Harness Therapeutics announces the nomination of HRN001 as its lead drug candidate for Huntington's disease and the formation of a clinical advisory board to support the programme's advancement towards clinical evaluation.
News coming in today of the development of a new drug with the formation of a clinical advisory board.
The press release states:
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 Board formed to support progression of HRN001 into first-in-human studies in 2027
Dr Andy Billinton, CSO will present preclinical data at the upcoming CHDI Foundation Huntington’s Disease Therapeutics Conference
Harness Therapeutics (‘Harness’), a biotechnology company unlocking previously undruggable targets to transform the treatment of neurodegenerative diseases, today announces the nomination of HRN001 as its lead drug candidate for Huntington's disease and the formation of a clinical advisory board to support the programme's advancement towards clinical evaluation.
Huntington’s disease (HD) is a devastating, inherited neurodegenerative disorder that leads to progressive cognitive, psychiatric and motor decline, with death often occurring within 15 years of symptom onset. Despite significant advances in understanding HD disease biology, there are currently no approved disease-modifying treatments available.
HD 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. FAN1 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. Our first-in-class candidate HRN001 is a potent and specific antisense oligonucleotide targeting FAN1, designed to drive controlled upregulation of this key DNA repair nuclease. It leverages Harness' proprietary MISBA® (microRNA site blocking ASO) platform, which enables precise upregulation of target protein levels without risk of over-expression.
HRN001 has demonstrated robust upregulation of FAN1 and slowing of somatic expansion in models of HD, as well as favourable PK and tolerability characteristics. Preclinical development will continue throughout 2026 to support clinical entry in 2027. Harness is exploring the potential of the MISBA® platform in other triplet repeat disorders and across a broader pipeline of neurodegenerative disorders.
To support the progression of HRN001 towards the clinic, Harness has established a clinical advisory board (CAB) comprising leading experts in the HD field, including:
- Dr. Irina Antonijevic (Chair) – Chief Medical Officer, Trace Neuroscience
- Dr. Anne Rosser – Professor of Clinical Neuroscience, Cardiff University
- Dr. Jeffrey Long – Professor of Psychiatry and Biostatistics, University of Iowa Health Care
- Dr. Ralf Reilmann – Founding Director and Chief Executive Officer, George-Huntington-Institute
- Dr. Roger Barker – Professor of Clinical Neuroscience, University of Cambridge
- Dr. Sarah Tabrizi – Professor of Clinical Neurology, University College London
- Dr. William Gray – Professor of Functional Neurosurgery, Cardiff University
The CAB will provide strategic guidance on clinical development, trial design, and translational strategy as the programme advances towards the clinic.
Dr Andy Billinton, Chief Scientific Officer of Harness Therapeutics, will present the company's work in HD at the upcoming annual CHDI Huntington’s Disease Therapeutics Conference in Palm Springs.
In summary, Huntington's disease 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. FAN1 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.
Harness Therapeutics have found a target that potentially will reduce/stop somatic expansion (that is the CAG repeats getting bigger) which has the potential to delay disease onset. They are planning to go to trial in 2027.
Although this is in the early stages, it is promising to see the development of another treatment for Huntington's disease.
Harness has developed a really useful Factsheet: