Biotechnology startup Haya Therapeutics has secured $65 million in Series A funding to advance its pioneering work on drug development targeting the "dark genome" – regions of DNA once dismissed as "junk" but now recognized as critical regulators of gene expression.
The financing round, led by Sofinnova Partners and Earlybird Venture Capital, included participation from pharmaceutical giant Eli Lilly, Alexandria Venture Investments, and eight other investment firms. This latest funding builds upon Haya's $25 million seed round in 2021 and follows a strategic partnership with Eli Lilly focused on obesity that was established last September.
Mining the 'Dark Genome' for Novel Therapeutics
Scientists have increasingly recognized that the vast portions of the human genome that don't code for proteins – previously labeled as "junk DNA" – actually serve crucial biological functions. These regions are transcribed into long non-coding RNAs (lncRNAs), which act as key components in the molecular machinery that regulates gene expression.
Haya is developing computational tools to create an internal "atlas" of the dark genome, helping identify potential drug targets and develop "RNA-guided therapeutics" that can influence them. According to Samir Ounzain, co-founder and CEO of Haya, the company's treatments aim to "reprogram disease-driving cell states into healthy ones."
"What was once dismissed as genetic junk is now understood to be a treasure trove of potential therapeutic targets," Ounzain explained. "Long non-coding RNAs are central to cellular regulation, and by targeting them specifically, we can address disease mechanisms that have proven difficult to treat with conventional approaches."
Advancing Treatment for Non-Obstructive Hypertrophic Cardiomyopathy
The new funding will primarily accelerate Haya's lead program into clinical testing. The company is developing HTX-100, a drug targeting a long non-coding RNA called "Wisper" that is overexpressed in certain cardiovascular conditions, particularly non-obstructive hypertrophic cardiomyopathy (HCM).
Non-obstructive HCM is an inherited heart condition characterized by dangerous thickening of heart tissue and progressive buildup of scar tissue. While Bristol Myers Squibb brought the first drug (Camzyos) to market in 2022 for the more common obstructive form of HCM, the pharmaceutical giant recently failed to demonstrate efficacy in non-obstructive HCM patients.
This setback has created an opportunity for companies with alternative approaches. Unlike competitors Bristol Myers, Cytokinetics, and Edgewise Therapeutics – whose drugs aim to reduce the force of heart contractions – Haya's HTX-100 takes a fundamentally different approach by targeting the underlying disease mechanisms.
"Non-obstructive hypertrophic cardiomyopathy is primarily driven by mechanisms, such as progressive buildup of scar tissue, that current therapies do not adequately target," Ounzain noted in an email. "HTX-100 is designed specifically to address these underlying drivers, as Wisper plays a central role in the fibrosis that occurs."
The scientific foundation for Haya's approach was established in a 2017 paper published in Science Translational Medicine, co-authored by Ounzain in the same year the company was founded. While the concept is scientifically sound, Haya has yet to demonstrate efficacy in human trials.
Expanding Research Portfolio
Beyond its lead program in cardiac disease, the Series A funding will support Haya's earlier-stage programs targeting pulmonary fibrosis and obesity, as well as broader expansion of the company's research capabilities.
Haya joins other biotechnology firms like Rome Therapeutics and NextRNA Therapeutics in the emerging field of dark genome drug discovery. While research in this area remains relatively early and has not yet produced an approved medicine, continued investment signals growing confidence in the approach's potential.
Ounzain indicated that Haya's first clinical trial in non-obstructive hypertrophic cardiomyopathy will commence "in the near future," while competitors Cytokinetics and Edgewise Therapeutics have drugs that are either in or approaching Phase 3 testing for the same condition.
As the race to develop effective treatments for non-obstructive HCM intensifies, Haya's unique approach targeting the dark genome could potentially address a significant unmet medical need for patients with limited therapeutic options.
