Development Stages by Application

As of April 2024, we are developing four assets for various applications. The candidates are EXP01, EXP02, and EXP04 (for pets), native Prime-EV^® products with enhanced medicinal effects of Mesenchymal Stem Cells, and EXP03, a modified Smart-EV™ with exogenous antigen and nucleic acid.

*Please click here to see why we target each disease.

① Application 1 of EXP01; treatment of lung disorders (IPF, ARDS)

EXP01 is a bone marrow mesenchymal stem cell (MSC)-derived EV formulation. It is expected to be applied to a wide range of disease areas, mainly inflammatory diseases and fibrosis, as it is characterized by its low risk of side effects while having excellent anti-inflammatory and anti-fibrosis effects based on our proprietary cell activation technology. We are conducting research and development of EXP01 as a therapeutic agent for lung disorders (idiopathic pulmonary fibrosis (IPF) and acute respiratory distress syndrome (ARDS)). To date, we have completed a data package of non-clinical pharmacology, pharmacokinetics, and safety studies, and are preparing for clinical trials in the U.S. in 2025.
In addition, with a view to conducting clinical trials in Japan in the future and expanding the indication to other respiratory diseases, we have started a joint research project with Professor KAGE Hidenori of the University of Tokyo Graduate School of Medicine from 2024.

② Application 2 of EXP01; Chronic Kidney Disease (CKD) treatment

CKD is a disease that is accompanied by epithelial cell damage and tissue changes mainly fibrosis, and EXP01 is considered to be a promising target for CKD because of its ability to inhibit epithelial cell death and fibrosis mediated by fibroblasts. Therefore, in 2024, we started a joint research with Assistant Professor Yutaro Mori of the Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University, who developed the tubular epithelial cell organoid derived from CKD patients, an evaluation system with high predictive value for clinical drug efficacy. Using the organoids derived from CKD patients constructed by Assistant Professor Mori, we will conduct an efficient and clinically predictive efficacy evaluation.

③ Applications of EXP02; Reproductive field, infertility treatment (Premature ovarian dysfunction (POF)) therapeutic agent.

EXP02 is an EV preparation derived from umbilical cord-derived MSCs using our proprietary process. It contains an abundance of molecules that are important for follicle and embryo development.
When EXP02 was administered to a mouse model in which ovarian function was impaired by anti-cancer drugs, a certain improvement in function was observed, and exploratory research is underway with a view to developing EXP02 as a therapeutic agent for POF.
In the development of these products, we collaborate with Associate Professor Tokiko Nagamura of the University of Tokyo Institute of Medical Science Hospital and Associate Professor Keiichi Kumazawa of the University of Tokyo Graduate School of Medicine.

④ Usage of EXP03; therapeutic agent for autoimmune diseases

EXP03 is the first candidate for "Smart-EV™," a modified EV that aims to control immunity at will through the design of antigens and nucleic acids to be loaded on the EV. In collaboration with Professor Ken J. Ishii of the University of Tokyo Institute of Medical Science and Associate Professor Ryosuke Kojima of the University of Tokyo Graduate School of Medicine, prototype production and evaluation are underway with specific autoimmune diseases as the target.

⑤ Application of EXP04: Therapeutics for pet (cat and dog) diseases

EXP04 is an MSC-derived EV formulation that is being evaluated as a drug candidate for pets. Based on a co-development agreement with Kyoritsu Holdings Corporation and Kyoritsu Seiyaku Corporation, we have begun preclinical evaluation of EXP04 for the treatment of chronic kidney disease, heart disease, and enteritis, which are high medical needs in cats and dogs. In the future, we are considering submitting an application for manufacturing and marketing approval as a veterinary drug.