Wave Life announces approval of first CTA for RestorAAtion-2 trial

Wave Life Sciences announced the approval of its first clinical trial application, CTA, for its RestorAATion-2 clinical trial of WVE-006, the company’s first-in-class RNA editing oligonucleotide, which is being developed for the treatment of alpha-1 antitrypsin deficiency, AATD. WVE-006 is GalNAc-conjugated and subcutaneously administered; it does not use a lipid nanoparticle delivery system. “The approval of our first CTA for the RestorAATion-2 clinical trial of WVE-006 marks an important milestone as we continue extending our leadership in RNA editing. It is also important for the alpha-1 community as WVE-006 has the potential to enable correction of the disease-causing RNA mutation and provide a single therapeutic option regardless of whether patients have AATD liver disease, lung disease or both,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “Our rapid progress in dose escalating healthy volunteers enabled us to demonstrate the translation of safety and pharmacokinetics of WVE-006 in humans and quickly identify a starting dose level that, based on preclinical data, is expected to engage the target in patients. With proof-of-mechanism data from RestorAATion-2 expected later this year, we look forward to the opportunity to provide clinical demonstration of RNA editing and proof-of-concept for our wholly owned pipeline of RNA editing candidates.”