Wave Life Sciences Highlights Therapeutic Potential for WVE-006 for Alpha-1 Antitrypsin Deficiency and Progress Bringing RNA Editing to the Clinic During Analyst and Investor Event

WVE-006 is the most advanced candidate for AATD designed to restore functional wild-type AAT protein and reduce Z-AAT protein aggregation with potential for disease modification in both lung and liver phenotypes

IND-enabling activities for WVE-006 are underway and Wave expects to submit clinical trial applications for WVE-006 in 2023

Wave is also pioneering new therapeutic applications for RNA editing, including activating gene pathways and upregulating gene expression

Excerpt from the Press Release:

CAMBRIDGE, Mass., Sept. 28, 2022 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today hosted a virtual event, “Towards the Clinic: Spotlight on RNA Editing for AATD,” highlighting its WVE-006 program, which is Wave’s preclinical A-to-I RNA base editing oligonucleotide (“AIMer”) candidate for the treatment of alpha-1 antitrypsin deficiency (AATD). The event featured a presentation from an AATD clinical expert, D. Kyle Hogarth, MD, FCCP, Professor of Medicine in the Section of Pulmonary and Critical Care Medicine at the University of Chicago. The company also outlined future therapeutic applications of its leading RNA editing capability, including modulating protein interactions as well as addressing diseases outside the liver. A replay of the event and slide presentation are available here.

“Wave continues to lead the genetic medicines field in advancing therapeutic RNA editing toward the clinic with our WVE-006 AIMer program and we expect to submit clinical trial applications for our first-in-human study in 2023. WVE-006 is distinct from other approaches in development for AATD, as it offers the opportunity to treat the root genetic cause of the disease and restore functional, wild-type protein that remains under physiological regulation. This would be a holistic solution and applicable to those with lung disease, liver disease, or both,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “Additionally, the opportunity for AIMers is much broader than restoring or correcting protein function in genetic diseases, as we demonstrated today with our preclinical data supporting the use of AIMers to modulate protein-protein interactions and upregulate gene expression. RNA editing is emerging as a distinct class of therapeutics with potential to address disease biology in novel and innovative ways and reach patients suffering from an array of diseases, including neurological disorders, as well as renal, cardiometabolic, or immunologic diseases. We are proud to be pioneers in this new area of genetic medicine and to have a best-in-class RNA editing capability with our AIMers.”

Highlights from today’s event, “Towards the Clinic: Spotlight on RNA Editing for AATD,” are below.

Best-in-Class Potential of WVE-006 for AATD
WVE-006 is a first-in-class, GalNAc-conjugated RNA editing candidate and the most advanced program currently in development using an oligonucleotide to harness an endogenous enzyme for editing.

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