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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, fibrosis and other disease types. Many of our programs are against 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-Hodgkins 
Lymphoma

SKY-1214

FANCL/ FANCI

Small molecule splicing modulator

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Spinocerebellar Ataxia Type 3

SKY-1300

ATXN3

Small molecule splicing modulator

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Frontotemporal Dementia

SKY-1500

Small molecule splicing modulator

MAPTe10

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HRD Solid

Tumors

SKY-1800

Undisclosed

Small molecule splicing modulator

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Fibrosis

SKY-1700

Undisclosed

Small molecule splicing modulator

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Indication

Internal/

Partner

Oncology Assets

Neurology Assets

Fibrosis 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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