WVE - Wave Life Sciences posts proof-of-concept data from genetic disorder study

Wave Life Sciences (WVE) announces the first proof-of-concept in vivo data for RNA editing using endogenous ADAR enzymes in genetic disorder alpha-1 antitrypsin deficiency ((AATD)).ADAR editing resulted in therapeutically meaningful restoration of circulating functional AAT protein, the company said.The initial in vivo study utilized Wave’s proprietary transgenic mouse model, which has both the human SERPINA1 Z-allele as well as human ADAR that is expressed comparably to human cells.Up to 40% editing of human SERPINA1 Z-allele mRNA in the liver was observed at a single timepoint, which resulted in a therapeutically meaningful increase in circulating functional wild-type AAT protein.Wave’s preclinical studies for its AATD program are ongoing and additional data on durability and dose response are expected in the second half of 2021.

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Wave Life Sciences posts proof-of-concept data from genetic disorder study