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SKY-4100
- Development stage
- Unknown
- Lead developer
- Skyhawk Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
SKY-4100 is an orally bioavailable small molecule splicing modulator being developed by Skyhawk Therapeutics for the treatment of rare neurological disorders. Developed using the proprietary SKYSTAR platform, SKY-4100 is designed to target and correct RNA splicing errors, addressing the underlying genetic drivers of specific rare diseases. The compound is engineered to cross the blood-brain barrier, enabling it to address central nervous system indications with high unmet medical needs. SKY-4100 is currently in Phase 1/2 clinical development, although the specific biological target and primary disease indication have not been publicly disclosed by the developer.
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