Skyhawk Therapeutics reported extension results for its Huntington’s disease candidate, SKY-0515, describing symptom improvements after 15 months and a continued lowering of mutant huntingtin. On a complex clinical scale, treatment patients averaged a 0.94-point improvement versus a natural-history comparison group that declined by 0.65 points—an absolute gap of 1.59. Skyhawk said the drug was generally well-tolerated with no serious adverse events reported, and at higher doses it reduced mutant huntingtin by more than 60%. The company is now evaluating SKY-0515 across two larger clinical trials, with one completed enrollment. The update reinforces investor and partner interest in RNA-splicing strategies that attempt to tackle underlying disease biology while potentially improving CNS target engagement and tolerability.
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