EpiFrontier Therapeutics, Inc., a Pennington-based biotechnology company dedicated to developing novel therapies for beta globin disorders, recently announced it was awarded up to $32 million in non-dilutive funding from the Japan Agency for Medical Research and Development (AMED). The grant is earmarked to accelerate the clinical development of EPF-001, a first-in-class therapy targeting sickle cell disease and beta thalassemia.
The funding arrives via AMED’s “Strengthening Program for Pharmaceutical Startup Ecosystem,” signaling strong institutional confidence in EpiFrontier’s epigenetic approach to treating chronic blood disorders.
EPF-001 is the result of over ten years of collaborative research between RIKEN and the Tokyo University of Pharmacy and Life Sciences. The drug’s journey to clinical trials involved:
- Massive Screening: Testing of approximately 140,000 compounds.
- Precision Design: Utilizing X-ray crystallographic data to refine the molecule.
- Extensive Synthesis: Evaluating over 1,000 synthesized variations to identify the most effective G9a inhibitor.
Patients with sickle cell disease and beta thalassemia suffer from defects in adult hemoglobin. EPF-001 functions as a G9a inhibitor, a small molecule designed to “switch on” the production of fetal hemoglobin. By increasing fetal hemoglobin levels, the therapy aims to compensate for defective adult hemoglobin, potentially reducing severe complications and improving quality of life.
“The development of EPF-001 exemplifies RIKEN’s commitment to translating fundamental scientific discoveries into therapies that address critical unmet medical needs,” Dr. Minoru Yoshida, executive vice president at RIKEN said.
The $32 million grant will support EPF-001 through Phase 2 clinical development.
Founded in July 2025, EpiFrontier Therapeutics bridges Japanese scientific discovery with global drug development expertise. The company is backed by The University of Tokyo Edge Capital Partners (UTEC) and maintains operations in both the U.S. and Japan.
“This AMED grant recognizes the potential of the underlying science and provides crucial support as we advance EPF-001 toward clinical proof-of-concept,” Bruce Goldsmith, CEO of EpiFrontier Therapeutics said.
