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A phase I clinical proof-of-concept evaluation of EPF-001 to demonstrate the efficacy and safety in humans

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Trial Profile

A phase I clinical proof-of-concept evaluation of EPF-001 to demonstrate the efficacy and safety in humans

Status: Planning
Phase of Trial: Phase I

Latest Information Update: 07 Apr 2026

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At a glance

  • Drugs EPF 001 (Primary)
  • Indications Beta-thalassaemia; Sickle cell anaemia
  • Focus Adverse reactions; Proof of concept
  • Sponsors EpiFrontier Therapeutics

Most Recent Events

  • 07 Apr 2026 New trial record
  • 25 Mar 2026 According to EpiFrontier Therapeutics media release, the company 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.

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