Wave Life Sciences Shows Durable ADAR-Editing In Multiple Tissues In Animal Studies
- Wave Life Sciences Ltd (NASDAQ: 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.
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