AIRNA nets $155M series B to launch clinical test of RNA editing tech

AIRNA, a biotechnology company focused on RNA editing, has raised $155 million in a Series B funding round to advance its lead candidate into clinical trials for a rare genetic disease2 14.

Key Details

- Funding Amount:
$155 million Series B2 14

- Lead Candidate:
AIR-001, targeting alpha-1 antitrypsin deficiency (AATD)14 16

- Technology:
RNA editing using chemically optimized guide RNAs to direct ADAR enzymes to specific RNA targets14

Technology Overview

AIRNA's platform utilizes adenosine deaminase acting on RNA (ADAR) enzymes to make targeted adenosine-to-inosine edits in RNA13 14. This approach aims to provide a reversible and potentially safer alternative to DNA editing for treating genetic diseases1 3.

Lead Program

The company's lead candidate, AIR-001, is designed to correct the most common mutation associated with AATD in the SERPINA1 gene17. By repairing this mutation, AIRNA hopes to restore production of functional alpha-1 antitrypsin protein in patients17.

Advantages of RNA Editing

Compared to DNA editing approaches, RNA editing offers several potential benefits:

Reversibility and dose adjustability3
Potentially earlier intervention in disease progression3
Improved specificity for certain targets like SERPINA13

Next Steps

With this new funding, AIRNA plans to initiate its first clinical trial later this year, testing AIR-001 in AATD patients16. The company aims to demonstrate the therapeutic potential of its RNA editing platform in treating both rare and common genetic conditions3.