Many medications available and in development development pointing to proteins, an approach that comes with limitations. On the one hand, proteins are not the root cause of the disease, said Samir Ouszain, co -founder and CEO of Hay Therapeutics. The promoter of the disease is the response of the cells to their surroundings. The regulation of this response occurs in a part of the genome once overlooked and equally discarded by many scientists such as garbage.
The vast majority of the genome, about 98%, consists of DNA that does not encode proteins. Called “garbage DNA” because scientists thought it had no function, research in the last decade has found that this non -coding DNA plays a key role in the regulation of gene expression, including disease states, Ouszain said. While non -coding DNA referred to the dark genome, Ouszain said it is called more than the regulatory genome. This portion of the genome, not protein, is what controls the cells and leads to the disease.
Haya, based in Lausana, Switzerland, and maintains US operations in San Diego, drug development intends to address the objectives in the dark genome to reprogram the cells that conduct the disease. On Thursday, the startup released $ 65 million to begin human evidence with a main therapeutic candidate who could demonstrate this approach to a type of heart failure.
“We need to think about the fundamental unity of progression of the disease and drive in diseases, such as the cell, not a gene, not a path, they are cell states,” said Ouszain. “The cells are what manifest the pathophysiology that supports many of these indications. And if the question is asked where the root cause, the causal biology that controls the cellular states, everything is in the dark genome. That is worse for the financing of demons, we are excited to prove that clinically.”
Haya is the product of Ouszain’s academic research, more recently at the Hospital of the University of Lausana. He said he spent his academic career trying to decipher and unlock the meaning of garbage DNA. Ounzain published some of the first scientific articles that show that the dark genome produces non -coding (LNCRNA) RNAs that regulate specific genes of certain diseases. The investigation led him to cell states in the cardiovascular system, including fibrosis driven disorders, the formation of scar tissue that harms heart function.
Antifibrotic drugs that are directed to proteins face a particular challenge. The same protein that causes the disease in a cell type is also found in other types of cells in the body, said Ouszain. Consistently, a drug designed to address this protein can also hit tissues outside the target, generating security problems. But using a lower dose to reduce this risk of complication means that a earlier is effective.
Haya has developed a platform technology that applies computational biology and automatic learning techniques to patient biopsies. The analysis of these samples allows to build integrated data sets that Ounzain calls an “atlas” or the regulatory genome. With this Atlas, the company develops medicines guided by RNA to reprogram the cells that drive a disease. When studying the dark genome, it has identified the lncrnas that control fibrosis in a fabric, Ounzain said. Ha are developing medications that pursue specific objectives to reprogram fibroblasts, the type of cell that leads to fibrosis.
The Hague drugs are mainly antisntido oligonucleotides, but the company is also investigating small interferential RNA therapies, Bolh or which are established modalities. The main HTX-001 program is an oligonucleotide designed to direct the RNA associated with the super environment of Wisp2 (Wisper), a LNCRNA that regulates the fibroblast activity. The preclinical investigation showed that this approach can potentially block cardiac fibrosis, said Ouzain. Because Wisper is alone in the cardiac tissue, pointing it should not lead for the purpose of noticing in other parts of the body.
“In Tox Tox[icity] In the tissues outside the objective, it is a characteristic that we do not see, “said Ouszain.” It will never involve those objectives outside the cells that lead to the disease because they did not express, they are not active. “
The ability to offer better efficiency and safety in genetic medicine allows them to obtain common and chronic diseases. The objective of lead disease is the non -obstructive hypertrophic myocardiopathy (HCM), a genetic condition in which the heart muscle muscle is thick but does not block the organ flow of the organ. While this disorder does not prevent blood flow, it still makes it difficult for the heart to pump blood and can cause heart failure. The non -obstructive HCM arose as the main indication of Hague, since the company’s research showed a very strong association between the fibrotic load and cardiac dysfunction experienced by patients with this disease.
HCM drug treatment includes older heart drugs, such as beta blockers. Bristol Myers Squibb Drug Camzyos, a small oral molecule designed to block a protein that leads to the thickness of the heart muscle, was approved in 2022 as a treatment for obstructive HCM. But the recent drug failed a phase 3 test aimed at supporting the expansion of the medication to non -obstructive HCM. Imbria Pharmaceuticals aims to treat the non -obstructive HCM with a medication called ninerafaxstat. The startup last month raised $ 57.5 million to advance in this small oral molecule to phase 2b tests.
Ouszain said that achieving a non -obstructive HCM proof could pave the way for there to be looking for other types of heart failure. The startup also has a separate program that addresses pulmonary fibrosis. Hay aims to maintain heart failure and pulmonary fibrosis research at home, but Ouszain does not rule out associations if they could bring patients therapies faster. Out of those main indications, the startup has a research alliance with Eli Lilly. Last September, Lilly signed an agreement to collaborate with Haya to discover drugs for metabolic indications, including obesity. The specific financial details were not revealed, but the companies said that the payment in advance, the capital investment and the payments of milestones could reach up to $ 1 billion.
There was a stealth backed by 18 million Swiss francs (around $ 20 million) in seed financing directed by Broadview Ventures. The new financing announced on Thursday was led by Sofinnova Partners and Earlybird Venture Capital. Other participants in financing include Eli Lilly, Athos, +ND Capital, Alexandria Venture Investments and Lifelink Ventures. The previous investors Apollo Health Ventures, Longview Ventures (a Broadview affiliate), 4see Ventures, Bernina Bioinvest and Schroder Capital also participated in the last minimum.
With the new capital, Haya aims to start phase 1 HTX-001 tests in the first half of 2026, Ouszain said. In the closest term, Haya, who prepares to present preclinical data for its main floors ora -class guidance program next week, is the annual meeting of the American Society of Genetic and Cell Therapy. Hay will have another presentation focused on its technological platform. The meeting will also give Ouszain the opportunity to explain to the scientific community the name of the company, which is inspired by “Hayah”, a word with the same meaning both in Hebrew and in Arabic.
“Real means life, so [the name is] Really ‘therapeutic of life, “said Ouszain.” We hope to extend the health capacity and give life to patients who unfortunately suffer from many of common chronic diseases that affect society. “
Photo: Champipixs, Getty Images
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