Wave Life Sciences to Highlight Leading RNA Editing Capability and Multimodal Discovery and Development Platform at ASGCT 26th Annual Meeting

Presentations include new data showing WVE-006 improved markers of liver disease in mice; WVE-006 is a first-in-class RNA editing therapeutic for alpha-1 antitrypsin deficiency (AATD) which is designed to restore healthy, wild-type AAT protein

Best-in-class exon skipping clinical data for WVE-N531 in Duchenne muscular dystrophy (DMD) also highlighted in oral presentation

Poster presentations will focus on Wave’s pioneering application of RNA editing to upregulate gene expression, as well as Wave’s emerging best-in-class siRNA formats

CAMBRIDGE, Mass., May 16, 2023 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced it will highlight its leading RNA editing capability and its multimodal discovery and development platform at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, taking place May 16-20, 2023, in Los Angeles, California.

New preclinical data for WVE-006, Wave’s investigational, GalNAc-conjugated RNA editing candidate for the treatment of alpha-1 antitrypsin deficiency (AATD), will be presented in an oral abstract session. These data demonstrate that treatment with WVE-006 improved several markers of liver disease in mice, including a decrease in liver inflammation and hepatocyte turnover and reduction in the size of PAS-D positive globules. WVE-006 is uniquely designed to address both liver and lung disease related to AATD and is poised to be the industry’s first RNA editing candidate to enter clinical trials, with CTA submissions by Wave expected in the second half of 2023.

Additionally, Wave will present preclinical data in a poster session regarding the ability of ADAR-mediated RNA editing to upregulate gene expression. Wave is pioneering this novel editing application with potential to address diseases with large patient populations, independent of genetic mutation status.

“The ingenuity of our scientists has resulted in a best-in-class RNA medicines platform and the industry’s most advanced and promising RNA editing capability. We are proud to be highlighting two innovative applications of our RNA editing oligonucleotides – correction of single point mutations and upregulation of gene expression – as well as our first editing clinical candidate, WVE-006, at this year’s ASGCT meeting,” said Chandra Vargeese, PhD, Chief Technology Officer at Wave Life Sciences. “Additionally, we will present on the impact of our novel chemistry in DMD, which translated to the greatest level of exon skipping ever seen in the clinic with our WVE-N531 program and has the potential to result in clinically meaningful, functional dystrophin protein in our upcoming Phase 2 study.”