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

FUKUOKA,Japan,Oct. 18,2022 --EditForce,Inc. (hereinafter "EditForce"),headquartered in Fukuoka,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 possibilityof editing gene mutations which could not have been the target with theexisting technologies,and is expected to contribute to the R&D of therapiesfor 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 "CommunicationsBiology" on September 15 (September 16 in JST),2022.

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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 ahuman body. Cells generate RNA consisting of four nucleotide bases -- A,and U -- based on genomic sequences,and then produce protein according to thesequences of RNA. However,changes in a single nucleotide on a genome or RNA sequence cause a variety of diseases. Treatment of these diseases requiressingle base-editing technology of repairing genomic or RNA mutations to bringthe sequences back on the normal track.

Currently,genome-editing technology has been developed rapidly,but thedevelopment of editing technology for RNA sequences remains limited. Especiallyas regards a single base substitution,technologies have been established tosubstitute "C" with "U" and "A" with "G," but the substitution of other basesis 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 bya single mutation,and "U-to-C" RNA-editing technology of the study opens upthe possibility to edit mutations which could not have been the target with theexisting technologies. Further,repairing mutations by RNA editing withoutchanges in genome sequence will enable EditForce to provide safer treatments topatients. The company expects that this technology will establish a novel genetherapy by improving safety and editing efficiency in its developmentactivities.

This work was supported by the Research Grant for Young Scholars funded byYamanashi 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-DYWproteins 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 (*)),wasestablished in May 2015 by KISCO Ltd. and Prof. Takahiro Nakamura of KyushuUniversity (former president of EditForce and currently its scientific advisor)and funded by companies and funds that have track records of investing in lifesciences and biotechnology. EditForce aims for drug discovery that applies thePPR 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 bindingto DNA and RNA in a sequence-specific manner. The PPR proteins are also foundin humans and yeasts,and they have similar functions. Prof. Takahiro Nakamuraand Dr. Yusuke Yagi,CTO of EditForce,have focused on the PPR proteins andelucidated the mechanism that determines sequence specificity,and establisheda technology for creating various PPR proteins,each of which binds to aspecific target DNA or RNA sequence. Furthermore,it is possible to manipulateand modify the target genome and RNA both inside and outside the cell by fusionwith effector proteins.

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