Harness Therapeutics Nominates HRN001, a First-in-Class Drug Candidate for Huntington’s Disease and Establishes Clinical Advisory Board
View original at globenewswire.comHarness Therapeutics Nominates HRN001, a First-in-Class Drug Candidate for Huntington’s Disease and Establishes Clinical Advisory Board HRN001 is a potent and specific antisense oligonucleotide designed to drive controlled upregulation of FAN1, a genetically validated target in Huntington’s disease Clinical Advisory Bo…
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There are currently no approved disease-modifying treatments available for Huntington's disease.
80% confidenceHarness is exploring the potential of the MISBA® platform in other triplet repeat disorders and across a broader pipeline of neurodegenerative disorders.
80% confidenceFAN1 nuclease has emerged as one of the most compelling targets to suppress somatic expansion, demonstrating the strongest genetic association to disease onset in genome-wide association studies.
80% confidenceHD is caused by the expansion of CAG repeats in the huntingtin (HTT) gene. Ongoing somatic expansion of these repeats is now recognised as a key driver of disease onset and progression.
80% confidenceHRN001 has demonstrated robust upregulation of FAN1 and slowing of somatic expansion in models of HD, as well as favourable PK and tolerability characteristics.
80% confidenceThe nomination of HRN001 represents a pivotal milestone for Harness and underscores commitment to the Huntington's disease community. HRN001 represents a differentiated, first-in-class therapeutic approach for addressing somatic expansion.
80% confidenceFAN1 is one of the most compelling and consistently validated genetic modifiers of Huntington's disease identified to date, with a clear mechanistic link to somatic expansion and disease progression. Harness' approach with HRN001 offers a novel and highly targeted way to therapeutically modulate this pathway.
80% confidenceHD leads to progressive cognitive, psychiatric and motor decline, with death often occurring within 15 years of symptom onset.
80% confidence