04.17.2026
AIRNA (Headquarters: Cambridge, USA), a biotech startup focused on RNA editing therapeutics, has dosed the first patient in a global Phase 1 clinical trial of AIR-001 for alpha-1 antitrypsin deficiency (AATD). In addition, the FDA has granted Orphan Drug Designation to AIR-001 for the treatment of AATD.
The Phase 1 program has already received authorization from multiple national regulatory authorities and is planned as a global, multi center study enrolling patients across approximately 20 sites in 11 countries.
To view the original announcement, please visit https://airna.com/news/airna-announces-first-patient-dosed-in-phase-1-trial-of-air-001-potential-best-in-class-rna-editing-therapy-for-alpha-1-antitrypsin-deficiency-aatd/
About AIRNA
AIRNA is pioneering the discovery and development of RNA editing therapeutics to deliver on the promise of genetically defined medicines for patients with rare and common diseases. RNA editing is poised to lead the next generation of RNA therapeutics by targeting diseases not accessible through other approaches with a medicine that can be conveniently re-dosed and manufactured. AIRNA’s founders, Thorsten Stafforst (University of Tübingen) and Jin Billy Li (Stanford University), were the first to elucidate a therapeutic approach for precise editing of RNA. AIRNA has advanced this groundbreaking research to establish the RESTORE+™ RNA editing platform to restore patients’ health. AIRNA has headquarters in Cambridge, MA, with research operations in Tübingen, Germany. Further information can be found at www.airna.com.
About Fast Track Initiative:
Established in 2004, Fast Track Initiative, Inc. (FTI) is a life science venture capital firm focusing its investments on early-stage startups with a strong focus on biotech and therapeutics. FTI has a strict mission to make a ground-breaking impact on the quality of our life and human wellness through investments across a broad range of areas in the life science community including therapeutics, diagnostics, digital health, healthcare services, and more. The company provides valuable, hands-on support to its 30+ portfolio companies through business and R&D strategy, partnering, investor syndication, and more, including access to its network of professionals in Japan.
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