Harness Therapeutics Nominates HRN001, a First-in-Class Drug Candidate for Huntington’s Disease and Establishes Clinical Advisory Board

Core Insights - Harness Therapeutics has nominated HRN001 as its lead drug candidate for Huntington's disease, aiming for first-in-human studies in 2027 [1][5] - The company has formed a Clinical Advisory Board (CAB) to provide strategic guidance for the clinical development of HRN001 [6][8] - Dr. Andy Billinton, the Chief Scientific Officer, will present preclinical data at the CHDI Foundation Huntington's Disease Therapeutics Conference [7] Company Overview - Harness Therapeutics is a biotechnology company focused on addressing previously undruggable targets to transform treatments for neurodegenerative diseases [20] - The company utilizes its proprietary MISBA® platform technology to enable precise upregulation of target protein levels, which is crucial for developing disease-modifying therapies [20][21] - The company is based in Cambridge, UK, and has attracted leading life science investors, including Takeda Ventures and SV Health Investors' Dementia Discovery Fund [22] Product Development - HRN001 is an antisense oligonucleotide designed to upregulate FAN1, a key DNA repair nuclease, which has shown promise in slowing somatic expansion in Huntington's disease models [4][5] - Preclinical development of HRN001 will continue throughout 2026, with the goal of supporting clinical entry in 2027 [5] - The MISBA® platform is also being explored for other triplet repeat disorders and a broader pipeline of neurodegenerative diseases [5] Clinical Advisory Board - The CAB consists of leading experts in Huntington's disease, including Dr. Irina Antonijevic, Dr. Anne Rosser, and Dr. Jeffrey Long, among others [9][10] - The CAB will provide strategic guidance on clinical development, trial design, and translational strategy as the program advances [6][10] Scientific Rationale - Huntington's disease is caused by the expansion of CAG repeats in the huntingtin gene, with ongoing somatic expansion recognized as a key driver of disease progression [2][3] - FAN1 has emerged as a compelling target for suppressing somatic expansion, demonstrating a strong genetic association with disease onset [3][10]