Many drugs currently available and in development target proteins, but this approach has its limitations, according to Samir Ounzain, co-founder and CEO of HAYA Therapeutics. Ounzain argues that proteins are not the root cause of disease; instead, it is the response of cells to their environment that drives disease. This response is regulated by a part of the genome that was once dismissed as “junk” by many scientists.
Recent research has shown that the vast majority of the genome, about 98%, consists of non-coding DNA that does not produce proteins. This non-coding DNA, previously referred to as “junk DNA,” actually plays a crucial role in regulating gene expression and disease states, Ounzain explained. He prefers to call it the regulatory genome rather than the dark genome, emphasizing its role in controlling cell behavior and contributing to disease.
HAYA Therapeutics, based in Lausanne, Switzerland, and San Diego, is focusing on developing drugs that target the regulatory genome to reprogram disease-driving cells. The company recently secured $65 million in funding to initiate human trials with a lead therapeutic candidate aimed at addressing heart failure.
Ounzain’s academic research, particularly at Lausanne University Hospital, led him to discover the importance of non-coding DNA in regulating gene expression. This research identified long non-coding RNAs (lncRNAs) in the dark genome that influence specific diseases, particularly those related to cell states in the cardiovascular system.
HAYA’s approach involves using computational biology and machine learning techniques to analyze patient biopsies and create an “atlas” of the regulatory genome. By targeting specific lncRNAs identified in the dark genome, the company aims to reprogram cells involved in disease processes, such as fibrosis in heart failure.
The lead program, HTX-001, focuses on targeting a specific lncRNA called Wisper that regulates fibroblast activity in cardiac tissue. Preclinical studies have shown promising results in blocking and potentially reversing cardiac fibrosis without affecting other tissues in the body.
HAYA’s innovative approach aims to offer better efficacy and safety in genetic medicine, allowing the company to pursue common chronic diseases like non-obstructive hypertrophic cardiomyopathy (HCM). By targeting the regulatory genome, HAYA hopes to address the underlying causes of heart failure and other diseases driven by cell dysfunction.
While traditional treatments for HCM involve older heart drugs or new small molecules that target specific proteins, HAYA’s approach focuses on reprogramming disease-driving cells at the genetic level. This novel strategy could pave the way for more effective and targeted therapies for a range of cardiovascular and respiratory conditions.
In addition to its work on heart failure and pulmonary fibrosis, HAYA has established partnerships with leading pharmaceutical companies like Eli Lilly to explore new therapeutic targets for metabolic conditions such as obesity. These collaborations reflect the growing interest in leveraging the regulatory genome to develop innovative treatments for a variety of diseases.
Overall, HAYA Therapeutics is at the forefront of a new era in genetic medicine, leveraging the power of the regulatory genome to revolutionize the treatment of complex diseases. With its cutting-edge technology and strategic partnerships, the company is poised to make significant advances in the field of precision medicine.
Inversores anteriores Apollo Health Ventures, Longview Ventures (un afiliado de Broadview), 4see ventures, BERNINA Bioinvest y Schroder Capital también participaron en la última financiación.
Con el nuevo capital, HAYA tiene como objetivo comenzar las pruebas de la Fase 1 de HTX-001 en la primera mitad de 2026, dijo Ounzain. En el corto plazo, HAYA se está preparando para presentar datos preclínicos de su programa principal dirigido a Wisper en Nueva Orleans la próxima semana durante la reunión anual de la Sociedad Americana de Terapia Génica y Celular. HAYA tendrá otra presentación centrada en su plataforma tecnológica. La reunión también le dará a Ounzain la oportunidad de explicar a la comunidad científica el nombre de la empresa, que está inspirado en “hayah”, una palabra con el mismo significado en hebreo y árabe.
“En realidad significa vida, así que [el nombre es] realmente ‘Life Therapeutics’”, dijo Ounzain. “Esperamos extender la duración de la vida y llevar vida a los pacientes que desafortunadamente están sufriendo muchas de las enfermedades crónicas comunes que están aquejando a la sociedad.”
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