HAYA Therapeutics, SA, a biotechnology company pioneering precision RNA-guided regulatory genome targeting therapeutics aimed at reprogramming disease-driving cell states for rare, common, chronic, and age-related diseases, today announced that it will present data at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans from May 13-17, 2025.

Our platform goes beyond traditional methods of blocking single genes or proteins by reprogramming the fundamental biology driving disease, said Samir Ounzain, Ph.D., Co-founder and CEO of HAYA Therapeutics.

HAYA will highlight its progress with an invited presentation on its lead program, HTX-001, an antisense oligonucleotide targeting the long non-coding RNA (lncRNA) Wisper, the master regulator of fibrosis in the heart. HTX-001 is currently in Investigational New Drug-enabling studies and is being developed for treating non-obstructive hypertrophic cardiomyopathy (nHCM). Additionally, the company will feature a poster presentation on its innovative multimodal omics approach, which allows precise and potent silencing of novel lncRNA targets for antisense oligonucleotide-based therapeutics.

Our approach goes beyond blocking individual genes or proteins—we’re reprogramming the fundamental biology behind diseases, said Samir Ounzain, Ph.D. We’re discovering and targeting novel regulatory genome-derived drivers to reprogram sick cells into healthy ones.

We’ve made significant progress with HTX-001, targeting myocardial fibrosis, and are on track to initiate clinical trials soon. This progress brings us closer to our goal of delivering disease-modifying therapies to patients, said Daniel Blessing, Ph.D., Co-founder and CTO of HAYA Therapeutics.

Poster Presentation:
Title: Harnessing Functional Genomics to Advance the Discovery and Development of Oligonucleotide-Based Therapies Targeting the Regulatory Genome
Presenter: Rudi Micheletti, Senior Director of Biology and Functional Genomics at HAYA
Date/Time: Tuesday, May 13, 2025, 6:00 PM - 7:30 PM CDT
Location: Poster Hall I2
Abstract Number: 660

Scientific Symposium:
Title: A Wisper from the Heart: Targeting a Cardiac Myofibroblasts-specific LncRNA to Treat Myocardial Fibrosis
Presenter: Daniel Blessing, CTO and Co-founder of HAYA
Session Title: Targeting Myocardium: To The Heart Of The Matter (Organized by the Cardiovascular CGT Committee)
Date/Time: Wednesday, May 14, 2025, 3:45 PM - 5:30 PM CDT
Location: Room 388-390

HAYA’s RNA-guided therapeutic platform represents a groundbreaking approach to treating disease at the cellular level by targeting the root causes. Central to the platform is HAYA’s Regulatory Genome Atlas, which maps connections between noncoding regulatory genome elements, such as lncRNAs, and disease-driving cell states. By integrating multimodal functional genomics with proprietary computational tools and machine learning, HAYA is developing genetic medicines designed to reprogram diseased cells into healthy ones.

Source: https://www.businesswire.com/news/home/20250502322005/en/HAYA-Therapeutics-to-Present-on-RNA-Guided-Regulatory-Genome-Platform-at-the-Annual-Meeting-of-the-American-Society-of-Gene-Cell-Therapy-ASGCT