HAYA Therapeutics has dosed the first cohort in a Phase 1 clinical trial of HTX-001, an investigational therapy for nonobstructive hypertrophic cardiomyopathy (nHCM). The antisense oligonucleotide targets WISPER, a long non-coding RNA overexpressed in patients with hypertrophic cardiomyopathy. HTX-001 aims to reprogram cardiac myofibroblast cells by decreasing WISPER expression, potentially reducing pathological cardiac fibrosis and improving heart function. Preclinical studies have demonstrated these effects. WISPER was discovered in 2017 by HAYA co-founder and CEO Samir Ounzain. The condition affects an estimated 30-60% of all hypertrophic cardiomyopathy cases. Current treatments do not address the underlying fibrotic process driving the disease. The Phase 1a/b study will evaluate safety, tolerability, pharmacokinetics and pharmacodynamics in healthy volunteers and nHCM patients.

HAYA Therapeutics, a Swiss biotech startup, secured €57 million in a Series A funding round in May 2025. The funding will be used to advance clinical trials for its heart failure therapy and expand its RNA-guided platform. Investors include Sofinnova Partners, Schroders Capital, and others.