RNA editing

AIRNA’s lead candidate AIR-001 works by correcting the most common pathologic mutation driving the rare disease alpha-1 antitrypsin deficiency.

AI offers tremendous potential but there are critical and time-consuming flaws in black box AI predictions.

On the agenda for the FDA this month are two RNA-based treatments for rare diseases.

With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery breakthroughs are needed to propel RNA editing to the next stage.