Funding will support clinical development of EPF-001, a first-in-class G9a inhibitor for sickle cell disease and beta thalassemia
Company built on pioneering research from RIKEN and Tokyo University of Pharmacy and Life Sciences, with backing from leading Japanese venture capital firm UTEC
PENNINGTON, N.J. and TOKYO, March 24, 2026 /PRNewswire/ — EpiFrontier Therapeutics, Inc., a biotechnology company developing transformative therapies for beta globin disorders, today announced its award of up to $32 million in non-dilutive funding from the Japan Agency for Medical Research and Development (AMED). The grant will advance the clinical development of EPF-001, a novel small molecule G9a inhibitor designed to increase fetal hemoglobin expression in patients with sickle cell disease and beta thalassemia.
EPF-001 represents the culmination of over a decade of pioneering drug discovery research originally conducted through a collaborative effort between RIKEN Program for Drug Discovery and Medical Technology Platforms (DMP) and Tokyo University of Pharmacy and Life Sciences. The compound emerged from a comprehensive screening campaign of approximately 140,000 compounds, followed by sophisticated molecular design using X-ray crystallographic data and the synthesis and evaluation of more than 1,000 compounds. This integrated approach leveraged RIKEN’s advanced drug discovery infrastructure to enable the identification of a highly specific G9a inhibitor with demonstrated ability to increase fetal hemoglobin expression in human cells and multiple preclinical animal models.
Dr. Minoru Yoshida, Executive Vice President, RIKEN stated: “At RIKEN, we are tackling challenges facing humanity by integrating the world-leading technological platforms possessed by each research center. One such challenge is the realization of groundbreaking drug discovery. The development of EPF-001 exemplifies RIKEN’s commitment to translating fundamental scientific discoveries into therapies that address critical unmet medical needs through such collaborative efforts. We are pleased to see this research transition from RIKEN to EpiFrontier Therapeutics for clinical development, and we are grateful for AMED’s continued support of this project—first through the Research Project for Intractable Diseases and now through the Strengthening Program for Pharmaceutical Startup Ecosystem. This partnership represents an important model for advancing Japanese innovation to benefit patients globally.”
“We are honored to build upon the groundbreaking research conducted by our scientific founders at RIKEN and Tokyo University of Pharmacy and Life Sciences,” said Bruce Goldsmith, Chief Executive Officer of EpiFrontier Therapeutics. “Their innovative approach to targeting G9a has yielded a clinical candidate with the potential to meaningfully improve outcomes for patients suffering from beta globin disorders. This AMED grant recognizes the potential of the underlying science and provides crucial support as we advance EPF-001 toward clinical proof-of-concept. We are committed to realizing the therapeutic promise of this molecule for patients worldwide.”
Beta globin disorders, including sickle cell disease and beta thalassemia, affect millions of patients globally and represent significant unmet medical needs. Current treatment options remain limited, and many patients continue to experience severe complications and reduced quality of life. EPF-001 has demonstrated significant advantages over standard treatments in preclinical studies in terms of both efficacy and safety, positioning it as a potentially transformative therapy for these patient populations.
The AMED funding will support EPF-001’s advancement through Phase 2 clinical development, with trials planned to be conducted primarily at overseas medical institutions with large patient populations. Patient enrollment in Japan is specifically planned for severe beta thalassemia. The funding will enable comprehensive clinical evaluation to demonstrate the drug’s efficacy and safety in humans, supporting the company’s goal of achieving key commercialization milestones.
EpiFrontier Therapeutics was founded in July 2025 as a U.S.-based corporation with a Japanese subsidiary (EpiFrontier Therapeutics G.K.), following extensive business feasibility discussions with The University of Tokyo Edge Capital Partners (UTEC), which began in spring 2024. The company has secured an exclusive license to the core intellectual property arising from research conducted at RIKEN and Tokyo University of Pharmacy and Life Sciences and will pursue additional patent applications covering formulation, dosage and administration, and combination therapies to establish a comprehensive intellectual property portfolio.
On behalf of existing investors, Azusa Shiohara, Principal at UTEC and Board Member of EpiFrontier Therapeutics, stated: “EpiFrontier exemplifies the powerful combination of world-class Japanese science and global drug development expertise. The pioneering work by RIKEN and Tokyo University of Pharmacy and Life Sciences has created a truly differentiated clinical candidate with the potential to transform treatment for patients with beta globin disorders. We are proud to support this company as it brings this important Japanese innovation to patients worldwide, and we are grateful for AMED’s significant investment in validating and advancing this breakthrough therapy.”
About EPF-001
EPF-001 (RK-701) is a first-in-class, highly selective small molecule inhibitor of the histone methyltransferase G9a, designed to increase fetal hemoglobin expression in patients with beta globin disorders. The compound has demonstrated robust pharmacological activity in human cells and various animal models, with a favorable safety and pharmacokinetic profile. EPF-001 is being developed for the treatment of sickle cell disease and beta thalassemia, with initial clinical development targeting severe beta thalassemia patients in Japan and broader beta globin disorder populations internationally.
About EpiFrontier Therapeutics
EpiFrontier Therapeutics, Inc. is a biotechnology company dedicated to developing novel therapies for beta globin disorders. Headquartered in the United States with operations in Japan, EpiFrontier is built on pioneering research from RIKEN Program for Drug Discovery and Medical Technology Platforms and Tokyo University of Pharmacy and Life Sciences. The company’s lead program, EPF-001, represents a first-in-class approach to treating sickle cell disease and beta thalassemia through selective G9a inhibition. EpiFrontier leverages an international network of research institutions, contract research organizations, and clinical development partners to advance breakthrough therapies for patients with significant unmet medical needs.
For more information, visit [www.epifrontiertx.com]
About AMED
The Japan Agency for Medical Research and Development (AMED), established in 2015, is a government agency dedicated to accelerating medical innovation and strengthening Japan’s global competitiveness in healthcare. AMED supports integrated research and development across the medical spectrum, from basic science to clinical applications, to translate cutting-edge discoveries into real-world treatments. As part of this mission, AMED launched the Strengthening Program for Pharmaceutical Startup Ecosystem to foster the growth of Japan’s biotech sector. This program provides substantial non-dilutive funding to startups developing innovative therapies, contingent upon investment from pre-certified venture capital firms. By supporting early-stage pharmaceutical development, including non-clinical and clinical studies, the program aims to accelerate breakthrough medical innovations and position Japan as a leader in biotech entrepreneurship.
For more information, visit https://www.amed.go.jp/en/
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