Massachusetts-based biotech Wave Life Sciences revealed it has regained full global rights to WVE-006, a GalNAc-conjugated RNA editing therapeutic candidate for alpha-1 antitrypsin deficiency (AATD) originally included in a collaboration with UK giant GSK. Wave Life itself plans to continue with regulatory filings for the drug with the US FDA, and will engage with the regulator on a potential accelerated approval pathway for the program.

Under the revised arrangement with GSK plc, Wave now controls the program that aims to address both lung and liver manifestations of AATD, a rare genetic disorder with significant unmet medical need, and expects clinical data from ongoing cohorts of the RestorAATion-2 trial to support regulatory discussions.

Wave said it regained rights to the asset from GSK as part of an agreement reached ahead of interim data readouts from the Phase Ib/IIa RestorAATion-2 study. Specifically, the press release notes that GSK’s respiratory portfolio is focused on large-scale diseases, while Wave is well placed to efficiently advance the WVE-006 program in AATD, a rare condition.

GSK continues to advance additional programs using Wave’s RNA platform, with potential milestones of up to USD 2.8 billion associated with collective efforts under that partnership. In January 2026, GSK optioned a fourth program to advance to development candidate, and may select up to eight programs leveraging Wave’s PRISM platform under the firms’ original 2022 deal.

Research context

WVE-006 is a first-in-class A-to-I RNA editing oligonucleotide (AIMer) designed to correct the disease-causing mutation in the SERPINA1 gene that leads to insufficient wild-type alpha-1 antitrypsin (M-AAT) protein and accumulation of misfolded Z-AAT, the molecular defect underlying AATD’s lung and liver pathology. It is delivered subcutaneously via a GalNAc conjugate to hepatocytes, where RNA editing is intended to restore functional protein expression.

AATD is an inherited condition that can lead to emphysema and progressive liver disease in people with homozygous PiZZ genotypes due to deficiency of protective alpha-1 antitrypsin and toxic accumulation of misfolded protein. Data from the 400 mg multidose cohort of RestorAATion-2 remain on track for the first quarter of 2026, with additional data from single and multidose 600 mg cohorts expected later in the year.

Wave’s RNA editing approach for AATD sits alongside other novel genetic and molecular approaches in development. Companies such as Beam Therapeutics are pursuing base editing of the SERPINA1 locus, while RNA interference programs such as fazirsiran from Takeda/Arrowhead target mRNA substrates to reduce pathogenic protein products.