HAYA
Therapeutics

The company that decided junk DNA had a job

In a lab in Lausanne and a second one in San Diego, a team of about sixty-seven people is doing something most of pharma spent decades avoiding: drugging RNA that doesn't build proteins. HAYA Therapeutics calls this territory the regulatory genome. Biologists used to call it junk. The distinction matters, because in May 2025 investors put $65 million behind the idea that the junk was mislabeled.

HAYA is a clinical-stage precision medicines company. Its lead drug, HTX-001, is an antisense oligonucleotide - a short, designed strand of genetic material - aimed at a single cardiac RNA called Wisper. The goal is heart failure, starting with a hard-to-treat form called non-obstructive hypertrophic cardiomyopathy. Around that one program sits a platform built to do the same trick for fibrosis, metabolism and cancer.

Chronic disease doesn't have a target problem. It has a wrong-target problem.

Here is the uncomfortable fact HAYA was built on. The human genome is roughly 98% non-coding. For years, drug developers crowded into the other 2% - the protein-coding stretch - because that's where the well-understood targets lived. It was, to borrow a phrase, like searching for keys under the streetlight because the light is better there.

Chronic and age-related diseases - heart failure, fibrosis, obesity, many cancers - are messy. They aren't one broken protein. They are whole populations of cells locked into the wrong behavior, a "disease-driving cell state." And the instructions for those states are written largely in the non-coding genome, in long non-coding RNAs that turn programs on and off with surgical, cell-type specificity.

The problem, then, was never a shortage of drugs. It was a shortage of the right addresses. HAYA's bet is that the addresses are in the dark.

A hospital researcher who kept finding RNAs nobody had names for

Before HAYA, Samir Ounzain was a research fellow at Lausanne University Hospital (CHUV), where his lab discovered hundreds of cardiac-enriched long non-coding RNAs and gave them names - CARMEN, Meteor, Wisper. Naming things is the privilege of people who found them first. In 2019 he and co-founder Daniel Blessing turned that discovery work into a company, securing an exclusive license to the Wisper asset from CHUV.

Ounzain took the CEO seat; Blessing became CTO. In 2024 the pair were handed the Andreas & Thomas Struengmann Award for visionary scientific entrepreneurship - which is a long way of saying the people who fund European biotech think the bet is a serious one.

Two co-founders, one licensed RNA, and the quiet confidence of people who already named the molecule they're now trying to drug.

One platform, many cell states to reprogram

The engine room is HAYA's regulatory genome platform: multimodal functional genomics fused with computational and AI tools. It maps which non-coding RNAs actually cause a disease state - not merely correlate with it - and then designs RNA-guided therapeutics to switch them off. The point of attacking a regulatory RNA rather than a protein is reach: one switch can govern a whole cellular program, in one cell type, leaving the rest of the body alone.

• HTX-001 - lead candidate. An antisense oligonucleotide silencing the cardiac lncRNA Wisper, for heart failure (initially non-obstructive hypertrophic cardiomyopathy).
• Fibrosis programs - including pulmonary fibrosis, where scarring cell states are the enemy.
• Obesity & metabolic disease - the focus of the Eli Lilly collaboration.
• Oncology - reprogramming tumor-driving cell states via regulatory RNA.

When the partner is also the patient's biggest pharma

Skeptics of platform biotech ask one fair question: does anyone serious actually want this? In September 2024, Eli Lilly answered. The two signed a multi-year deal to find regulatory-genome RNA targets for obesity and related metabolic conditions - HAYA taking an upfront payment plus equity, and eligible for up to $1 billion in milestones and royalties. Lilly then turned up again in the Series A, and HAYA moved its San Diego lab straight into Lilly Gateway Labs.

The $65M round itself reads like a who's-who of life-science capital: Sofinnova Partners and Earlybird Venture Capital leading, with ATHOS, +ND Capital, Alexandria Venture Investments, LifeLink Ventures, and returning backers Apollo Health Ventures, Longview Ventures, 4see ventures, BERNINA Bioinvest and Schroders Capital.

Programming lasting health

HAYA's stated mission is to bring the promise of the regulatory genome to life through programmable RNA medicines - with, in its own words, "speed and precision in execution." The phrase on the front door is shorter: Programming Lasting Health. Underneath the slogan is a genuinely different theory of chronic disease, which holds that aging and chronic illness are less about broken parts and more about cells stuck running the wrong software.

If that's right, the job isn't to replace the hardware. It's to rewrite the instructions. That's an unusually patient ambition for an industry that often prefers quick wins - and it's the reason the company keeps using the word "lasting."

If the dark genome is real estate, HAYA got there early

Back to that lab in Lausanne, and the second one now sitting inside its partner's building in San Diego. The streetlight metaphor has flipped. For decades the light was best over the protein-coding 2%, so that's where everyone searched. HAYA wandered into the dark, brought its own instruments, and started reading the 98% that was always there.

None of this is settled. HTX-001 still has to clear the clinic, and platform biotechs have a long history of promising more genome than they deliver. But the company that once got a "hiya!" pun for a headline now has a billion-dollar pharma partner, a $65M war chest, and a lead drug pointed at one of medicine's most stubborn problems. The junk drawer, it turns out, may have been the control panel all along.

The same lab, the same twelve letters - only now the part of the genome they claimed has a price tag, a partner, and a drug heading for patients.