Company intelligence / Profile preview
AIRNA Pharmaceuticals
Cambridge, Massachusetts, United Statesairna.com ↗
- Ownership
- Private
- Development stage
- Preclinical
01
Overview
AIRNA’s lead candidate targets alpha‑1 antitrypsin deficiency using precision ADAR-mediated RNA editing. Preclinical data presented at ASGCT 2025 demonstrated effective engagement of endogenous ADAR isoforms and significant in vivo correction of disease-driving mutations. The company is preparing to enter clinical trials with this candidate, aiming for a best-in-class profile with reversible, re-dosable genetic medicine.
- Therapeutic areas
- OtherRare Diseases
- Modalities
- RNA therapyAntisense oligonucleotide (oligonucleotide-based editing guides)
- Industry
- Biotech
02
Drug pipeline
2 assets1 more asset in the full pipeline.
03
Partnerships
No major pharma partnerships announced as of June 2025; open to strategic collaborations
Beyond the preview
Go deeper on AIRNA Pharmaceuticals.
Explore clinical development, deal activity, and the competitive landscape with Gosset’s full data platform.
Clinical trials
Follow clinical development from trial design and recruitment through results.
- Trial phase
- Status
- Readouts
Licensing & deals
Explore licensing agreements, acquisitions, and the assets behind each deal.
- Partners
- Deal terms
- Milestones
Funding history
Trace financing activity and the investors behind a company’s development.
- Funding rounds
- Investors
- Capital raised
Competitive landscape
Compare peer pipelines by target, modality, and therapeutic area.
- Peer companies
- Targets
- Pipeline overlap
Technologies & patents
Explore technology platforms and the patent landscape around relevant assets.
- Platforms
- Patents
- Assignees
Research & analysis
Connect company news and source material to the questions you’re investigating.
- Company news
- Sources
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on AIRNA Pharmaceuticals.