WaVe Life Sciences

WVE-003 Clin1/Clin2 Huntington's disease (partner: Takeda); WVE-N531 Clin1/Clin2 DMD; WVE-006 Clin0 AATD - lung & liver disease; 3 undisclosed programs Clin0 spinocerebellar ataxia 3, CNS diseases

Wave Life Sciences is developing novel oligonucleotide therapies for the treatment of rare genetic diseases. The company's PRISM platform enables the precise design, optimization, and production of stereopure oligonucleotides. Unlike traditional oligonucleotides, which are racemic mixtures of stereoisomers, stereopure oligonucleotides are composed of only one stereoisomer. This makes stereopure oligonucleotides more potent and less toxic than traditional oligonucleotides. Wave Life Sciences is using its PRISM platform to develop stereopure oligonucleotide therapies for a variety of rare genetic diseases, including Huntington's disease, Duchenne muscular dystrophy, and Batten disease. The company's lead product candidate, WVE-001, is a stereopure oligonucleotide therapy for the treatment of Huntington's disease.