Antisense Oligonucleotide VO659 Reduces Mutant Huntingtin Protein, Interim Phase 1/2 Data Show
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.
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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.
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.