Wave Life Sciences Ltd. (NASDAQ:WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today presented new data for its ADAR-mediated RNA editing capability (ADAR editing), including new preclinical editing data across multiple tissues, as well as an update on its discovery-stage alpha-1 antitrypsin deficiency (AATD) program during the company’s 2021 Analyst and Investor Research Webcast. The webcast also included updates on the company’s PRISM™ platform and initial results from the application of ADAR editing to neurology targets.
“Our presentations today represent robust and meaningful contributions to the rapidly advancing field of RNA editing, where we are at the forefront defining both new levels of editing, as well as the tissues and cell types amenable to this approach,” said Chandra Vargeese, PhD, Chief Technology Officer of Wave Life Sciences. “The application of PRISM to RNA editing means that there is the potential for therapeutic applications extending beyond the restoration of protein function, such as upregulation of protein expression, modification of protein function by altering post-translational modifications or protein-protein interactions, or alteration of protein stability. Additionally, with our AATD program, we have shown an ability in preclinical experiments to drive alpha-1 antitrypsin protein significantly above levels that are potentially therapeutically meaningful and increase the overall percentage of secreted wild-type M-AAT protein.”
A summary of the RNA editing presentations is below. A replay of the Analyst and Investor Research Webcast is available on Wave’s Investor Relations website.
