Harness Therapeutics steps up fight against Huntington's disease
The Babraham Research Campus company has nominated HRN001 as lead drug candidate for Huntington’s and formed a top-class 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.
HRN001 is described as 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 California, (February 23-26) - providing further insights into the scientific rationale and development progress for HRN001.
Dr Jan Thirkettle, CEO of Harness Therapeutics, said: "The nomination of HRN001 represents a pivotal milestone for Harness and underscores our commitment to the Huntington’s disease community.
"By precisely upregulating FAN1, a target with compelling genetic validation in delaying disease onset, HRN001 represents a differentiated, first-in-class therapeutic approach for addressing somatic expansion, a fundamental driver of disease progression.
"The formation of a Clinical Advisory Board brings deep clinical and translational expertise to the programme. The CAB will work closely with Harness as we advance HRN001 towards the clinic and seek to deliver a truly disease-modifying therapy for patients and families living with Huntington’s disease."
Harness has assembled a world-class team, supported by leading experts in neurodegeneration and RNA biology. Its leading life science investors include the foundational investors Takeda Ventures and SV Health Investors’ Dementia Discovery Fund, alongside Epidarex Capital and Ono Ventures Investment.