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About

We are developing a fundamentally different treatment for repeat expansion disorders — including Huntington’s disease, myotonic dystrophy and spinocerebellar ataxias — by addressing the underlying cause of the genetic dysfunction that drives disease onset and progression. To accomplish this goal, we’ve assembled a team with decades of expertise across drug discovery, genetics, clinical research and operations. We’r...
We are developing a fundamentally different treatment for repeat expansion disorders — including Huntington’s disease, myotonic dystrophy and spinocerebellar ataxias — by addressing the underlying cause of the genetic dysfunction that drives disease onset and progression. To accomplish this goal, we’ve assembled a team with decades of expertise across drug discovery, genetics, clinical research and operations. We’re supported by a top-tier syndicate of investors who share our commitment to transforming the lives of patients and families affected by repeat expansion disorders.

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