“We are thrilled with Wave Life Sciences commitment to the Alpha-1 patient community. Their acceleration of regulatory engagement of the ongoing RestorAATion-2 clinical trial brings hope and advancement in research and science in the Alpha-1 space. The work that companies, like Wave are doing in the rare disease sector are transformative and have potential to impact many lives,” said Scott Santarella, A1F President & CEO.
WVE-006 is a first-in-class RNA editing therapeutic candidate designed to correct the root cause of disease for the 200,000 individuals in the U.S. and Europe living with AATD; no currently approved therapies address both lung and liver manifestations of this disease
Wave plans to engage FDA on potential accelerated approval pathway for WVE-006, with regulatory feedback anticipated mid-2026
Data from the 400 mg multidose cohort of RestorAATion-2 clinical trial remain on track for first quarter of 2026 and data from the 600 mg single and multidose cohorts are expected in 2026
Research collaboration with GSK is ongoing and continues to expand with fourth program now selected to advance
Wave continues to expect cash runway into 3Q 2028
CAMBRIDGE, Mass., Feb. 02, 2026 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced it has regained full rights to WVE-006, an investigational GalNAc-conjugated RNA editing oligonucleotide (AIMer) for alpha-1 antitrypsin deficiency (AATD), from GSK. This follows agreement with GSK, whose respiratory portfolio is focused on large-scale diseases, that Wave is well placed to efficiently advance the WVE-006 program in AATD, a rare condition. This agreement was made prior to data becoming available from the next cohort of the RestorAATion-2 clinical trial, which remains on track for the first quarter of 2026. Wave is now accelerating its registrational strategy for WVE-006 and plans to engage with the U.S. Food and Drug Administration (FDA) on a potential accelerated approval pathway, with regulatory feedback expected mid-2026.
“We have been eager to accelerate our registrational strategy for WVE-006 since reporting our interim data that achieved key AATD treatment goals in recapitulating the healthier MZ phenotype, including dynamic AAT production of over 20 micromolar during an acute phase response. AATD is one of the largest rare disease indications and the 200,000 individuals in the U.S. and Europe living with homozygous ‘ZZ’ AATD face extremely limited treatment options. Only weekly IV augmentation therapy is approved for AATD lung disease and no treatments are approved for AATD liver disease. WVE-006 is well-suited to Wave’s strengths and ability to execute on a commercial strategy. We look forward to engaging with regulators on how to rapidly advance this potentially transformative, first-in-class therapy to address both lung and liver manifestations with convenient, infrequent subcutaneous dosing,” said Paul Bolno, MD, MBA, President and Chief Executive Officer at Wave Life Sciences. “WVE-006 has demonstrated a favorable safety profile, does not require LNP delivery, and comes without the irreversible, collateral bystander edits and indels, which are associated with DNA base editing. With WVE-006’s highly differentiated profile, we look forward to delivering additional, higher dose datasets from our ongoing RestorAATion-2 clinical trial throughout this year.”
Data from the 400 mg multidose cohort of the ongoing RestorAATion-2 clinical trial are on track for the first quarter of 2026; single and multidose data from the 600 mg final cohort are expected in 2026.
Tony Wood, Chief Scientific Officer, GSK, said, “Our research collaboration with Wave continues with exciting opportunities ahead combining our complementary expertise to advance novel oligonucleotide therapies.”
Wave and GSK’s collaboration continues to progress and in January 2026, GSK selected a fourth program to advance to development candidate. Under the collaboration, GSK can advance up to eight programs leveraging Wave’s PRISM® platform, with target validation work ongoing across multiple therapy areas. Assuming advancement of these programs, Wave would be eligible for up to $2.8 billion in initiation, development, launch, and commercialization milestones, as well as tiered royalties. Wave anticipates milestone payments in 2026 and beyond.
Wave continues to expect that its current cash and cash equivalents will be sufficient to fund operations into the third quarter of 2028. Potential future milestones and other payments to Wave under its GSK collaboration are not included in its cash runway.
Read the original press release here.
