Skyhawk Therapeutics reports symptom improvement for Huntington's disease in Phase 1/2 extension study

Skyhawk Therapeutics announced that its RNA-splicing candidate SKY-0515 showed a measurable improvement in symptoms for patients with Huntington's disease during an extension of its Phase 1/2 trial.

The trial reported an average 1.59-point gain on the disease rating scale, indicating a modest but statistically meaningful benefit compared with baseline measurements.

Based on these results, the company said it will advance SKY-0515 into pivotal development, aiming to confirm efficacy in larger, later-stage studies.

The data adds to the growing pipeline of RNA-targeted therapies for neurodegenerative disorders, though further trials will be needed to establish long-term safety and clinical impact.