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Targeted Therapy for Huntington Disease

Michael Panzara, MD, MPH, Chief Medical Officer at Wave Life Sciences provides an overview of the pathophysiology of Huntington disease and how that knowledge is guiding treatment development. Huntington disease is an inherited, neurodegenerative disorder that usually begins to be symptomatic starting around 40 years of age. Persons with Huntington disease suffer from a progressive movement disorder, cognitive decline, psychiatric disturbances, and behavioral symptoms. All symptoms progress which eventually leads to immobility, dementia, and premature death. Wave Life Sciences is currently developing a treatment for Huntington disease that targets the mutant huntingtin (mHTT) allele. More specifically, WVE-003 is a stereopure antisense oligonucleotide designed to target SNP3, a single nucleotide polymorphism on the mHTT allele. Using this approach, the company is hopeful that the drug will selectively lowering mHTT protein while sparing the healthy, wild-type huntingtin (wtHTT) protein. A clinical trial [NCT05032196] is currently underway to test dose escalation and dosing frequency in adults with Huntington disease.