Develops novel genetic therapies for serious, genetically defined diseases.
Wave Life Sciences Ltd. is a pioneering clinical stage genetic medicine company that specializes in the design, optimization, and production of novel stereopure oligonucleotides through its proprietary PRISM platform. These oligonucleotides target ribonucleic acid to either reduce the expression of disease-promoting proteins, restore the production of functional proteins, or modulate protein expression. The company's innovative approach aims to address a spectrum of genetic diseases with significant unmet medical needs.
In its robust pipeline, Wave Life Sciences is advancing several promising therapeutic candidates. These include WVE-004, a molecule targeting C9orf72 for treating amyotrophic lateral sclerosis (ALS) and frontotemporal dementia, and WVE-003, designed for Huntington's disease by targeting mutant huntingtin SNP3. Additionally, the company is developing WVE-N531 for Duchenne muscular dystrophy and exploring ATXN3 for spinocerebellar ataxia 3, alongside multiple preclinical programs focusing on central nervous system disorders.
Beyond its genetic medicine platform, Wave Life Sciences is expanding into hepatic indications with GalNAc-conjugated AIMers, particularly for Alpha-1 antitrypsin deficiency (AATD). The company also has two promising preclinical programs targeting retinal diseases: Usher syndrome type 2A (USH2A) and retinitis pigmentosa caused by the P23H mutation in the RHO gene (RhoP23H). These initiatives highlight Wave Life Sciences' commitment to addressing a broad spectrum of genetic disorders affecting both the central nervous system and other vital organs.
Founded in 2012 and headquartered in Singapore, Wave Life Sciences operates through strategic collaborations with leading institutions such as Pfizer Inc., Takeda Pharmaceutical Company Limited, and several prestigious universities. These partnerships bolster the company's research and development efforts, enabling it to advance innovative therapies and potentially transform the treatment landscape for genetic diseases globally.