Alpha-1 antitrypsin deficiency released by AiRNA Pharmaceuticals

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