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SKY-2100 is an orally bioavailable small molecule splicing modulator being developed by Skyhawk Therapeutics for the treatment of rare neurological disorders. Developed using Skyhawk's proprietary SKYSTAR platform, SKY-2100 is designed to target RNA and modulate splicing patterns to address the underlying genetic drivers of disease. While the specific molecular target and the exact rare neurological indication remain undisclosed, the drug is part of Skyhawk's internal neurology pipeline and is currently in Phase 1/2 clinical development. As a small molecule, SKY-2100 offers a potential oral alternative to more invasive RNA-targeting modalities such as antisense oligonucleotides.
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