Support HDYO

HDYO is the only organization who focus solely on supporting young people impacted by HD around the world. Will you help us?

<span class="translation_missing" title="translation missing: en.hdyo.land.loading">Loading</span>
Home Language and Accessibility What is HD? New to HD? Find Resources Research Ask for Help About HDYO News Videos PDF Resources Join Mailing List Kids Teens Young Adults Parents and Family JoHD Friends Professionals DONATE! Privacy Contact Us

Skyhawk Therapeutics Topline Data for Phase 1 Trial

July 10, 2024

Huntington's Disease Youth Organization

HDYO has more information about HD available for young people, parents and professionals on our site:


In a press release to the community today, Skyhawk Therapeutics announced topline results from their Phase 1 trial:

Skyhawk Therapeutics Announces Positive Topline Results from Parts A and B of its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington's Disease, Reaching 72% Huntingtin mRNA Reduction

  • Skyhawk's SKY-0515 demonstrated dose-dependent huntingtin (HTT) mRNA reduction in healthy volunteers, with 72% reduction at the highest dose tested in the multiple ascending dose study

  • SKY-0515 was generally well tolerated at all doses tested

  • Given these positive topline results, we anticipate dosing in the patient arm of the study in Q3 2024 and initiation of a Phase 2 study early next year

Read Full Press Release

Our content is split into different groups. Choose the group that best describes your needs for more information on HD.

Kids Teens Young Adults Parents and Family JoHD Friends Professionals