Research suggests that people who naturally have higher levels of FAN1 tend to experience slower disease progression. HRN001 is designed to boost FAN1 levels in a controlled way, with the goal of slowing down the harmful repeat expansion process.
HRN001 is an antisense oligonucleotide (ASO), a type of medicine that works by influencing how certain proteins are made in cells. It is not a gene therapy and does not edit or permanently change a person’s DNA. Because ASO medicines are not permanent, repeat dosing would likely be needed if the treatment is proven safe and effective in the future.
HRN001 is still in early (preclinical) development and has not yet been tested in people. More research is needed before its safety or effectiveness can be determined. Harness Therapeutics plans to continue preclinical work with the goal of moving toward clinical studies in 2027.
To support the progression of HRN001 towards the clinic, Harness has established a clinical advisory board (CAB) comprising leading experts in the HD field. 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, California,
