Startup lands $65m and targets clinical trials of precision RNA-guided medicines that reprogram disease-driving cell states into healthy ones.
Precision medicine biotech HAYA Therapeutics has secured $65 million in Series A funding to accelerate the development of RNA-guided medicines that target chronic and age-related diseases by modulating the regulatory genome. The investment will enable the company to advance its lead therapeutic candidate into clinical trials for heart failure and to broaden its pipeline of therapies that target long non-coding RNAs (lncRNAs) in various disease contexts.
Central to HAYA’s approach is its ability to harness the regulatory genome, described as the “dark genome” because it does not encode proteins and is relatively underexplored. However, according to HAYA, this segment of the genome serves as a critical control layer for gene expression and cellular identity. By combining multimodal functional genomics with proprietary machine learning tools, the company says it has created a comprehensive atlas of the regulatory genome, enabling the development of highly targeted, RNA-guided therapies that can reprogram pathological cell states across a spectrum of diseases.
With facilities in Lausanne, Switzerland, and San Diego, HAYA’s foundation stems from co-founder and CEO Samir Ounzain’s lifelong fascination with molecular biology and genomic regulation. His vision for the company was shaped by the idea that the genome is essentially the source code of life, and that lncRNAs act as critical information-processing units within this code. By programming these units, HAYA aims to shift cell behavior from diseased to healthy states, particularly in conditions driven by fibrosis and other chronic pathological mechanisms.
“This is a defining moment for HAYA as we are advancing our lead program into the clinic,” said Ounzain. “This funding validates our organization, pioneering approach and vision for improving the way chronic and complex diseases are treated by creating a new generation of therapies that reprogram disease-driving cell states into healthy ones.”
The company’s lead candidate, HTX-001, targets a heart-specific lncRNA involved in non-obstructive hypertrophic cardiomyopathy (nHCM), a condition in which persistent fibrotic remodeling impairs cardiac function. In cases of heart injury, resident fibroblasts activate into myofibroblasts, producing scar tissue that, while initially protective, can become pathological when prolonged. This process, known as interstitial fibrosis, leads to stiffening of the cardiac muscle, reduced function, and potentially fatal outcomes such as arrhythmia and heart failure. While current therapies often target protein-coding genes, which are widely expressed across organs, increasing the risk of off-target effects, HAYA’s approach seeks to avoid this by specifically targeting the regulatory RNAs that drive fibrosis in a cell- and tissue-specific manner.
In addition to cardiac fibrosis, HAYA’s platform has demonstrated relevance in other chronic conditions including pulmonary fibrosis, obesity and squamous cell carcinoma. The platform’s capacity to identify lncRNAs that are expressed in specific cells or in response to pathological stimuli underpins its potential across various therapeutic areas. Indeed, the company recently entered into a strategic partnership with Eli Lilly focused on developing RNA-based therapies for obesity and metabolic disorders.
The funding round was co-led by Sofinnova Partners and Earlybird Venture Capital, with participation from Eli Lilly and Company, ATHOS, +ND Capital, Alexandria Venture Investments and LifeLink Ventures, and ongoing support from existing investors including Apollo Health Ventures, Longview Ventures, 4see ventures, BERNINA Bioinvest and Schroders Capital.
“HAYA’s platform unlocks the dark genome’s therapeutic potential by targeting disease-driving cell states via long non-coding RNAs,” said Sofinnova’s Henrijette Richter. “This novel approach opens a new frontier in precision medicine, moving beyond traditional target classes to address disease at its epigenetic and cellular roots.”
Photograph of Samir Ounzain courtesy of HAYA Therapeutics.
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