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Wave Life Sciences Ltd (NASDAQ: WVE) has announced the first proof-of-concept preclinical data for its ADAR (adenosine deaminases acting on RNA)-mediated RNA editing program in alpha-1 antitrypsin deficiency (AATD).
AATD is a genetic disorder that may result in lung disease or liver disease.
Up to 40% editing of human SERPINA1 Z-allele mRNA in the liver was observed at a single time point, which resulted in a therapeutically meaningful increase in circulating functional wild-type AAT protein.
Editing was highly specific, with no bystander edits.
A three-fold increase in circulating human AAT compared with placebo was observed, similar to the fold difference seen between patients with the highest risk of disease and patients with lower risk of disease.
75% of circulating AAT protein was confirmed as wild-type protein M-AAT. It also suggested a reduction of misfolded protein (Z-AAT) in the liver and serum.
The company expects to present additional in vivo data in the second half of 2021.
In March, the company's antisense oligonucleotide program crashed in an early-stage Huntington's disease study and wiped out two experimental drug candidates.
Price Action: WVE shares closed at $6.73 on Wednesday.
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