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SKY-0925
- Development stage
- Unknown
- Lead developer
- Skyhawk Therapeutics
- Modality
- Small Molecules
01
Overview
SKY-0925 is a small molecule splicing modulator being developed by Skyhawk Therapeutics for the treatment of rare neurological indications. Utilizing Skyhawk's proprietary SKYSTAR platform, the drug is designed to target and modulate RNA expression, specifically by correcting or altering splicing patterns to address the underlying genetic causes of disease. As of early 2024, SKY-0925 is in Phase 1/2 clinical development, representing one of the company's lead internal programs for rare neurodegenerative or neurogenetic conditions with significant unmet needs. While the specific biological target and indication remain undisclosed, the drug functions by modifying RNA splicing to restore or regulate protein production.
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