Skyhawk presents today on its novel SKY-0515
small molecule RNA splicing modulator targeting Huntington's Disease to members of the
Huntington's Disease Youth
Organization, at the annual HYDO International Congress in
Prague, Czech Republic.
BOSTON, March 15,
2025 /CNW/ -- Skyhawk Therapeutics, Inc., a
clinical-stage biotechnology company developing novel small
molecule therapies designed to modulate critical RNA targets, today
presents on its novel SKY-0515 small molecule RNA splicing
modulator targeting Huntington's
Disease to members of the Huntington's Disease Youth Organization
(HDYO), at the biannual HYDO International Congress in Prague, Czech Republic.
Skyhawk Presents at Huntington's Disease Youth Organization
International Congress 2025
SKY-0515 is Skyhawk's investigational small molecule RNA
splicing modifier developed through the company's novel
RNA-splicing platform. SKY-0515 is designed to reduce both HTT
protein and PMS1 protein, an additional key driver of somatic CAG
repeat expansion and HD pathology.
"We are excited to present on our Huntingtin-lowering clinical
trials at the HDYO International Congress this month," said Sergey
Paushkin, Chief Scientific Officer and Head of R&D at Skyhawk,
"with positive results including SKY-0515's average HTT mRNA
reduction of 72% at a daily oral dose of 9mg in trials thus far,
and the drug's predicted suppression of the PMS1 protein, SKY-0515,
if approved, could make a meaningful difference in Huntington's patients' lives."
Huntington's disease is a rare
hereditary neurodegenerative disease affecting over 40,000 patients
in the United States and many more
worldwide. There are no approved treatments that can reverse or
slow its course of progression.
"We are pleased with the speed at which we've conducted our
studies thus far and are focused on this patient community in great
need", said Bill Haney, Chief
Executive Officer at Skyhawk. "SKY-0515's compelling results thus
far demonstrate the immense potential of our platform to target
indications for which there are no approved disease modifying
therapies."
About Skyhawk Therapeutics
Skyhawk Therapeutics is a
clinical-stage biotechnology company focused on the discovery and
development of novel small molecule therapies designed to modulate
critical RNA targets and revolutionize patient treatment for some
of the world's most intractable diseases. Skyhawk's discovery
expertise is rooted in its proprietary drug discovery platform,
which assesses, identifies, and tests RNA splicing targets and
small molecules across a broad range of therapeutic areas and
disease states. Skyhawk has built collaborations with multiple
pharma partners that leverage Skyhawk's novel platform across
disease areas including neurodegenerative disease, autoimmune
disease, and oncology. For more information visit
www.skyhawktx.com.
Skyhawk Contact
Maura
McCarthy
maura@skyhawktx.com
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SOURCE Skyhawk Therapeutics