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Wednesday, October 16, 2024

Wave Life Sciences 1st Therapeutic RNA Editing in Humans for Alpha-1 Antitrypsin Deficiency

 Achieved proof-of-mechanism for Wave’s RNA editing platform; restoring levels of wild-type (edited) M-AAT that are consistent with the heterozygous “MZ” genotype with low risk of AATD lung and liver disease

A single subcutaneous dose of WVE-006 in the first two patients with homozygous “ZZ” AATD resulted in mean plasma total AAT levels of ~11 micromolar, with mean wild-type M-AAT representing more than 60% of total AAT; durable editing with M-AAT protein observed through 57 days

Wave expects to share multidose data from the RestorAATion-2 trial in 2025

Annual Research Day on October 30th will highlight WVE-006 proof-of-mechanism data and Wave’s new wholly owned GalNAc-RNA editing programs, in addition to an obesity program update with new preclinical data for WVE-007 (INHBE GalNAc-siRNA)

https://www.globenewswire.com/news-release/2024/10/16/2964053/0/en/Wave-Life-Sciences-Announces-First-Ever-Therapeutic-RNA-Editing-in-Humans-Achieved-in-RestorAATion-2-Trial-of-WVE-006-in-Alpha-1-Antitrypsin-Deficiency.html

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