Wave Life Sciences Shows Durable ADAR-Editing In Multiple Tissues In Animal Studies

Wave Life Sciences Ltd WVE presented new preclinical data for its ADAR-mediated RNA editing capability (ADAR editing) and an update on its discovery-stage alpha-1 antitrypsin deficiency (AATD) program.
Related Link: Wave Life Sciences' RNA Editing Platform Shows Restoration of Functional AAT Protein in Preclinical Study
The webcast also included updates on the company's PRISM platform and initial results from the application of ADAR editing to neurology targets.
In vivo data demonstrated durable restoration of M-AAT protein in the liver of animal models following initial doses of a GalNAc-conjugated SERPINA1 AIMer.
Serum concentrations of human AAT protein remained at least three-fold higher over PBS control for 30 days post-last dose.
Wave also shared data demonstrating progress in enhancing editing activity and protein restoration following PRISM chemistry optimization.
The AIMers achieved mean editing of approximately 50% of SERPINA1 mRNA in vivo.
Also, with chemistry optimization, Wave demonstrated in vivo a four-fold increase over PBS control in AAT protein restoration in serum.
Approximately 85% of circulating AAT was confirmed to be M-AAT in treated transgenic mice.
Wave expects to have an AATD development candidate in 2022.
Read Next: Wave Life Sciences Stock Plunges After Antisense Oligonucleotide Candidates Disappoint In Huntington's Disease Study.
Price Action: WVE stock closed 1.67% higher at $5.40 during after-hours trading On Tuesday.