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Our Pipeline

Skyhawk develops a broad portfolio of small molecule RNA-modifying drug candidates that have the potential to improve the lives of patients by directly addressing the causes of disease.

Our wholly-owned pipeline includes programs across neurodegenerative, oncology, neuromuscular, fibrotic diseases, and other disease types. Many of our programs address targets formerly considered “undruggable” for traditional protein-targeting mechanisms.

Huntington's Disease

SKY-0515

HTT

Small molecule splicing modulator

Phase 3

Discovery

Preclinical

Phase 1

Phase 2

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Target

Platform

Product Candidate

Multiple Myeloma,
Non-Hodgkin’s
Lymphoma

SKY-1214

FANCL/
FANCI

Small molecule splicing modulator

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Neurology

SKY-1300

Undisclosed

Small molecule splicing modulator

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Neurology

SKY-1500

Small molecule splicing modulator

Undisclosed

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Oncology

SKY-1800

Undisclosed

Small molecule splicing modulator

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Fibrosis

SKY-1700

Undisclosed

Small molecule splicing modulator

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Indication

Owner

Oncology Assets

Neurology Assets

Fibrotic Disease Assets

Partnered Pipeline

Our confidential partnered pipeline with major Pharma includes over a dozen preclinical programs
across neurodegenerative, oncology, neuromuscular, rare and other disease types.

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