Polyglutamine disease proteins: Commonalities and differences in interaction profiles and pathological effects.
Megan Bonsor, Orchid Ammar, Sigrid Schnoegl...
https://pubmed.ncbi.nlm.nih.gov/38615323/Actively Recruiting
Phase 1
Phase 2
Age: 25Years - 60Years
All Genders
ID05822908
Phase 12a Study of VO659 Delivered by Spinal Injection to Assess Safety and Effects in Adults With Spinocerebellar Ataxia Types 1, 3, and Early Huntingtons Disease
Led by Vico Therapeutics B. V. · Updated on 2025-08-28
68
Participants Needed
14
Research Sites
28 weeks
Total Duration
AI-Summary
What this Trial Is About
Researchers are studying spinocerebellar ataxia types 1 and 3 SCA1 and SCA3 and Huntingtons disease HD, which are serious genetic neurodegenerative disorders without current treatments to slow their progression. This first-in-human trial evaluates a new drug called VO659, designed to target harmful gene mutations by binding to specific RNA sequences. The study aims to assess the safety, tolerability, and how the drug moves through and affects the body when given via spinal injections.
CONDITIONS
Brief Title
A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD
Research Team
C
Chief Medical Officer
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