According to a recent article on PharmaBiz, HAYA Therapeutics has announced the completion of dosing in the first cohort of its phase 1 clinical study evaluating HTX-001, an investigational RNA-targeting therapy for patients with nonobstructive hypertrophic cardiomyopathy (nHCM). The milestone marks an early but significant step in the company’s effort to develop disease-modifying treatments that address cardiac fibrosis at its molecular roots.
HTX-001 is designed as an antisense oligonucleotide that targets WISPER, a long non-coding RNA implicated in cardiac fibrosis. By suppressing WISPER activity in cardiac myofibroblasts, the therapy aims to alter the behavior of these cells, which play a central role in scar formation and adverse myocardial remodeling. Preclinical findings have suggested that modulating this pathway can reduce fibrotic tissue accumulation and improve cardiac function, supporting further clinical investigation.
The phase 1a/b study will assess safety, tolerability, pharmacokinetics, and pharmacodynamic effects across ascending dose levels. Initial testing is being conducted in healthy volunteers, followed by individuals with nHCM. This stepwise design is intended to establish early safety signals while beginning to explore biological activity in the target patient population.
Nonobstructive hypertrophic cardiomyopathy represents a substantial proportion of hypertrophic cardiomyopathy cases, with estimates suggesting it accounts for roughly one-third to over half of diagnoses. The condition is characterized by thickened cardiac muscle, impaired relaxation of the left ventricle, and extensive fibrosis. Despite advances in disease understanding, currently available therapies do not directly target the fibrotic processes that contribute to disease progression and functional decline.
HAYA’s approach focuses on the regulatory genome—regions of genetic material that control gene expression rather than encode proteins. WISPER, first identified in 2017 by the company’s co-founder and CEO, Samir Ounzain, has been shown to regulate fibrotic signaling pathways in the heart. By intervening at this level, HTX-001 is intended to reprogram pathogenic cell states into a more normal phenotype, offering a potentially causal strategy rather than symptomatic management.
Company leaders view the transition of HTX-001 into human testing as validation of their broader platform. The program has been advanced through internal research efforts led by the scientific team, including co-founder and chief scientific officer Daniel Blessing, who helped translate early biological insights into a clinical candidate.
Clinical experts involved in the trial emphasize the unmet need in nHCM. Existing therapies largely focus on symptom control or hemodynamic improvement but do not address the fibrosis driving disease progression. A targeted antifibrotic intervention could represent a meaningful shift in treatment paradigms if clinical efficacy is demonstrated.
HTX-001 remains an investigational agent and has not yet received regulatory approval. As with all early-stage studies, its safety profile and therapeutic impact in humans have yet to be established. Nevertheless, the initiation of clinical testing underscores growing interest in RNA-based strategies that target previously inaccessible elements of disease biology.
HAYA Therapeutics is headquartered in Lausanne, Switzerland, with additional research operations in San Diego, California. The company is focused on advancing precision RNA-guided therapies for chronic and age-related diseases by leveraging regulatory genome science.
