HAYA Therapeutics starts clinical trial for heart disease therapy
20.05.2026
Swiss biotech company HAYA Therapeutics has reached a new milestone as it officially entered the clinical stage with the dosing of the first cohort in its Phase 1 trial for HTX-001, an investigational therapy targeting cardiac fibrosis in patients with nonobstructive hypertrophic cardiomyopathy (nHCM).
 |
 |
HTX-001, HAYA’s experimental RNA-based therapy, is designed to address fibrosis, or scarring, in the heart, a process linked to reduced heart function and disease progression. Current treatments for nHCM (nonobstructive hypertrophic cardiomyopathy) mainly focus on managing symptoms and improving blood flow, but do not directly target the fibrotic changes driving the condition.
The therapy is based on an antisense oligonucleotide, a type of RNA-targeting molecule, developed to reduce the expression of WISPER, a long non-coding RNA linked to cardiac fibrosis. By lowering WISPER activity in cardiac myofibroblasts, HTX-001 aims to reprogram these cells from a disease-driving state toward a healthier profile. Preclinical studies showed reduced fibrosis and improved heart function.
The Phase 1a/b clinical study will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of HTX-001 in healthy volunteers and nHCM patients through multiple ascending-dose cohorts.
WISPER was first identified in 2017 by Samir Ounzain, who co-founded HAYA Therapeutics, winner of the Top100 Swiss Startup Award 2023, recipient of Venture Kick funding, and a Venture Leaders Life Sciences participant, alongside Daniel Blessing. The company’s research team subsequently advanced HTX-001 from discovery into clinical development.
Nonobstructive hypertrophic cardiomyopathy is estimated to represent between 30% and 60% of all hypertrophic cardiomyopathy cases. The disease is characterized by thickening of the heart muscle, impaired relaxation of the heart, and fibrosis, which can progressively affect cardiac function.
HTX-001 remains an investigational therapy and has not yet been approved by regulatory authorities, including the U.S. Food and Drug Administration or the European Medicines Agency.
The therapy is based on an antisense oligonucleotide, a type of RNA-targeting molecule, developed to reduce the expression of WISPER, a long non-coding RNA linked to cardiac fibrosis. By lowering WISPER activity in cardiac myofibroblasts, HTX-001 aims to reprogram these cells from a disease-driving state toward a healthier profile. Preclinical studies showed reduced fibrosis and improved heart function.
The Phase 1a/b clinical study will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of HTX-001 in healthy volunteers and nHCM patients through multiple ascending-dose cohorts.
WISPER was first identified in 2017 by Samir Ounzain, who co-founded HAYA Therapeutics, winner of the Top100 Swiss Startup Award 2023, recipient of Venture Kick funding, and a Venture Leaders Life Sciences participant, alongside Daniel Blessing. The company’s research team subsequently advanced HTX-001 from discovery into clinical development.
Nonobstructive hypertrophic cardiomyopathy is estimated to represent between 30% and 60% of all hypertrophic cardiomyopathy cases. The disease is characterized by thickening of the heart muscle, impaired relaxation of the heart, and fibrosis, which can progressively affect cardiac function.
HTX-001 remains an investigational therapy and has not yet been approved by regulatory authorities, including the U.S. Food and Drug Administration or the European Medicines Agency.