HAYA Therapeutics has begun dosing the first patients in a Phase 1 trial of HTX-001, a new drug designed to treat heart failure by targeting a specific piece of RNA called WISPER. This RNA drives the formation of scar tissue in the heart, which stiffens the organ and impairs its ability to pump blood. HTX-001 is an antisense oligonucleotide — a synthetic molecule that silences this RNA — aiming to reverse the fibrotic process and restore healthier heart cell behavior. The trial starts with healthy volunteers and will progress to patients with nonobstructive hypertrophic cardiomyopathy, a condition marked by significant scarring and impaired heart relaxation. Early results will focus on safety and how the drug behaves in the body.
