EditForce

EditForce Inc. is a biotechnology firm established in May 2015, specializing in gene therapy development. The company originated from research conducted at Kyushu University by Professor Takahiro Nakamura, who co-founded the firm with KISCO LTD. Dr. Nakamura's work on pentatricopeptide repeat (PPR) proteins, initially discovered in plants, forms the foundation of the company's proprietary technology. Headquartered in Fukuoka, Japan, with a Tokyo office and a laboratory at Kyushu University, EditForce is strategically positioned within Japan's academic and commercial biotechnology ecosystem.

The core of EditForce's business is its PPR platform technology, a proprietary tool for gene and RNA editing. This technology is distinguished by its ability to target and bind to specific DNA and RNA sequences, offering a versatile approach to genetic manipulation. A key feature is the ability to perform single nucleotide substitutions, such as changing cytosine to uridine (C to U) or vice versa, using proprietary enzymes. This allows for the precise repair of RNA mutations that were previously difficult to target. The platform's modularity, which combines an RNA-binding PPR domain with an effector domain, enables various types of RNA control beyond simple substitutions, including the removal of RNA mutations through exon skipping. This positions the technology as a potential new modality for treating a range of diseases.

EditForce's business model centers on the discovery and development of novel drugs, primarily through joint research and development agreements and licensing its PPR platform technology to pharmaceutical companies. The company targets therapies for central nervous system (CNS), muscular, and rare diseases. A notable milestone is a license agreement with Mitsubishi Tanabe Pharma Corporation for the research, development, and commercialization of drugs for a specific CNS disease. The company has also entered into joint R&D agreements with firms like Solasia Pharma K.K. Its primary clients are pharmaceutical and biotechnology companies seeking advanced gene therapy solutions. EditForce is developing its PPR platform for delivery via various systems, including viral vectors like adeno-associated viruses (AAV). One of its lead programs, EF-210, is being developed to treat myotonic dystrophy type 1.

Keywords: gene therapy, RNA editing, DNA editing, PPR platform, pentatricopeptide repeat proteins, drug discovery, biotechnology, Kyushu University, Takahiro Nakamura, nucleic acid manipulation, CNS diseases, muscular diseases, rare diseases, nucleotide substitution, myotonic dystrophy, AAV delivery, pharmaceutical partnerships, genetic disorders, therapeutic development, gene control