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A biotech startup called HAYA Therapeutics has raised $65 million in Series A funding to advance a new class of precision medicines aimed at chronic and age-related diseases. Published in company statements and industry media, the work is led by HAYA’s CEO and co-founder Dr. Samir Ounzain. The funding will accelerate the development of HAYA’s lead drug candidate, HTX-001, which targets heart failure by modulating a little-known section of the genome called the regulatory genome—often referred to as the “dark genome.”
Unlike traditional medicines that focus on protein-coding genes, HAYA’s approach targets long non-coding RNAs (lncRNAs)—segments of RNA that don’t make proteins but control how and when genes are expressed. These lncRNAs are believed to act like “software” in the cell’s operating system, helping to regulate cell identity and function. Using machine learning and high-throughput genomics, HAYA has built an atlas of these lncRNAs, allowing them to design RNA-guided drugs that precisely reprogram diseased cells into healthy ones.
HTX-001, the company’s lead therapeutic, is being developed to treat non-obstructive hypertrophic cardiomyopathy (nHCM), a type of heart failure caused by scarring of the heart tissue. Current treatments often affect many tissues and come with side effects, but HAYA’s RNA-targeting method aims to minimize these risks by acting only where needed—on the specific lncRNAs that trigger fibrosis in the heart.
Beyond heart disease, HAYA’s platform shows promise in tackling other chronic conditions like pulmonary fibrosis, obesity, and even cancer. A newly announced partnership with pharma giant Eli Lilly will focus on developing RNA-based therapies for obesity and metabolic disorders, further validating the potential of HAYA’s lncRNA-guided approach. According to investors like Sofinnova’s Henrijette Richter, HAYA is opening “a new frontier in precision medicine” by exploiting the dark genome to treat disease at its root.
