Announcement of Paper Publication: EditForce Demonstrates Efficacy of Treatment for Myotonic Dystrophy Type 1 Using PPR Platform Technology

FUKUOKA, Japan, April 25, 2025 /PRNewswire/ -- EditForce, Inc. (Headquarters: Fukuoka, Japan) is pleased to announce that a research paper on the results of a joint study with the research group led by Professor Masayuki Nakamori of the Department of Neurology of the Yamaguchi University Graduate School of Medicine, and Professor Hideki Mochizuki of the Department of Neurology of the University of Osaka Graduate School of Medicine, was published in Science Translational Medicine on April 16, 2025 (Eastern Daylight Time).

In this study, it was demonstrated that a single administration of the jointly developed PPR protein (CUG-PPR1), which specifically binds to the abnormal RNA causing Myotonic Dystrophy Type 1 (hereinafter referred to as "the Disease"), resulted in long-term amelioration of muscle symptoms in mice, with minimal immune response and side effects.

The results of this study open the way for the development of treatments for the Disease, which currently has no fundamental cure, and are evaluated as demonstrating the innovativeness and efficacy of EditForce's proprietary PPR platform technology. It will continue to strive in its R&D activities to deliver the treatment for the Disease to patients as quickly as possible.

Paper Information

- Journal: Science Translational Medicine

- Publication Date: Wednesday, April 16, 2025, 2:00 PM (Eastern Daylight Time)

- Title: Pentatricopeptide repeat protein targeting CUG repeat RNA ameliorates RNA toxicity in a myotonic dystrophy type 1 mouse model

- Authors: Takayoshi Imai1*, Maiko Miyai2*, Joe Nemoto3, Takayuki Tamai1, Masaru Ohta1, Yusuke Yagi1, Osamu Nakanishi1, Hideki Mochizuki2, and Masayuki Nakamori2,3**

Affiliations:

1. EditForce, Inc.

2. Graduate School of Medicine, University of Osaka, Department of Neurology

3. Graduate School of Medicine, Yamaguchi University, Clinical Neurology

*: These authors contributed equally to this work.

**: Corresponding author

DOI: 10.1126/scitranslmed.adq2005
https://www.science.org/doi/10.1126/scitranslmed.adq2005