Haya Therapeutics Secures $65M to Explore 'Dark Genome' for Novel Therapeutics

Haya Therapeutics, a biotechnology startup focused on developing treatments derived from long non-coding RNAs (lncRNAs), has announced a successful $65 million Series A funding round. The financing, led by Sofinnova Partners and Earlybird Venture Capital, will support the advancement of Haya's lead program for non-obstructive hypertrophic cardiomyopathy (HCM) into clinical trials and fuel the expansion of its research efforts.

Unveiling the Potential of the 'Dark Genome'

Once dismissed as "junk DNA," the non-protein-coding regions of the genome have emerged as a rich source of potential drug targets. These areas, which comprise the majority of the human genome, produce lncRNAs that play crucial roles in gene regulation. Haya Therapeutics is among a growing cohort of companies, including Rome Therapeutics and NextRNA Therapeutics, that are exploring this "dark genome" for novel therapeutic approaches.

Haya's proprietary platform utilizes computational tools to create an internal "atlas" of the dark genome, facilitating the identification of drug targets and the development of "RNA-guided therapeutics." According to CEO Samir Ounzain, these treatments aim to "reprogram disease-driving cell states into healthy ones."

Targeting Non-Obstructive Hypertrophic Cardiomyopathy

The company's lead candidate, HTX-100, targets a lncRNA called "Wisper" that is overexpressed in certain cardiovascular conditions, including HCM. Unlike current treatments for obstructive HCM, which focus on reducing the force of heart contractions, HTX-100 takes a different approach by addressing the underlying mechanisms of the disease, particularly the progressive buildup of scar tissue.

This novel strategy could potentially fill a significant gap in the treatment landscape for non-obstructive HCM. The recent failure of Bristol Myers Squibb's Camzyos in non-obstructive HCM patients has raised questions about the efficacy of traditional approaches in this subset of the disease, opening opportunities for innovative therapies like HTX-100.

Expanding Research and Industry Partnerships

The Series A funding builds upon Haya's previous $25 million seed round in 2021 and a strategic partnership with Eli Lilly focused on obesity research. The latest financing round included participation from Eli Lilly, Alexandria Venture Investments, and eight other investment firms, underscoring the growing interest in lncRNA-based therapeutics.

Haya plans to use the funds to advance its lead program into clinical trials in the near future, while also supporting earlier-stage programs in pulmonary fibrosis and obesity. As the company moves forward, it will face competition from established players like Cytokinetics and Edgewise Therapeutics, which are currently conducting or preparing for Phase 3 trials in HCM.