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Wave Life Sciences Achieves First-Ever Therapeutic RNA Editing in Humans, Stock Surges 55%

Oct 16, 2024, 02:44 PM

Wave Life Sciences announced a groundbreaking achievement in the field of RNA editing with their RestorAATion-2 trial of WVE-006. The trial, which targets Alpha-1 Antitrypsin Deficiency (AATD), demonstrated the first-ever therapeutic RNA editing in humans. A single subcutaneous dose of WVE-006 in the first two patients resulted in mean plasma total AAT levels of approximately 11 micromolar, with a mean wild-type M-AAT protein increase to 6%. This milestone has led to a significant surge in Wave Life Sciences' stock, which rose by 55%. The achievement is seen as a major de-risking of the new modality and has potential implications for other companies in the RNA editing space, including PRQR and KRRO. Wave Life Sciences will host a Virtual R&D day on October 30th to discuss this breakthrough, which is the result of over 10 years of oligonucleotide innovation.