Pipeline

SCA2 is a rare, autosomal dominant neurodegenerative disorder caused by CAG repeat expansions in the ATXN2 gene. SCA2 is characterised by progressive cerebellar ataxia, leading to decreased coordination, balance, and fine motor skills. Over time, individuals may experience additional symptoms such as speech difficulties, tremors, and even cognitive impairment, with severe cases resulting in significant disruption in patients’ lives. The condition typically manifests in mid-adulthood, although the age of onset can vary depending on repeat length. Epidemiologically, SCA2 is one of the most common spinocerebellar ataxias, with prevalence ranging from 1–5 per 100,000 people globally. As a relentlessly progressive disorder, SCA2 has no cure, and current treatment is limited to managing symptoms such as spasticity and mobility challenges. The significant unmet need for disease-modifying therapies remains a driving force in SCA2 research.

At Evox Therapeutics, our goal is to transform the treatment of SCA2 using our exosome-based delivery system combined with advanced genome-editing techniques, where exosomes deliver genome-editing components directly to the affected neurons. Our therapeutic candidate specifically targets the root cause of SCA2 by addressing expansions in the ATXN2 gene, with the potential to halt or even reverse disease progression.