“We have rapidly advanced our RestorAATion-2 study following positive proof-of mechanism data last year where we observed mean total AAT protein that met the level that has been the basis for regulatory approval for AAT augmentation therapies following a single, lowest planned dose of WVE-006, our GalNAc-RNA editing candidate for AATD,” said Paul Bolno, MD, MBA, President and Chief Executive Officer at Wave Life Sciences. “We remain on track to share two comprehensive data sets from our RestorAATion-2 trial this year, beginning with multidose data in the third quarter, which will inform the therapeutic potential of WVE-006, and our pipeline of wholly-owned GalNAc-RNA editing programs.”
Dr. Bolno continued, “We are also advancing our INLIGHT trial of WVE-007, our INHBE GalNAc-siRNA that uses our propriety chemistry and best-in-class design, in individuals living with overweight and obesity. Since our last update, we have expanded Cohort 2 based on the favorable safety and tolerability data and robust target engagement we observed in Cohort 1, our lowest single dose cohort, and dosed 24 additional patients in Cohort 2. This achievement confirms the successful clinical translation of our siRNA platform and preclinical modeling. It also strengthens our conviction in upcoming data from Cohort 2, which tests a dose that is projected to be therapeutically active based on preclinical weight loss data. Additionally, WVE-N531, for DMD, and WVE-003, for HD, remain on track, both of which have demonstrated potential best-in-class therapeutic profiles. Our consistent execution in the clinic has positioned us to deliver multiple robust data sets in the second half of 2025 that carry the potential to further extend our leadership in RNA medicines.”
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