Wave Life Sciences (NASDAQ: WVE) has marked a significant breakthrough in RNA-editing therapies with its alpha-1 antitrypsin deficiency (AATD) candidate, WVE-006. In an early-phase clinical trial, the therapy delivered the first clinical proof-of-mechanism for RNA editing in humans, a pioneering achievement in the field. WVE-006, a GalNAc-conjugated, subcutaneous A-to-I RNA editing oligonucleotide (AIMer), is being developed to address AATD-related lung and liver diseases. Currently, no approved therapies for AATD exist, leaving patients to rely on invasive options such as liver transplants.
This innovation stems from Wave’s collaboration with GlaxoSmithKline (LSE: GSK), a partnership inked in December 2022. Under the agreement, GSK secured an exclusive global license for WVE-006, committing up to $525 million in milestone payments across development, launch, and sales stages, along with tiered royalties.
Following the release of the positive trial data, analysts have raised their expectations for WVE-006. Visible Alpha consensus shows that analysts now project peak global sales of $272 million by 2035, up from $163 million. The probability of success (POS) for the drug is currently estimated at 47%. Although WVE-006 remains years away from market readiness, the results are a landmark for RNA-editing technology, offering early validation and paving the way for further advancements in the field.