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Wave Life Sciences Announces Initiation of Dosing in RestorAATion Clinical Program Evaluating First-Ever RNA Editing Candidate, WVE-006, for Alpha-1 Antitrypsin Deficiency
WVE-006 is uniquely designed to correct the disease-causing RNA mutation in AATD, thereby restoring circulation of wild-type M-AAT protein and reducing Z-AAT
About this update from Wave Life Sciences Ltd.
[{"type":"text","content":"WVE-006 is uniquely designed to correct the disease-causing RNA mutation in AATD, thereby restoring circulation of wild-type M-AAT protein and reducing Z-AAT protein levels to address both lung and liver manifestations of the disease Wave earned $20 million USD milestone from GSK for initiation of dosing; eligible for additional development, launch, and sales-related milestones of up to $505 million for WVE-006 Proof-of-mechanism data in individuals with AATD, as measured by restoration of M-AAT protein, expected in 2024 As shared at recent R&D Day, Wave is also advancing a pipeline of wholly owned RNA editing therapeutics beyond WVE-006 and across a range of high-impact GalNAc-hepatic and extra hepatic targets CAMBRIDGE, Mass., Dec. 06, 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 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 (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 ...