Wave Life Sciences initiates dosing in the RestorAATion clinical trial program

Wave Life Sciences announced the initiation of dosing in healthy volunteers in the RestorAATion clinical trial program, which is investigating WVE-006 as a potential treatment for alpha-1 antitrypsin deficiency or AATD. WVE-006 is a first-in-class, GalNAc-conjugated RNA editing oligonucleotide, AIMer . It is designed to restore circulation of healthy, wild-type alpha-1 antitrypsin, M-AAT, protein and reduce dysfunctional Z-AAT protein, thereby potentially addressing AATD-related lung disease, liver disease, or both. “Initiating dosing in RestorAATion represents an important milestone for the alpha-1 community, where treatment options are limited and there are no medicines that address the underlying genetic mutation that most commonly causes AATD. In preclinical studies, WVE-006 led to potent and durable RNA editing and restoration of AAT protein up to 30 micromolar, underscoring the impact of our novel chemistry. WVE-006 has the potential to transform the treatment paradigm for this disease, and we are well-positioned to achieve this vision as part of our collaboration with GSK,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “Moreover, with the first RNA editing therapeutic to ever be dosed in humans, we are making a significant contribution to the scientific field by bringing an entirely new class of medicines into clinical development, and we expect to continue unlocking the potential of RNA editing more broadly.”