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Wave Life Sciences is a publicly traded biotechnology company based in Cambridge, Massachusetts. It focuses on designing, developing, and commercializing stereopure nucleic acid therapeutics that precisely targets the underlying cause of rare genetic diseases through its proprietary synthetic chemistry drug development platform.
The company's pipeline initially focused on neurological disorders and later extended across several other therapeutic areas including, muscular, opthalmology, and hephatic disorders. In the central nervous system category, Wave Life Sciences is developing treatments for Huntington's disease, amyotrophic lateral sclerosis, frontotemporal dementia, spinocerebellar ataxia 3, and other CNS diseases. Of these, two therapies focused on Huntington's disease is in Phase 1b/2a clinical trials, while the therapies for the other conditions are in pre-clinical stages. Commercial rights for these are shared with Takeda.
The company's pipeline also has several possible therapies for Duchenne muscular dystrophy and other neuromuscular diseases. Of these, one therapy for Duchenne muscular dystrophy has entered Phase 1 clinical trials with the rest in various pre-clinical stages. Wave Life also has a couple of product candidates in the discovery stage focusing on retinal diseases and metabolic liver diseases. Of these, the latter is in partnership with Pfizer.
