Wave Life Sciences achieved the first-ever therapeutic RNA editing in humans, specifically targeting the genetic disorder alpha-1 antitrypsin deficiency (AATD)51.
Their candidate, WVE-006, is designed to edit the mutated SERPINA1 mRNA, restore functional wild-type AAT enzyme levels in the body, and address both liver and lung manifestations of AATD13.
Recent phase 1b/2a clinical data show that multiple patient cohorts (single and multidose, 200 mg and 400 mg) reached 'therapeutically relevant' levels of the enzyme, meeting regulatory thresholds for AAT augmentation therapies35.
Despite this scientific milestone and safety profile (mild-to-moderate side effects), Wave's stock price dropped by 18.6% post-announcement, signaling investor disappointment—possibly due to anticipation for stronger efficacy or durability of results53.
In contrast to Wave, competitor Beam Therapeutics experienced a 12% stock rise following the same news cycle, reflecting shifting investor sentiment in the RNA editing field5.
Sources:
1. https://www.labiotech.eu/trends-news/first-ever-rna-editing-humans-achieved/
3. https://www.biopharmadive.com/news/wave-rna-editing-aatd-study-results-multidose/759122/
5. https://www.fiercebiotech.com/biotech/wave-rna-editing-restores-enzyme-alpha-1antitrypsin-deficiency-trial-investors-unimpressed