HAYA Therapeutics Company Profile

Background

HAYA Therapeutics, founded in 2019, is a precision medicines company specializing in the development of RNA-guided programmable therapeutics targeting the regulatory genome. The company's mission is to identify and develop breakthrough disease-modifying therapeutics with enhanced efficacy, safety, and accessibility. By focusing on long non-coding RNAs (lncRNAs) within the "dark genome," HAYA aims to reprogram disease-driving cell states, addressing serious health conditions such as cardiovascular diseases and cancer.

Key Strategic Focus

HAYA's strategic focus centers on leveraging its proprietary platform to map and decode the biology of the regulatory genome, enabling the precise identification and modulation of pathogenic cell states across diverse diseases. The company's lead therapeutic candidate, HTX-001, is an antisense oligonucleotide targeting the lncRNA Wisper, a cardiac tissue-enriched driver of fibrosis in the heart, developed for the treatment of non-obstructive hypertrophic cardiomyopathy (nHCM). Additionally, HAYA is expanding its pipeline to include lncRNA-targeting candidates for the treatment of fibrotic diseases in other tissues, including lungs, kidneys, liver, and the microenvironment of solid tumor cancers.

Financials and Funding

HAYA Therapeutics has successfully secured significant funding to advance its research and development initiatives:

Seed Financing (May 2021): Closed a CHF 18 million seed round led by Broadview Ventures, with participation from Apollo Health Ventures, BERNINA BioInvest, 4See Ventures, Schroder Adveq, and Viva BioInnovator.

Seed Extension (February 2022): Raised an additional US$5 million in a seed extension led by Humboldt Fund, bringing the total seed funding to approximately US$25 million.

Series A Funding (May 2025): Secured $65 million in Series A funding led by Sofinnova Partners and Earlybird Venture Capital, with participation from Eli Lilly and Company, ATHOS, +ND Capital, Alexandria Venture Investments, and LifeLink Ventures.

The funds are allocated to advance the clinical development of HTX-001 and expand the company's RNA-guided regulatory genome pipeline.

Pipeline Development

HAYA's pipeline is anchored by its lead candidate, HTX-001, an antisense oligonucleotide targeting the lncRNA Wisper, developed for the treatment of non-obstructive hypertrophic cardiomyopathy (nHCM). HTX-001 is currently in Investigational New Drug (IND)-enabling studies, with plans to initiate clinical trials in the near future.

Beyond HTX-001, HAYA is utilizing its proprietary platform to develop a pipeline of lncRNA-targeting candidates for the tissue-specific treatment of fibrotic diseases in other tissues, including lungs, kidneys, liver, and the microenvironment of solid tumor cancers.

Technological Platform and Innovation

HAYA's proprietary platform focuses on the regulatory genome, often referred to as the "dark genome," which constitutes 98% of the human genome. This platform combines integrated multimodal functional genomics with proprietary computational and machine learning methodologies to map and decode the biology of the regulatory genome. This approach enables the precise identification and modulation of pathogenic cell states across diverse diseases, facilitating the development of RNA-guided therapeutics with unprecedented precision, speed, and scalability.

Leadership Team

HAYA Therapeutics is led by a team of experts in lncRNA biology and precision therapeutics:

Samir Ounzain, PhD – Co-founder and Chief Executive Officer

Daniel Blessing, PhD – Co-founder and Chief Technology Officer

Richard Law, PhD – Chief Business Officer

Eric Adam, PhD – Chief Operating Officer

Mihaela Roberts – Senior Vice President, Finance

Jordan Shin, MD, PhD – Chief Medical Officer

James G. Karras, PhD – Vice President, Translation

Carsten Merkwirth, PhD – Senior Director, Biomarkers

Fanny Kling, MSc – Associate Director, Regulatory & Pre-Clinical

Marco Mina, PhD – Senior Director, Computational Biology & Data Science

Rudi Micheletti, PhD – Senior Director, Biology & Functional Genomics

Ana Silva, PhD – Director, Strategic Scientific Communication

Greg Smith, MSc – Senior Director, Human Resources

Leadership Changes

In February 2023, HAYA expanded its advisory boards with the addition of:

Ahmad Masri, MD, MS – Director of the Hypertrophic Cardiomyopathy Center at Oregon Health & Science University, joined the Clinical Advisory Board.

Cédric Feschotte, PhD – Professor in the Department of Molecular Biology and Genetics at Cornell University, joined the Scientific Advisory Board.

Competitor Profile

Market Insights and Dynamics

The biotechnology sector focusing on RNA-based therapeutics is experiencing significant growth, driven by advancements in understanding the regulatory genome and its role in disease pathology. The market for lncRNA-targeting therapies is emerging, with increasing interest from both biotech startups and established pharmaceutical companies.

Competitor Analysis

HAYA Therapeutics operates in a competitive landscape with several key players:

CAMP4 Therapeutics – Develops therapies targeting lncRNAs for various diseases, including urea cycle disorders.

Ionis Pharmaceuticals – Focuses on RNA-targeted therapies, including antisense oligonucleotides for conditions like Angelman syndrome.

NextRNA Therapeutics – Specializes in targeting non-coding RNAs for therapeutic applications.

These companies, along with HAYA, are pioneering the exploration of the "dark genome" to develop novel treatments for complex diseases.

Strategic Collaborations and Partnerships

HAYA has established significant collaborations to bolster its research and development efforts:

Innosuisse Funding (May 2022): Secured funding from the Swiss Innovation Agency to support research collaborations with the University of Bern, University Hospital of Bern, and Lausanne University Hospital, focusing on lncRNA therapies for cardiomyopathy and squamous cell carcinoma.

Eli Lilly Partnership: Entered into a collaboration with Eli Lilly and Company to focus on RNA-based drug targets for obesity and metabolic disorders.

Operational Insights

HAYA's strategic positioning in the biotechnology sector is strengthened by its focus on the regulatory genome and lncRNAs, setting it apart from competitors. The company's proprietary platform enables the development of precision therapeutics that reprogram disease-driving cell states, offering potential advantages in efficacy and safety over traditional treatments. HAYA's collaborations with leading academic institutions and pharmaceutical companies further enhance its capabilities and market reach.

Strategic Opportunities and Future Directions

Looking ahead, HAYA aims to:

Advance HTX-001 into Clinical Trials: Initiate and progress clinical trials for its lead candidate targeting nHCM.

Expand the Therapeutic Pipeline: Develop additional lncRNA-targeting candidates for various fibrotic diseases and other serious health conditions.

Enhance Platform Capabilities: Continue to refine and expand its proprietary platform to identify and modulate pathogenic cell states across a broader range of diseases.

By leveraging its current strengths and strategic partnerships, HAYA is well-positioned to make significant contributions to the field of precision medicine.