List of Gene Therapy Companies in Japan - 11

aceRNA Technologies Co., Ltd. is a biotech startup established in 2018 based on RNA design technology developed in synthetic RNA biology research by Prof. Hirohide Saito at Kyoto University. We are developing "smart gene therapy” (mRNA-based and virus-base) that enables control of therapeutic transgene expression in cell-type and -state specific manner. By that, we are committed to develop new therapeutic approach, such as in vivo cell programming.

AnGes was founded In December 1999, with the aim of developing hepatocyte growth factor (HGF) gene therapy drugs which can help regenerate blood vessels. Originating from the word angiogenesis meaning the formation of new blood vessels in English, our corporate name AnGes encapsulates our desire to be an angel (ange in French) offering new drugs for patients with hard-to-treat and rare diseases. Both our founder and our employees are still motivated by this desire.

Celaid Therapeutics is a Japan-based biotechnology company, which has developed a platform technology to efficiently expand hematopoietic stem cells ex vivo without choosing its origins, including bone marrow, cord blood, or peripheral blood. By using the novel technology, we are developing a cell therapy product that brings HSC transplantation to the next level and helps patients with blood disorders. Our technology has many potential applications including gene/cell therapy that utilize gene editing on HSC. Our mission is to improve QOL of patients, their families, and HSC donors all over the world.

Gene Therapy Research Institution Co., Ltd. is headquartered in Japan. Based on extensive research, the company is committed to providing safe and effective gene therapies for refractory diseases for which effective therapies have not yet been established. Focusing on Parkinson's disease, amyotrophic lateral sclerosis (ALS) and Alzheimer's disease, the company strives to find safer viral vectors and more effective genetic technologies to bring new treatment options to patients.

Our founding discoveries came from the labs of Prof. Takagi and Prof Suga (the 2023 winner of the Wolf Prize in Chemistry), both of whom are deeply involved in our R&D to this day. Their innovations enable a novel class of biologics, called “Neobiologics,” that break existing boundaries in biological drug development and will ultimately better, more cost-effective treatments for patients. Internally and with our partners, we are advancing a robust portfolio of products: 1. cMet agonist to HGF (hepatocyte growth factor) to tackle NASH (non-alcoholic steatohepatitis)/fibrosis; currently in IND enabling studies. 2. TrkB agonist of BDNF (brain-derived neurotrophic factor) to tackle neurodegenerative diseases like Alzheimer's Disease and Parkinson’s Disease; currently in vivo studies. 3. Immuno-oncology bi-specific with T-cell engaging activity and a tumor antigen-specific binder; currently in vitro R&D. 4. Undisclosed assets in various stages. This pipeline builds off our core RaPID System and LassoGraft Technology® (LGT) platforms. The RaPID System selects highly active cyclic peptides that tightly bind to drug-target proteins from our diverse library. These peptides are then genetically grafted onto scaffold proteins using LGT to create Neobiologics. Neobiologics break multiple current boundaries for biologics, such as access across the blood-brain barrier, 3D topological design control, multifunctionality (bi-specific, tri-specific, etc.), wide applicability from AAV capsid modulation to antibodies to therapeutic proteins and superior development speeds that drop development costs significantly. Selected publications 1. Mihara, E et al. Lasso-grafting of macrocyclic peptide pharmacophores yields multi-functional proteins. Nature Communications (2021)2:1543 2. Sakai, K et al. Designing receptor agonists with enhanced pharmacokinetics by grafting macrocyclic peptides into fragment crystallizable regions. Nature Biomedical Engineering 7, 164-176 (2023)

Momotaro-Gene is a biotech start-up company from Okayama University whose main technology lies in tumour suppressor gene. They are focused on the development of a next-generation cancer drug.

Development of novel cancer immunotherapies mainly using CAR T cells

Creation of optimal nucleic acid medicine for disease treatment with proprietary technology PURMX's solution is a drug discovery seed of small RNA nucleic acid drugs that fine-tune many target genes and deliver unprecedented therapeutic effects. PURMX already possesses a large amount of functional RNA screening data, which is being further expanded. The first stage of innovative nucleic acid medicine seeds is MIRX002. MIRX002 is a nucleic acid medicine for malignant pleural mesothelioma. We have completed non-clinical trials and are conducting investigator-initiated clinical trials at Hiroshima University Hospital starting in 2021.

Takara Bio Inc. is committed to improving the human condition through biotechnology. Starting as the biomedical business of Takara Shuzo, a fermentation and beverage company founded in 1925 (now Takara Holdings Company, Inc.), Takara Bio has focused on developing research tools for the life sciences, cell and gene therapy technologies, and nutraceuticals since its launch in 1967. The three business units of TAKARA BIO INC are: • Genetic Engineering Research - manufactures and sells research reagents and scientific instruments used by biotechnology researchers worldwide, as well as contract research services. • Agribio - produces and sells health food products whose functionality have been proven by biotechnology, and conducts a mushroom business based on technologies for large-scale mushroom production. • Gene Medicine - conducts clinical development projects to work toward commercializing cell and gene therapies centered on a highly efficient gene transduction method and a lymphocyte expansion-culture system, both using RetroNectin® reagent.