HRN001

HRN001 is an antisense oligonucleotide (ASO) therapeutic in preclinical development by Harness Therapeutics for the treatment of Huntington's disease. The drug is designed to increase levels of FAN1 nuclease, which is recognized as the strongest genetic modifier of Huntington's disease onset. By upregulating FAN1, HRN001 aims to slow down the somatic expansion of CAG triplet repeats—a key driver in Huntington’s disease progression—through controlled physiological upregulation at the level of endogenous mRNA translation. This approach seeks to provide a first-in-class, truly disease-modifying therapy for Huntington’s disease and potentially other neurodegenerative disorders[1][3][6][8].

Other names

FAN1-targeting antisense oligonucleotideFAN-1-targeting antisense oligonucleotideFAN 1-targeting antisense oligonucleotideFAN1-targeting ASOFAN-1-targeting ASOFAN 1-targeting ASOfan1 targeting oligonucleotidefan-1 targeting oligonucleotidefan 1 targeting oligonucleotide