HAYA Therapeutics SA

Haya plans to use the funds to commence trials for HTX-001, initially aiming to target non-obstructive hypertrophic cardiomyopathy (nHCM). Credit: PanuShot / Shutterstock. Haya Therapeutics has procured $65m in Series A funding to expedite the clinical development of HTX-001 for treating heart failure. The financing also aims to support the expansion of its RNA-guided regulatory genome pipeline development engine. The Series A round was spearheaded by Earlybird Venture Capital and Sofinnova Partners. It also saw contributions from Eli Lilly, +ND Capital, ATHOS, LifeLink Ventures, and Alexandria Venture Investments. Additional financing came from current investors such as Apollo Health Ventures, BERNINA Bioinvest, and 4see ventures. Haya plans to use the funds to initiate clinical trials for the long non-coding RNA (lncRNA), HTX-001, initially aiming to target non-obstructive hypertrophic cardiomyopathy (nHCM). Concurrently, the company aims to bolster its platform offerings and broaden its disease-modifying therapeutic pipeline, addressing conditions such as obesity, age-related diseases, and pulmonary fibrosis. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Haya Therapeutics CEO and co-founder Samir Ounzain said: “This funding validates our organisation, pioneering approach, and vision for improving the way chronic and complex diseases are treated by creating a new generation of therapies that reprogramme disease-driving cell states into healthy ones. “We’re excited that Sofinnova Partners, Earlybird, and our syndicate of investors share our vision for the potentially industry-changing nature of our platform as we move beyond traditional approaches by leveraging novel therapeutic targets emerging from the regulatory genome.” The core of Haya’s platform lies in mapping and decoding the “dark genome”, the regulatory genome that constitutes 98% of the human genome. By combining integrated multimodal functional genomics with a proprietary computational stack and machine learning techniques, Haya claims to have created a comprehensive atlas of the regulatory genome. This platform is said to facilitate RNA-guided therapeutic development, offering scalability and speed. Haya’s approach aims to transcend symptom management by reprogramming cellular disease drivers. Last September, Eli Lilly teamed up with Haya Therapeutics in a $1bn deal to identify multiple regulatory-genome-derived RNA-based drug targets, as it looks for new targets in obesity.

After working off $20 million in seed funding for about four years, HAYA Therapeutics tapped the funding well and came back with $65 million to get its cardiac fibrosis treatment candidate into human trials. The Swiss biotech plans to use the Series A funds to help get HTX-001 into the clinic early next year, CEO Samir Ounzain told Endpoints News . It first wants to assess the long non-coding RNA-targeting candidate in patients with non-obstructive hypertrophic cardiomyopathy. HAYA is one of a handful of drug developers working in the “dark genome” space, and the Series A, which was announced Thursday morning, marks a pivotal moment for the startup as it moves from a longstanding seed-stage outfit to a near-clinical company. Many preclinical biotechs, especially those trying to advance new scientific ideas, have struggled to weather the industry’s yearslong funding downturn. The biotech, which has about 40 employees, seeks to reprogram cell states that spur common, chronic and age-related diseases. It’s an ambitious mission, but HAYA now has blue-chip European investors like Sofinnova Partners and Earlybird Venture Capital to bankroll its R&D. Additional investors include ATHOS, +ND Capital, Alexandria Venture Investments and LifeLink Ventures, plus existing backers like Apollo Health Ventures and others. “What actually is driving outcomes in common and chronic disease? It’s cells changing identity. It’s cell stage changes,” Ounzain said. “What regulates cell state changes? It’s your environment and your common genetic variation that predisposes how you respond to the environment. Where are those signals interpreted by your cell? It’s in the dark genome. RNA produced from the dark genome transacts that and can be targeted. That resonated strongly with investors.” The capital injection arrives two months after HAYA named Richard Law as its chief business officer, highlighting the former Exscientia executive’s experience in biopharma dealmaking and fundraising. HAYA signaled outsider confidence in its platform last fall, when Eli Lilly stepped in for an up to $1 billion bet that the fledgling biotech could help the Indianapolis behemoth make new medicines for obesity and metabolic diseases. The companies kept mum on the specific breakdown of the upfront and milestone payments. Elsewhere in the dark genome space, NextRNA also forged a large pharma pact last year with Bayer in cancer. Meanwhile, Rome Therapeutics, which has the backing of multiple drug giants , laid off 17 employees in December as it refocused its pipeline on autoimmune and neurodegenerative diseases and deprioritized other areas of dark genome research. Ounzain said HAYA may explore additional partnerships to take its platform into other therapeutic areas. Beyond its lead cardio drug, HAYA has a second project in the works for pulmonary fibrosis and a third idea generating steam for the tumor microenvironment, according to Ounzain.

Haya Therapeutics is approaching chronic disease like it's a linguistics problem. The biotech, which raised a $65-million series A on Thursday, is mapping the 'dark genome' — genes that don't encode for proteins — and is using that data almost like a Rosetta Stone to understand how cells communicate with the environment.  According to Samir Ounzain, Haya's co-founder and chief executive, the dark genome encompasses nearly 98% of the human genome. While it used to be thought of as 'junk DNA,' researchers now believe those genes play a key role in regulating gene expression and cell state."That 98% of your genome is really the software of the cell. It allows your cell to speak to the environment in very sophisticated, dynamic ways. And common and chronic diseases are really diseases caused by the environment," Ounzain told FirstWord. "So if you understand the language — how does your cell and how does your genome speak to the environment — you can start to make common and chronic diseases tractable to therapeutic intervention."Haya is integrating multimodal omics with machine learning and computational biology to map genes within the dark genome that could play a role in regulating cell states associated with disease. By deciphering the language of the genome, it  pinpoints RNA-based drivers at the root cause of these conditions, Ounzain said.The biotech then aims to manipulate those RNAs with oligonucleotide therapies. "If you can target those RNAs, you can, in a very precise way, start to reprogramme how the genome speaks to the environment and change the cell state — turn a cell into a healthy cell," Ounzain explained. Fibrosis firstHaya's lead candidate, HTX-001, is directed against an undisclosed long non-coding RNA target to treat heart failure. Thursday's financing, led by Sofinnova Partners and Earlybird Venture Capital, will help Haya bring the asset into the clinic in early 2026 for non-obstructive hypertrophic cardiomyopathy (nHCM).Heart failure can be caused by two symptoms on the opposite ends of the spectrum: either heart stiffness — called diastolic dysfunction — or over-contractility. While cardiac myosin inhibitors seem to be effective at tackling contractility, there are no approved medicines for diastolic dysfunction in the heart. "It's becoming very clear that stiffness and diastolic dysfunction is dose-dependently correlated with fibrosis. So if you can create a precision anti-fibrotic agent that can block fibrosis progression or potentially reverse it, you start to unlock all diastolic dysfunction in heart failure," Ounzain said. "And we believe a very large significant unmet need is the patients who are symptomatic for diastolic dysfunction as a consequence of hypertrophic cardiomyopathy… and we want to bring a solution in the non-obstructive patient population."He added that once Haya generates clinical proof-of-mechanism data, it plans to evaluate HTX-001 in other types of heart failure. For now, Haya is focusing its internal pipeline on fibrosis-related diseases, but the biology it has uncovered via its dark genome map "applies to any cell type that's responding to the environment," he said. "We want to unlock the dark genome for genetic medicines in many, many different areas," Ounzain commented. "And partnering and collaborating is going to be at the centre of that."Haya has already inked an agreement with Eli Lilly, worth up to $1 billion, to search the dark genome for obesity and metabolic disease candidates. The pharma also participated in Thursday's financing. Other investors in the round include ATHOS, +ND Capital, Alexandria Venture Investments, LifeLink Ventures, Apollo Health Ventures, Longview Ventures, 4see ventures, BERNINA Bioinvest and Schroder Capital.