Approval to Advance to Stage 2 of AMED's "Strengthening Program for Pharmaceutical Startup Ecosystem"
We are pleased to announce that the project titled "Development of an Innovative Treatment for Myotonic Dystrophy Type 1 by a Sequence-Specific RNA Binding Protein Targeting Pathogenic CUG repeat RNA" ("EF-210") - which was adopted by the Japan Agency for Medical Research and Development ("AMED") as a "Strengthening Program for Pharmaceutical Startup Ecosystem" ("the Program") in 2023 - has successfully passed its interim evaluation (stage-gate review) and received official approval to advance to Stage 2.
We view this outcome as recognition of the progress made in developing an innovative, RNA-targeting therapy to address an intractable genetic disease for which no effective treatment currently exists. Moving forward into Stage 2, we are fully committed to advancing the Investigational New Drug (IND) application process and working toward the early initiation of clinical trials in North America.
Please refer to the following for details regarding the evaluation results.
URL: https://www.amed.go.jp/program/list/19/02/005_r8_chukan_2.html