Wave Life Sciences

Wave Life Sciences

Wave is a leader in the space of stereochemistry, with an initial focus on antisense oligonucleotides (ASOs) and exon skipping. In simple terms, stereochemistry refers to the three-dimensional structure of a molecule and how this affects its chemical properties. Current ASOs can contain hundreds to hundreds of thousands of various enantiomers («stereomixture»), many of which do not contribute to efficacy, but could be causing toxicity. Wave is able to specifically design their individual molecules («stereo-pure») to contain the desired properties, thus potentially enhancing potency and minimizing toxicity. The company’s lead product is in Phase I/II development for Huntington’s disease and targets very specific point mutations in order to knock down the mutant protein. Proof of concept data support a clean safety profile and clear target engagement, but higher doses will be added to the Phase I/II study. Wave’s second program in DMD was discontinued.