法人別リリース

Announcement of Paper Publication: EditForce Demonstrates Efficacy of Treatment for Myotonic...

EditForce — Fri, 25 Apr 2025 15:00:00 +0900

FUKUOKA, Japan, Apr. 25, 2025 /Kyodo JBN/ --
EditForce, Inc.
Announcement of Paper Publication: EditForce Demonstrates Efficacy of Treatment for Myotonic Dystrophy Type 1 Using PPR Platform Technology

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

EditForce Realizes RNA-editing Technology of "U-to-C"

EditForce — Tue, 18 Oct 2022 15:00:00 +0900

FUKUOKA, Japan, Oct. 18, 2022 /Kyodo JBN/ --
EditForce, Inc.
EditForce, Inc. (hereinafter "EditForce"), headquartered in Fukuoka, Japan, has realized the world’s first RNA-editing technology that enables RNA-editing bases to be changed from "U" to "C," and demonstrated that this technology works even in human cells in a joint study with Professor Takahiro Nakamura, Faculty of Agriculture, Kyushu University. The study opens up the possibility of editing gene mutations which could not have been the target with the existing technologies, and is expected to contribute to the R&D of therapies for various genetic diseases. EditForce will improve safety and editing efficiency in its development activities to establish innovative gene therapy technologies. The study was published in the scientific journal "Communications Biology" on September 15 (September 16 in JST), 2022.

Logo: https://kyodonewsprwire.jp/prwfile/release/M107232/202210117969/_prw_PI1fl_AXiGsGJ1.jpg

Outline of research
A genome existing in each human cell consists of four nucleotide bases -- A, C, G, and T -- of which the sequence holds the information necessary to make up a human body. Cells generate RNA consisting of four nucleotide bases -- A, C, G, and U -- based on genomic sequences, and then produce protein according to the sequences of RNA. However, changes in a single nucleotide on a genome or RNA sequence cause a variety of diseases. Treatment of these diseases requires single base-editing technology of repairing genomic or RNA mutations to bring the sequences back on the normal track.

Currently, genome-editing technology has been developed rapidly, but the development of editing technology for RNA sequences remains limited. Especially as regards a single base substitution, technologies have been established to substitute "C" with "U" and "A" with "G," but the substitution of other bases is yet to be realized.

The study clarified the mechanism of RNA editing in plants to substitute "U" with "C," based on which EditForce has realized the world's first RNA-editing (base substitution) technology that substitutes "U" with "C," and the company demonstrated that this technology works in human cells.

Base-editing technologies can be applied to the treatment of diseases caused by a single mutation, and "U-to-C" RNA-editing technology of the study opens up the possibility to edit mutations which could not have been the target with the existing technologies. Further, repairing mutations by RNA editing without changes in genome sequence will enable EditForce to provide safer treatments to patients. The company expects that this technology will establish a novel gene therapy by improving safety and editing efficiency in its development activities.

This work was supported by the Research Grant for Young Scholars funded by Yamanashi Prefecture.

Article information
Ichinose M., Kawabata M., Akaiwa Y., Shimajiri Y., Nakamura I., Tamai T., Nakamura T., Yagi Y. and Gutmann B. "U-to-C RNA editing by synthetic PPR-DYW proteins in bacteria and human culture cells" Commun Biol. 5, 968 (2022).

About EditForce, Inc.
EditForce, Inc., a Kyushu University-originated venture company developing a unique DNA/RNA-editing technology (PPR platform technology (*)), was established in May 2015 by KISCO Ltd. and Prof. Takahiro Nakamura of Kyushu University (former president of EditForce and currently its scientific advisor) and funded by companies and funds that have track records of investing in life sciences and biotechnology. EditForce aims for drug discovery that applies the PPR technology through joint research with universities and private companies.

For more information, please visit: https://www.editforce.co.jp/

(*) Pentatricopeptide repeat (PPR) protein platform technology
PPR is a protein discovered in plants that regulates gene expression by binding to DNA and RNA in a sequence-specific manner. The PPR proteins are also found in humans and yeasts, and they have similar functions. Prof. Takahiro Nakamura and Dr. Yusuke Yagi, CTO of EditForce, have focused on the PPR proteins and elucidated the mechanism that determines sequence specificity, and established a technology for creating various PPR proteins, each of which binds to a specific target DNA or RNA sequence. Furthermore, it is possible to manipulate and modify the target genome and RNA both inside and outside the cell by fusion with effector proteins.

EditForce and Mitsubishi Tanabe Pharma Enter into License Agreement

EditForce — Tue, 05 Jul 2022 16:00:00 +0900

FUKUOKA, Japan, July 5, 2022 /Kyodo JBN/ --
EditForce, Inc.
EditForce and Mitsubishi Tanabe Pharma Enter into License Agreement

EditForce, Inc. (Headquarters: Fukuoka, President and CEO: Takashi Ono; hereinafter "EditForce") has entered into a License Agreement (hereinafter "Agreement") with Mitsubishi Tanabe Pharma Corporation (Headquarters: Osaka, Representative Director: Hiroaki Ueno; hereinafter "MTPC") to research, develop and commercialize potential gene therapy products for a specific target disease in the field of CNS by utilizing EditForce's proprietary PPR protein platform technology.

Logo of EditForce, Inc.: https://kyodonewsprwire.jp/prwfile/release/M107232/202206242954/_prw_PI1fl_09s1955H.jpg

In this alliance, MTPC and EditForce aim to create potential novel pharmaceuticals for the specific CNS disease by utilizing the drug R&D know-how and global business experience of MTPC and the novel biotechnology of EditForce. MTPC will acquire the exclusive right to conduct the selection of drug candidate molecules, preclinical and clinical development, manufacturing, and commercialization worldwide.

Under the terms of the Agreement, EditForce will receive an upfront payment and milestone payments amounting to over 20 billion yen depending on the development stage and commercialization progress, and royalties based on worldwide sales after the launch.

"I am so delighted to reach the agreement with MTPC, which has an interest in our proprietary PPR protein platform technology," said Takashi Ono, President and CEO of EditForce. "We look forward to working closely with MTPC to develop and deliver breakthrough pharmaceutical products with our technology to patients suffering from diseases."

EditForce will proceed with R&D of pharmaceuticals using its PPR protein platform technology.

About EditForce, Inc.
EditForce, Inc., a Kyushu University-originated venture company developing a unique DNA/RNA editing technology (PPR platform technology (*)), was established in May 2015 by KISCO Ltd. and Prof. Takahiro Nakamura of Kyushu University (former president of EditForce and currently its scientific advisor) and funded by companies and funds that have track records of investing in life sciences and biotechnology. EditForce aims for drug discovery that applies the PPR technology through joint research with universities and private companies.

For more information, please visit https://www.editforce.co.jp/

(*) Pentatricopeptide repeat (PPR) protein platform technology
PPR is a protein discovered in plants that regulates gene expression by binding to DNA and RNA in a sequence-specific manner. The PPR proteins are also found in humans and yeasts, and they have similar functions. Prof. Takahiro Nakamura and Dr. Yusuke Yagi, CTO of EditForce, have focused on the PPR proteins and elucidated the mechanism that determines sequence specificity, and established a technology for creating various PPR proteins, each of which binds to a specific target DNA or RNA sequence. Furthermore, it is possible to manipulate and modify the target genome and RNA both inside and outside the cell by fusion with effector proteins.

About Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma Corporation (MTPC), the pharma arm of Mitsubishi Chemical Group (MCG), is one of the oldest pharmaceutical companies in the world, founded in 1678, and focusing on ethical pharmaceuticals. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan’s pharmaceutical industry. MCG has positioned health care as its strategic focus in its management policy, "Forging the future." MTPC sets the MISSION of "Creating hope for all facing illness." To that end, MTPC is prioritizing work on "precision medicine" to provide drugs with high treatment satisfaction by identifying patient populations with high potential for efficacy and safety, focusing on the disease areas of the central nervous system and immuno-inflammation. In addition, MTPC is working to develop "around the pill solutions" to address specific patient concerns based on therapeutic medicine, including prevention of diseases, pre-symptomatic disease care, prevention of aggravation and prognosis.

For more information, go to https://www.mt-pharma.co.jp/e/