GSK Returns Rights to Wave's Pioneering RNA Editing Therapy, Citing Strategic Focus Shift
This analysis was originally reported by BioPharma Dive. For daily updates on the pharmaceutical industry, subscribe to our newsletter here.
In a notable shift for one of biotechnology's most watched experimental platforms, pharmaceutical giant GSK has returned global rights to the leading RNA editing therapy in clinical trials back to its developer, Wave Life Sciences. The therapy, WVE-006, is being studied for alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder that can cause severe lung and liver disease.
The companies announced Monday that their 2022 collaboration agreement has been amended, with Wave regaining full control of the program. According to Wave's statement, the decision stemmed from a mutual recognition that the biotech is better positioned to swiftly navigate the development and regulatory path for this rare disease asset. "GSK's respiratory portfolio is strategically centered on large-scale diseases," the company noted, implying a misalignment with the niche focus required for AATD.
Critically, the handover precedes the next data readout from the ongoing Phase 2 trial, expected by the end of March. Wave indicated it is now "accelerating" its submission planning and will seek U.S. regulatory discussions on a potential accelerated approval pathway, with feedback anticipated mid-year.
"WVE-006 aligns perfectly with Wave's core competencies and agile development model," said CEO Paul Bolno. "We are poised to engage with regulators to rapidly advance this first-in-class, potentially transformative therapy."
The move places a spotlight on the volatile yet promising field of RNA editing. As a technology, it aims to correct disease at the messenger RNA level, offering a potentially reversible and precise alternative to permanent DNA editing. WVE-006, as the first of its kind to enter human testing, serves as a critical bellwether for the entire approach. Early results have demonstrated its ability to restore functional protein in AATD patients, which Wave has touted as a step toward a "functional cure." However, investor appetite has been tempered by desires for more robust efficacy data and the recent failure of a rival RNA editing therapy for AATD from Korro Bio.
GSK's Chief Scientific Officer, Tony Wood, emphasized that the broader multi-program alliance between the companies remains intact, with a fourth program selected just this January. The deal could still see Wave receive up to $2.8 billion if all eight optional programs are advanced.
Wave reassured stakeholders that its current cash reserves are sufficient to fund operations into the third quarter of 2028, providing a runway to shepherd WVE-006 through key milestones.
Expert Reactions
Dr. Anya Sharma, Biotech Analyst at Horizon Insights: "This is a pragmatic recalibration. For GSK, it's a portfolio optimization. For Wave, it's a vote of confidence in their ability to execute a focused rare disease strategy. The real test comes with the Phase 2 data next month."
Michael T. Rossi, Managing Partner at LifeSci Ventures: "It streamlines Wave's path to market without a large partner's sometimes cumbersome processes. The retained broader collaboration shows GSK still sees long-term value in Wave's platform, even if this particular asset didn't fit their commercial engine."
Dr. Helen Carter, Patient Advocate & Founder of AATD Action Network: "While corporate maneuvering is complex, patients are watching closely. We need clarity and speed. Wave now has sole responsibility to deliver on its 'functional cure' promise without delay. Excuses are over."
Leo Finch, Columnist at 'The Pharma Skeptic': "Let's call this what it is: a quiet retreat. GSK saw the modest data, the competitor's failure, and the steep climb for an unproven technology in a small market, and decided to cut its losses. Wave's 'accelerated' spin is face-saving PR. The burden of proof just got much heavier for them alone."