FALCON-HD is a clinical study to assess the potential of SKY-0515, a small molecule RNA splicing modifier, to:

1. Reduce both Huntingtin (HTT) protein and PMS1 protein, an important driver of somatic CAG-repeat expansion and HD pathology.

2. To slow disease progression in HD patients.

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The study will explore 3 doses of SKY-0515, with the aim to demonstrate a dose(s) that slow Huntington's disease progression over time. The study includes key biomarkers and clinical endpoints.