HAYA Therapeutics to Present Breakthroughs in RNA-Guided Regulatory Genome Platform at 2025 ASGCT Annual Meeting
HAYA Therapeutics, SA, a biotechnology company at the forefront of developing precision RNA-guided therapeutics that reprogram pathogenic cell states, announced today its participation at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place from May 13–17, 2025, in New Orleans, Louisiana. The company will deliver an invited presentation as well as a scientific poster, showcasing new data from its leading platform and pipeline innovations designed to address a broad range of rare, chronic, and age-associated diseases.
HAYA Therapeutics is pioneering a next-generation therapeutic paradigm that targets long non-coding RNAs (lncRNAs) within the regulatory genome — the regions of the genome that orchestrate gene expression programs governing cellular identity and function. Unlike conventional therapies that typically aim to block proteins or single genes, HAYA’s strategy focuses on modulating upstream, non-coding RNA elements that serve as master regulators of complex disease processes. This approach enables the reprogramming of aberrant cell states at the root of pathological conditions such as fibrosis, cancer, and metabolic and cardiovascular diseases.
At the ASGCT 2025 Annual Meeting, HAYA’s scientific leadership will present new findings from its lead program, HTX-001, an antisense oligonucleotide (ASO) therapeutic targeting Wisper, a cardiac-enriched long non-coding RNA identified as a master regulator of myocardial fibrosis. The presentation will highlight preclinical data from HTX-001, which is currently advancing through Investigational New Drug (IND)-enabling studies. HTX-001 is initially being developed for the treatment of non-obstructive hypertrophic cardiomyopathy (nHCM), a serious and often underdiagnosed cardiac disorder characterized by abnormal thickening of the heart muscle and progressive fibrosis.
In addition to the oral presentation, HAYA will unveil a poster focused on its proprietary multimodal omics platform, which integrates transcriptomics, epigenomics, and spatial genomics to identify and validate novel lncRNA targets. This platform serves as the foundation for designing ASO-based therapies with unprecedented specificity and efficacy, enabling a new level of precision medicine for diseases driven by dysregulated gene expression and cellular misprogramming.
Presentation Details:
- Invited Talk: RNA-Guided Reprogramming of Fibrotic Cell States via lncRNA Targeting – Preclinical Development of HTX-001
- Presenter: Dr. Samir Ounzain, Ph.D., Co-founder and CEO, HAYA Therapeutics
- Session: RNA Therapeutics: Emerging Modalities and Disease Applications
- Poster Title: Multimodal Omics-Driven Discovery of Long Non-Coding RNA Targets for Precision Antisense Therapeutics
- Presenting Author: HAYA Therapeutics Scientific Team
“Unlike traditional approaches that block individual proteins or gene products, our strategy is to reprogram the core regulatory networks that determine whether a cell becomes healthy or diseased,” said Samir Ounzain, Ph.D., Co-founder and CEO of HAYA Therapeutics. “By targeting long non-coding RNAs in the regulatory genome — which act as command centers for disease-driving gene expression programs — we are able to intervene much earlier and more effectively in the disease cascade. This enables us to design medicines that don’t just treat symptoms, but fundamentally alter disease progression.”
HTX-001: A First-in-Class Therapy for Cardiac Fibrosis
HTX-001 represents a new class of disease-modifying therapeutics designed to halt and potentially reverse fibrotic remodeling in the heart. Myocardial fibrosis is a central pathological feature of various forms of heart failure and cardiomyopathy, including nHCM. Despite its clinical significance, effective anti-fibrotic therapies remain elusive.
The target of HTX-001, Wisper (Wisp2 super-enhancer-associated RNA), is a cardiac-specific long non-coding RNA discovered by HAYA’s research team and previously validated in translational studies. Wisper is selectively expressed in activated cardiac fibroblasts — the cells responsible for laying down excess extracellular matrix that stiffens the heart muscle and impairs its function. By silencing Wisper, HTX-001 has demonstrated the ability to attenuate fibroblast activation, reduce fibrosis, and preserve cardiac function in preclinical models of heart failure.
“Our work on HTX-001 has shown that Wisper is not just a biomarker of fibrosis — it is a driver of the pathological fibroblast phenotype,” said Daniel Blessing, Ph.D., Co-founder and Chief Technology Officer of HAYA Therapeutics. “We believe HTX-001 has the potential to redefine the treatment landscape for patients with nHCM and potentially other forms of cardiac fibrosis. We are excited to share our latest progress and anticipate initiating clinical trials in the near future.”
HTX-001 is currently in advanced IND-enabling studies, including pharmacology, toxicology, and formulation optimization. The program is on track for a planned regulatory filing and subsequent entry into Phase 1 clinical trials.
Transforming Drug Discovery with the Regulatory Genome Platform
HAYA Therapeutics’ core innovation lies in its Regulatory Genome Targeting Platform, a comprehensive discovery engine that maps, decodes, and therapeutically modulates disease-relevant lncRNAs. The platform integrates high-throughput sequencing technologies, machine learning algorithms, and proprietary databases of human regulatory elements to uncover previously unrecognized drivers of disease biology.
Through this approach, HAYA is building a robust pipeline of antisense oligonucleotide therapeutics aimed at targeting disease-causing cell states in a tissue-specific and mechanism-informed manner. In addition to HTX-001, the company is advancing multiple discovery-stage programs across fibrosis, oncology, metabolic disorders, and age-related diseases.
The company’s poster presentation at ASGCT will delve into the multimodal omics approach that underpins its discovery efforts. This methodology combines single-cell RNA sequencing, chromatin accessibility mapping (ATAC-seq), and spatial transcriptomics to comprehensively profile diseased tissues and uncover actionable lncRNA targets. Once identified, these targets are functionally validated and optimized for silencing using chemically modified antisense oligonucleotides with favorable pharmacokinetic and safety profiles.
“Our platform doesn’t just identify novel lncRNA targets — it provides the roadmap to precisely drug them,” Dr. Blessing added. “This allows us to design ASOs that can penetrate the nucleus, bind to their RNA targets with high specificity, and efficiently degrade them, leading to durable modulation of disease-driving pathways.”
A New Frontier in RNA Therapeutics
The scientific community is increasingly recognizing the critical role of non-coding RNAs in regulating cellular function and disease. While messenger RNAs (mRNAs) and proteins have historically dominated the therapeutic landscape, non-coding RNAs represent a vast and largely untapped class of targets with transformative potential.
HAYA Therapeutics is one of the few companies systematically pursuing lncRNA-based therapeutics, and its early results suggest that the regulatory genome may hold the key to durable disease modification across a broad range of indications.
“The biology of the regulatory genome is incredibly rich and complex, but also highly druggable with the right tools,” Dr. Ounzain said. “We are proud to be contributing to this emerging field and look forward to engaging with the scientific and medical community at ASGCT as we continue to build momentum toward the clinic.”
Founded in Lausanne, Switzerland, HAYA Therapeutics is a biotechnology company developing RNA-guided therapeutics that reprogram disease-driving cell states by targeting the regulatory genome. The company’s proprietary discovery platform enables the identification and therapeutic silencing of long non-coding RNAs that orchestrate pathogenic gene expression programs. HAYA’s lead program, HTX-001, targets the cardiac-specific lncRNA Wisper and is in development for non-obstructive hypertrophic cardiomyopathy. The company is advancing a broad pipeline of ASO-based therapies for fibrosis, cancer, and other chronic diseases with high unmet medical need.
