Haya Therapeutics is a biotechnology company developing RNA-targeted therapeutics. These precision medicines are designed to address various age-related chronic diseases by modifying disease progression at a fundamental cellular level.
Targeting the Dark Matter of the Genome
The human genome contains vast regions that do not code for proteins, known as the “dark genome,” which constitutes about 98% of our genetic material. Within this non-coding landscape reside long non-coding RNAs (lncRNAs). These lncRNAs function as master regulatory switches within cells, influencing gene expression and controlling complex cellular processes.
Haya Therapeutics leverages this understanding by targeting lncRNAs. This approach focuses on reprogramming disease-driving cell states back to healthy ones. Unlike traditional drugs that often target proteins, lncRNA-targeted therapies can be designed for specific tissues and cell types, potentially leading to fewer off-target effects. The company employs advanced computational biology and machine learning to build HAYAtlas, a comprehensive atlas of the regulatory genome, to identify and modulate pathogenic cell states.
Focus on Fibrotic Diseases
Haya Therapeutics focuses on fibrotic diseases, which involve harmful scarring of organs that impairs function, leading to significant health complications. A primary area of interest is cardiac fibrosis, where excessive scar tissue forms in the heart, contributing to conditions like heart failure.
Cardiac fibrosis causes the heart muscle to stiffen, reducing its ability to pump blood effectively and increasing the risk of life-threatening conditions such as ventricular arrhythmia. Haya’s lead therapeutic candidate, HTX-001, is specifically designed to target Wisper, a lncRNA found predominantly in cardiac tissue that drives fibrosis. By suppressing Wisper, which is overexpressed in certain heart pathologies, HTX-001 aims to reduce or reverse the fibrotic processes underlying heart failure, particularly in non-obstructive hypertrophic cardiomyopathy. The tissue-specific nature of lncRNAs allows for precise intervention in affected heart cells.
Haya’s Development and Pipeline
Haya Therapeutics is advancing its lead drug candidates through preclinical development. HTX-001, their primary candidate for heart failure, is undergoing research and testing. The company anticipates initiating first-in-human clinical trials for HTX-001, specifically targeting non-obstructive hypertrophic cardiomyopathy, in the near future.
Beyond cardiac fibrosis, Haya is expanding its pipeline to address other fibrotic conditions, including pulmonary fibrosis, and is exploring treatments for obesity and other age-related diseases. The company recently secured $65 million in Series A funding in May 2025, bringing its total funding to $90 million across four rounds. This round was co-led by Sofinnova Partners and Earlybird Venture Capital, with participation from Eli Lilly and Company. Haya also entered a strategic multi-year partnership with Eli Lilly, potentially valued at up to $1 billion in milestone payments and royalties, to discover novel regulatory genome targets for obesity and metabolic disorders. In June 2025, Haya relocated its U.S. operations to Lilly Gateway Labs in San Diego, California, further strengthening its collaborative efforts.