Skyhawk Therapeutics develops small molecule therapeutics that modify RNA expression to target diseases such as Huntington's Disease, with its lead candidate, SKY-0515, demonstrating a 72% reduction in HTT mRNA during clinical trials. The company's proprietary SKYSTAR® platform accelerates the creation of RNA-targeting drugs across neurology, oncology, and fibrotic diseases, addressing significant unmet medical needs.
Funding
$296M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
FFounders
Product
Problem
Many diseases are caused by errors in RNA expression, but traditional small molecule therapeutics struggle to selectively target RNA and correct these errors. Developing effective treatments for these diseases requires new approaches that can precisely modulate RNA expression.
Solution
Skyhawk Therapeutics develops small molecule therapeutics that modify RNA expression to treat diseases with high unmet need. The company's SKYSTAR® platform integrates multiple technologies and machine learning to accelerate the discovery of RNA-targeting drugs. These small molecules can correct errors in RNA expression, offering a novel approach to treating a range of diseases. Skyhawk's lead candidate, SKY-0515, is currently in clinical trials for Huntington's Disease and has demonstrated a significant reduction in HTT mRNA.
Target Audience
Skyhawk's primary focus is on patients suffering from diseases caused by RNA expression errors, particularly those with neurological disorders, cancers, and fibrotic conditions.
Features
- SKYSTAR® platform integrates distinct technologies and machine learning models to accelerate drug discovery
- Small molecule therapeutics designed to modify RNA expression
- SKY-0515, a clinical-stage candidate for Huntington's Disease, has shown a 72% reduction in HTT mRNA in Phase 1 trials
- Pipeline includes programs targeting Spinocerebellar Ataxia Type 3 (SKY-1300) and Frontotemporal Dementia (SKY-1500)
- Focus areas include neurology, oncology, and fibrotic diseases