HAYA Therapeutics has successfully closed a $65 million Series A funding round to advance its pioneering approach to precision medicine targeting the regulatory genome. The financing 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, alongside existing investors.
The Switzerland and San Diego-based biotechnology company is developing RNA-guided regulatory genome targeting therapeutics designed to reprogram disease-driving cell states for both rare and common chronic diseases. This substantial investment signals strong confidence in HAYA's innovative platform and positions the company to accelerate its clinical programs.
Advancing First-in-Class Heart Failure Therapy
A significant portion of the new funding will support the initiation of clinical trials for HTX-001, HAYA's lead therapeutic candidate. HTX-001 is a first-in-class therapy targeting long non-coding RNA (lncRNA) for heart failure, with an initial focus on non-obstructive hypertrophic cardiomyopathy (nHCM).
"This is a defining moment for HAYA as we are advancing our lead program into the clinic," said Samir Ounzain, Ph.D., Co-founder and CEO of HAYA Therapeutics. "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."
Beyond cardiovascular applications, HAYA plans to strengthen its platform capabilities and expand its pipeline across multiple therapeutic areas, including pulmonary fibrosis, obesity, and age-related common and chronic diseases.
Unlocking the "Dark Genome"
At the core of HAYA's approach is its proprietary platform that maps and decodes the biology of the regulatory genome, often referred to as the "genome's dark matter" or "Dark Genome." This region constitutes approximately 98% of the human genome and, while not coding for proteins, serves as the master control layer for gene expression and cell identity.
The company has developed what it describes as the most comprehensive atlas of the regulatory genome by combining integrated multimodal functional genomics with proprietary computational and machine learning methodologies. This approach enables precise identification and modulation of pathogenic cell states across diverse diseases.
Henrijette Richter, Managing Partner at Sofinnova Partners, highlighted the significance of HAYA's approach: "HAYA's platform unlocks the dark genome's therapeutic potential by targeting disease-driving cell states via long non-coding RNAs. This novel approach opens a new frontier in precision medicine, moving beyond traditional target classes to address disease at its epigenetic and cellular roots."
Unlike conventional therapies that primarily manage symptoms, HAYA's next-generation approach aims to directly reprogram the cellular drivers of disease, potentially offering more effective and durable treatment options.
Strategic Growth and Industry Partnerships
To support its ambitious growth trajectory, HAYA recently assembled an industry-leading executive team to drive scalable growth, business development, and clinical advancement. The company has also begun expanding strategic collaborations with pharmaceutical and technology partners.
Notably, HAYA recently announced a partnership with Eli Lilly and Company, described as one of the largest collaborations to date in the regulatory genome space. This partnership focuses on identifying and developing RNA-based drug targets for obesity and metabolic disorders, further validating the potential of HAYA's platform technology.
The Series A funding represents a significant milestone for HAYA as it transitions from a research-focused organization to a clinical-stage company with a diverse pipeline of potential disease-modifying therapies. By targeting the previously underexplored regulatory genome, HAYA aims to pioneer a new class of precision medicines that could fundamentally change treatment paradigms for chronic and complex diseases.
