HTX-001 is in a Phase 1a/b trial and being developed for the treatment of nonobstructive hypertrophic cardiomyopathy (nHCM)
LAUSANNE, Switzerland and SAN DIEGO – July 28, 2026 – 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.
“HAYA was founded on the belief that the regulatory genome offers a new way to address the underlying biology of disease,” said Samir Ounzain, Ph.D., CEO and co-founder of HAYA Therapeutics. “This regulatory designation recognizes the potential of HTX-001 to offer a differentiated therapeutic approach for patients with nHCM.”
“Patients with nonobstructive HCM are seeking treatments that go beyond management of symptoms. This designation reflects the urgency of delivering better therapeutic options,” said Jordan Shin, M.D., Ph.D., CMO of HAYA Therapeutics. “With our Phase 1 clinical trial already underway, Fast Track designation allows us to work more closely with the FDA as we advance HTX-001, our investigational precision RNA-guided therapy designed to reprogram disease-driving cardiac fibroblasts – the sentinel effector cells which act to drive fibrosis and remodeling in the myocardium.”
The FDA’s Fast Track designation is intended to expedite the development and review of drugs that treat serious conditions and fill an unmet medical need. This designation may enable more frequent interactions with the FDA throughout the drug development process and potentially provides eligibility for accelerated approval, priority review and rolling review. This is particularly important for patients with nHCM, which accounts for an estimated 30-60% of all hypertrophic cardiomyopathy cases, where current therapies do not directly target the fibrotic pathology or diastolic dysfunction that drive the disease.
HTX-001 is an investigational therapy candidate and has not been approved by the U.S. Food and Drug Administration, the European Medicines Agency, or any other regulatory authority. Its safety and ability to translate into clinical benefit remain to be established.
About nonobstructive hypertrophic cardiomyopathy (nHCM)
nHCM is a chronic form of heart disease in which the heart muscle becomes abnormally thick and stiff, making it harder for the heart to function properly. It affects an estimated 30-60% of people with hypertrophic cardiomyopathy. The condition is characterized by hypertrophy in the left ventricle, impaired diastolic function and marked fibrosis. While significant progress has been made in understanding the cellular biology underlying nHCM, currently available therapeutic approaches do not directly address the fibrotic pathology or diastolic dysfunction that drive the disease.
About HAYA Therapeutics
HAYA Therapeutics is a clinical-stage precision medicine company developing programmable, first-in-class RNA-guided therapeutics that target the regulatory genome to reprogram disease-driving cell states. HAYA’s innovative platform decodes the causal biology of pathological cell states and the long non-coding RNAs (lncRNAs) that regulate them, translating these insights into tractable therapeutic candidates designed to address disease at its source and restore cellular health. HAYA is advancing a broad pipeline of RNA-guided medicines for chronic, age-related diseases.
HAYA’s lead investigational candidate, HTX-001, is an antisense oligonucleotide targeting WISPER, and is currently being evaluated in a Phase 1 clinical trial, initially for nonobstructive hypertrophic cardiomyopathy (nHCM).
HAYA is headquartered at the life sciences park Biopôle in Lausanne, Switzerland, with laboratory facilities at Lilly Gateway Labs in San Diego, California. For more information, please visit www.hayatx.com and follow HAYA on X and LinkedIn.
Media contact
For HAYA Therapeutics
Tim Ingersoll
Linnden Communications
