VO659

2024 marked significant progress in Huntington’s disease (HD) research, with breakthroughs in understanding somatic instability, advancements in drug development, and both challenges and triumphs in clinical trials. The HD community saw the power of collaboration and innovation, with new voices at HDBuzz and updates from global conferences. Despite setbacks, there's hope with ongoing trials and new drug approvals, moving closer to treatments that could slow or halt HD.

Vico Therapeutics' VO659, an antisense oligonucleotide targeting C-A-G repeats in Huntington's disease (HD), shows potential to reduce toxic HD protein levels and is being tested in a basket trial for HD, SCA1, and SCA3. Initial data suggest VO659's safety and efficacy, though radiculitis side effects were observed at higher doses, prompting future dosing adjustments.

Day 2 of the 2024 Huntington Study Group Conference featured talks on palliative care, clinical trial challenges, and updates from various drug development companies, emphasizing the importance of early intervention and the use of biomarkers in HD treatment.

Vico Therapeutics reports positive interim Phase 1/2a clinical data for VO659, an allele-preferential antisense oligonucleotide therapy for Huntington's disease, showing a 28% reduction in CSF mutant huntingtin protein and no changes in Nf-L protein, with potential for infrequent dosing.

VO659, an allele-preferential antisense oligonucleotide therapy, showed a 28% reduction in mutant huntingtin protein in cerebrospinal fluid among Huntington disease patients in a phase 1/2 trial, with no neuroaxonal damage observed. The therapy is safe, well-tolerated, and has a long half-life, suggesting infrequent dosing potential.