Drug Profile

AIR 001 - AiRNA Pharmaceuticals

Alternative Names: AIR-001 - AiRNA Pharmaceuticals

Latest Information Update: 14 Apr 2025

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At a glance

Originator AiRNA Pharmaceuticals
Class Oligonucleotides
Mechanism of Action Gene transference

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

Preclinical Alpha 1-antitrypsin deficiency

Most Recent Events

14 Apr 2025 AIR 001 - AiRNA Pharmaceuticals is available for licensing as of 14 Apr 2025. https://airna.com/science-platform/ (AiRNA Pharmaceuticals website, April 2025)
01 Apr 2025 Preclinical trials in Alpha 1-antitrypsin deficiency in USA (Parenteral), before April 2025
01 Apr 2025 AiRNA Pharmaceuticals plans to file clinical trial application for AIR 001 for alpha-1 antitrypsin deficiency in 2H 2025

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