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Wave Life Sciences Achieves First Human RNA Editing in AATD Trial, Shares Surge 75%

Oct 16, 2024, 03:26 PM

Wave Life Sciences announced that it has achieved the first-ever therapeutic RNA editing in humans in its RestorAATion-2 trial of WVE-006 for Alpha-1 Antitrypsin Deficiency (AATD). A single subcutaneous dose—the first and lowest dose tested—of WVE-006 in patients with homozygous “ZZ” AATD resulted in mean plasma total AAT levels of approximately 11 micromolar, with mean wild-type M-AAT representing 6% of normal levels. This milestone, achieved after over 10 years of oligonucleotide innovation at Wave, validates RNA editing as a therapeutic approach and demonstrates the company's innovative chemistry. Following the announcement, shares of Wave Life Sciences surged by up to 75%. Other companies in the RNA editing field, including ProQR Therapeutics and Korro Bio, also experienced significant stock price increases, with ProQR up 75% and Korro Bio up 126%. Additionally, GSK increased its stake in Wave Life Sciences by purchasing 2.8 million shares at $8 per share.