“Achieving the first-ever therapeutic RNA editing in humans is a significant milestone for our
organization, for our GSK collaboration, and for the entire oligonucleotide field. It also unlocks and derisks Wave’s RNA editing platform, in light of the continued strong clinical translation of our proprietary
best-in-class chemistry, including PN, stereochemistry and our N3U AIMer modification,” said Paul Bolno, MD, MBA, President and Chief Executive Officer
at Wave Life Sciences. “The level of mRNA editing we are observing with a single dose exceeded our expectations and we expect M-AAT levels to continue to increase with repeat dosing, based on our
preclinical data. These initial data, alongside WVE-006’s durability and convenient subcutaneous administration, are all supportive of a
best-in-class profile for WVE-006 relative to other editors and in the broader AATD space. These data also increase our
confidence in our wholly owned pipeline, including our HD, DMD and obesity programs, as well as our next RNA editing targets. We look forward to introducing the next RNA editing programs, as well as providing an update on our INHBE GalNAc-siRNA
program in obesity, at our Research Day on October 30.”
There are an estimated 200,000 individuals living with AATD in the US and Europe who are
homozygous for the SERPINA1 Z mutation. Treatment options are currently limited to weekly IV augmentation therapy for lung disease only (representing over $1.4 billion in worldwide sales in 2023). There are no approved therapies to address AATD
liver disease, which ultimately requires many individuals living with AATD to undergo liver transplantation.
GSK has the exclusive global license for WVE-006. Development and commercialization responsibilities will transfer to GSK after Wave completes the RestorAATion-2 study. In total, Wave is eligible for up to
$525 million in milestones, as well as tiered royalties on net sales, for WVE-006.
About Wave Life Sciences
Wave Life
Sciences (Nasdaq: WVE) is a biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health. Wave’s RNA medicines platform, PRISM®, combines
multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. Its toolkit of RNA-targeting modalities includes
editing, splicing, RNA interference and antisense silencing, providing Wave with unmatched capabilities for designing and sustainably delivering candidates that optimally address disease biology. Wave’s diversified pipeline includes clinical
programs in Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and Huntington’s disease, as well as a preclinical program in obesity. Driven by the calling to “Reimagine Possible”, Wave
is leading the charge toward a world in which human potential is no longer hindered by the burden of disease. Wave is headquartered in Cambridge, MA. For more information on Wave’s science, pipeline and people, please visit
www.wavelifesciences.com and follow Wave on X (formerly Twitter) and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including,
without limitation, our understanding of the anticipated therapeutic benefit of WVE-006 as a therapy for AATD and our estimates of the AATD patient population that may benefit from WVE-006; our plans and estimated timing to share multidose data from the RestorAATion-2 trial; our understanding of the mRNA being generated by
WVE-006; our expectations that M-AAT levels may to continue to increase with repeat dosing; our understanding of the safety profile of
WVE-006; potential milestone payments that we may earn for WVE-006; and the potential benefits of our RNA editing platform and our proprietary best-in-class chemistry, including PN, stereochemistry and our N3U AIMer modification. The words “may,” “will,” “could,” “would,”
“should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,”