Skyhawk Therapeutics reported symptom improvements in an extension update for its Huntington’s disease candidate SKY-0515, an oral “RNA splicing” therapy designed to address mutant huntingtin biology. The company said patients showed a 1.59-point improvement on a complex functional scale over 15 months, versus a natural-history comparison group that declined by 0.65 points over the same period. Skyhawk also reported the therapy’s ability to lower mutant huntingtin protein by more than 60% at a higher dose, supporting the rationale for continued dose optimization and pivotal readiness. The company is evaluating SKY-0515 across two larger trials, with one having completed enrollment. The update adds to a small-clinical-development evidence base for RNA splicing in HD and may influence how investors and trial participants view the probability of achieving clinically meaningful benefits in Phase 2/3-style settings.
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