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Phase 1/2 study of AIR-001

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Trial Profile

Phase 1/2 study of AIR-001

Status: Planning
Phase of Trial: Phase I/II

Latest Information Update: 19 May 2025

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At a glance

  • Drugs AIR-001-AiRNA-Pharmaceuticals (Primary)
  • Indications Alpha 1-antitrypsin deficiency
  • Focus Adverse reactions; Pharmacodynamics; Therapeutic Use

Most Recent Events

  • 13 May 2025 According to AiRNA Pharmaceuticals media release, AIR-001, and AIRNA expects to file a clinical trial application in 2H 2025.
  • 15 Apr 2025 New trial record
  • 01 Apr 2025 According to AiRNA Pharmaceuticals media release, company aannounced the closing of an oversubscribed $155 million Series B financing. AIRNA will use the proceeds to advance its lead RNA editing product candidate (AIR-001) for AATD into a Phase 1/2 clinical trial.

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