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AIRNA
Company Type: Therapeutics
Main focus: Development of therapies based on RNA-editing
Company stage: Pre-clinical
Diseases: Various diseases
Genome-editing tool: RNA-editing based technology (RESTORE)
Funding stage: Series A
Location: Cambridge, MA
Website: https://www.linkedin.com/company/airna-bio/
Pipeline:
Partners:
AIRNA is pioneering the discovery and development of RNA-editing therapeutics for patients with rare and common diseases. The company'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. RESTORE involves the recruitment of an endogenous cell factor to edit the target RNA to treat defined genetic conditions. AIRNA is advancing its first therapeutic programme, a best-in-class product candidate to treat the inherited genetic disease alpha-1 antitrypsin deficiency (AATD), as well as a pipeline of therapeutic candidates to address multiple diseases with high unmet need.