Wave Life Sciences, GSK partner on oligonucleotide-based gene therapies

Wave Life Sciences and GSK have forged a partnership to discover and develop oligonucleotide therapeutics, focusing on new genetic targets, for respiratory diseases.

The discovery collaboration with an initial four-year research term is expected to advance Wave Life Sciences‘ preclinical RNA editing program, WVE-006, to treat alpha-1 antitrypsin deficiency (AATD), a disease that impacts the lungs and liver.

Under the terms of the agreement, the Nasdaq-listed clinical-stage genetic medicines company will receive upfront payment of $170 million in cash and equity from GSK apart from milestone payments and royalties.

With an option to extend for up to three additional years, the partnership will integrate GSK’s expertise in genetics and genomics with Wave Life Sciences’ PRISM oligonucleotide platform.

As part of the agreement, GSK will advance up to eight preclinical programs while Wave Life Sciences will advance up to three preclinical programmes for targets informed by the former’s novel insights.

Wave Life Sciences, GSK partner on oligonucleotide-based gene therapies. Photo courtesy of GSK plc.

GSK will also receive the exclusive global license for Wave Life Sciences preclinical program for inherited genetic disease AATD called WVE-006 that leverages the latter’s proprietary “AIMer” technology (A-to-I(G) RNA editing).

For the WVE-006 program, Wave Life Sciences will be entitled to a payment of up to $225 million towards development and launch milestones and sales-related milestone payments of up to $300 million and tiered sales royalties.

For each of GSK’s eight collaboration programs, Wave will be eligible to receive up to $130-$175 million in development and launch milestones and $200 million in sales-related milestones, along with tiered sales royalties.

Tony Wood — GSK President and Chief Scientific Officer said: “Pairing GSK’s genetic expertise with the best-in-class PRISM platform enables us to accelerate drug discovery for newly-identified targets, by matching target to modality.

“The addition of WVE-006 complements more advanced, clinical-phase oligonucleotides in our pipeline, including bepirovirsen for chronic hepatitis B and GSK4532990 for non-alcoholic steatohepatitis (NASH).”

GSK will be responsible for development and commercialization of drug WVE-006 once Wave Life Sciences wraps up the first-in-patient study of its most advanced RNA editing program.

Paul Bolno — Wave Life Sciences President and CEO said: “In 2022, we started to deliver on the promise of our platform with the first data showing translation in the clinic for our next-generation stereopure PN-chemistry containing candidates.

“Now with our GSK collaboration, we are excited to leverage their expertise in genetics to continue building a differentiated oligonucleotide pipeline, with a focus on our best-in-class RNA editing and upregulation capability.

“Additionally, GSK is the ideal partner for our WVE-006 program, due to their longstanding history and global reach in respiratory diseases.”

WVE-006 adds a third oligonucleotide to GSK’s portfolio that already includes bepirovirsen for chronic hepatitis B and GSK4532990 for non-alcoholic steatohepatitis (NASH).

The equity investment and collaboration deal will close simultaneously and are subject to the Hart-Scott-Rodino Act clearance and other customary conditions.