HAYA Therapeutics SA, a clinical-stage biotechnology company pioneering precision RNA-guided therapeutics that target the regulatory genome to reprogram disease-driving cell states in common, chronic and age-related diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for HTX-001 for the treatment of symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM). The first-in-class investigational long non-coding RNA (lncRNA)-targeting therapy is in a Phase 1a/b trial, with the first cohort dosed this past May.
HTX-001 is a differentiated, potentially disease-modifying antisense oligonucleotide engineered to downregulate WISPER, a heart stress-specific lncRNA that is overexpressed in hypertrophic cardiomyopathy, including nHCM. By targeting WISPER in cardiac myofibroblasts, HTX-001 is intended to promote the precision reprogramming of this fibrotic and pathological cell state toward a healthy phenotype.
