Company profile
Harness Therapeutics is transforming the treatment of neurodegenerative diseases by unlocking a new class of genetically validated disease-modifying targets through precision protein upregulation. Our proprietary MISBA® platform enables finely tuned, controlled increases in protein expression, allowing for therapeutic targeting of complex biological pathways that have remained inaccessible with traditional approaches.
Our lead programme in Huntington’s disease (HD) targets FAN1, a DNA repair nuclease with compelling genetic validation linking it to delayed disease onset and progression. Harness is the first company capable of drugging this critical target which could, for the first time, yield a therapy capable of changing the course of disease before onset of symptoms.
Beyond HD, we are building a pipeline of first-in-class disease-modifying therapies. Targets have been selected in Parkinson’s disease, Alzheimer’s disease, and ALS, with other CNS disorders to be explored. As with FAN1, these programmes leverage our MISBA® platform to enable controlled modulation of targets with compelling genetic validation, which have been out of the reach of other platforms. We are actively exploring collaborations and partnerships to enable our MISBA® platform to be leveraged across a range of CNS disorders, with a research collaboration underway with Ono Venture Investment in an undisclosed rare triplet repeat disorder.
Harness is led by an expert team and supported by blue-chip investors and world-class advisors in RNA biology and neurodegeneration.