Target G (Wave Life Sciences Undisclosed Lung Target) (Target G)

Target G mRNA is a proprietary therapeutic target in Wave Life Sciences' pipeline, specifically focused on treating a rare genetic lung disease. The target is addressed using Wave's proprietary AIMer (ADAR-mediated RNA editing) platform, which utilizes endogenous ADAR (Adenosine Deaminase Acting on RNA) enzymes to perform precise A-to-I editing on the mRNA transcript. This editing is designed to correct a pathogenic G-to-A mutation, thereby restoring the production of a functional protein that is otherwise deficient or dysfunctional in patients. Target G is estimated to affect approximately 5,000 patients in the United States and Europe, representing a significant unmet medical need in the rare disease space. Preclinical data has demonstrated the feasibility of this approach, with significant editing efficiency observed in vitro and the potential for meaningful clinical benefit with relatively low levels of correction.

Other names

Undisclosed genetic lung disease targetTarget G mRNAWave Life Sciences Target G

ADAR-mediated RNA editing (A-to-I correction of a pathogenic G-to-A mutation in the mRNA transcript)