- Wave Life Sciences hopes to soon begin clinical testing of what it claims will be the first RNA editing treatment in humans and expects to deliver “proof-of-mechanism” data by next year.
- The Cambridge, Massachusetts-based company applied for regulatory permission to test its therapy, dubbed WVE-006, in healthy volunteers as well as patients with alpha-1 antitrypsin deficiency, or AATD. The inherited disorder is caused by a genetic mutation and can cause damage to the lungs and liver.
- The first dosing in humans should occur in the fourth quarter, Wave said Tuesday. The company expects to discuss the clinical program as well as other RNA editing research at a virtual “R&D Day” for investors and analysts on Sept. 28.
Success in the clinic would boost the RNA editing field as well as Wave. Broadly speaking, the technology is designed to edit specific sites in an RNA transcript without making permanent changes to a person’s genome.
It’s an approach that’s drawn interest from major players including Eli Lilly and Roche, as well as startups such as Ascidian Therapeutics. For its part, Wave signed a four-year development deal with GSK late last year that included $225 million in potential development and launch milestone payments for WVE-006.
There are about 200,000 AATD patients in the U.S. and Europe who have mutations that make them candidates for Wave’s therapy. At the moment, there are no approved medicines for AATD liver disease, and the only option for patients with AATD lung disease is weekly infusions to deliver the AAT protein.
A number of companies are also trying to fill the gap for AATD patients with new treatments. Arrowhead Pharmaceuticals is testing an RNA-based medicine, while Vertex Pharmaceuticals is studying two different treatments.
Editor’s note: This story has been updated to clarify the $225 million in additional payments potentially due to Wave are for achieving development and launch-related milestones. The company could receive other, sales-related payments tied to WVE-006.