EditForce and Mitsubishi Tanabi Pharma to work on gene therapy for CNS

July 5, 2022 - 2 minutes
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EditForce, Inc. has entered into a license agreement with Mitsubishi Tanabe Pharma Corporation (MTPC) to research, develop and commercialize potential gene therapy products for a specific target disease related to the central nervous system (CNS) by utilizing EditForce’s pentatricopeptide repeat (PPR) protein platform technology.

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 more than 20 billion yen ($147.3 million), 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.”

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PPR 

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. 

Takahiro Nakamura and 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. 

It is possible to manipulate and modify the target genome and RNA both inside and outside the cell by fusion with effector proteins.

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