Wave Life Sciences Announces Submission of First Clinical Trial Application for WVE-006, the First-ever RNA Editing Clinical Candidate, and Plans for Upcoming Virtual “R&D Day”

WVE-006 is the first-ever RNA editing program to enter clinical development and is designed to restore production and circulation of functional, wild-type alpha-1 antitrypsin (AAT) protein and reduce levels of mutant Z-AAT protein, thereby addressing alpha-1 antitrypsin deficiency-related lung disease, liver disease or both

Wave expects to initiate dosing in the first-in-human clinical program in 4Q 2023 and deliver AAT protein restoration data from AATD patients treated with WVE-006 in 2024

Under its collaboration with GSK, Wave is eligible to receive substantial milestone payments for WVE-006 in 2023 and beyond

Wave plans to host a virtual “R&D Day” on September 28, 2023; topics to include the WVE-006 clinical program and how Wave is extending its leadership in RNA editing with additional programs

CAMBRIDGE, Mass., Sept. 05, 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 the submission of its first clinical trial application (CTA) for WVE-006 in alpha-1 antitrypsin deficiency (AATD). WVE-006 is a first-in-class, GalNAc-conjugated RNA editing oligonucleotide (“AIMer”) and is designed to correct the single base mutation in messenger RNA (mRNA) coded by the SERPINA1 Z allele, thereby enabling restoration and circulation of functional, wild-type alpha-1 antitrypsin (M-AAT) protein. The WVE-006 clinical program will be highlighted in Wave’s virtual “R&D Day” on September 28, 2023 at 10:00 a.m. ET, among other programs.

“With the submission of the first CTA for WVE-006, we have officially initiated clinical development of the industry’s first-ever RNA editing therapeutic candidate,” said Anne Marie Li-Kwai-Cheung, Chief Development Officer at Wave Life Sciences. “We designed WVE-006 to correct the most common underlying genetic mutation that causes AATD, providing an innovative therapeutic option for individuals with lung disease, liver disease or both. Indeed, our preclinical data support this profile, with mouse models showing restored AAT protein well above 11 micromolar, as well as improvement in several markers of liver disease and inhibition of neutrophil elastase. As a GalNAc-RNA editing oligonucleotide, WVE-006 is reversible and re-dosable, with potential for infrequent subcutaneous dosing. WVE-006 is highly specific with no evidence of bystander editing and, by virtue of the mechanism of action, no permanent changes to the genome that occur with DNA-targeting approaches. For these reasons, we believe WVE-006 has potential to revolutionize how AATD is treated.”