EXORPHIA, INC.

EXORPHIA is a Japan-based EV drug discovery company aiming to translate extracellular vesicle (EV)-based next-generation therapeutics into practical medical applications. By leveraging the multi-mechanistic bioactivity, biocompatibility, and delivery potential of EVs, we are developing novel therapeutic and DDS platforms for diseases that remain difficult to address with existing modalities.

Our technology platform combines Prime EV technology, which is designed to enhance EV functionality and reproducibility through specific cellular stimulation; Engineered EV technology, which is based on the loading of antigens and protein-based cargos and is being explored for future expansion into nucleic-acid loading; and a CMC/QC foundation designed to support pharmaceutical development. Together, these technologies enable EV drug development with a strong focus on quality, reproducibility, and pharmacological evaluation. They are supported by our patent portfolio and accumulated quality and pharmacology data.

Our key programs are organized around the following three pillars:

(1) EXP01: Naïve EV therapeutic candidate for atopic dermatitis, ARDS, and IPF

EXP01 is a naïve MSC-derived EV therapeutic candidate designed to leverage anti-inflammatory, anti-fibrotic, and tissue-repair activities. In atopic dermatitis, EXP01 is positioned as a potential first-in-class candidate characterized by improvement and restoration of skin barrier function, with development potential both as a topical maintenance therapy and as a systemic treatment via subcutaneous administration. In lung injury and fibrosis, we are advancing EXP01 for IPF/ARDS with a nonclinical-stage license-out strategy.

(2) EXP03 / Engineered EV Platform: Engineered EV platform for multivalent vaccines, immune modulation, and DDS applications

Our Engineered EV platform is based on technology for loading multiple antigens and protein-based cargos onto or into EV particles. Building on nonclinical PoC in multivalent EV vaccines for infectious diseases, we are exploring broader applications including cancer immunotherapy, antigen-specific immune modulation for autoimmune diseases, protein delivery, and CNS-DDS. Nucleic-acid loading is positioned as a future expansion theme for further technical validation and potential joint evaluation. We are seeking an exclusive license partner for the Engineered EV Platform and its application to selected therapeutic areas.

(3) EXP02: IVF additive and potential expansion into POI / gynecologic diseases

EXP02 is being developed as an IVF additive designed to improve embryo development and good-quality blastocyst rates. Based on this starting point, we are also exploring its potential expansion into a non-hormonal, cell-free tissue-repair approach for POI and gynecologic tissue dysfunction. This program will be introduced selectively depending on the target company’s areas of interest.

Through these programs, EXORPHIA aims to establish EVs not only as individual therapeutic candidates but also as a versatile drug discovery and DDS platform, creating new treatment options for intractable diseases, immune disorders, preventive medicine, and regenerative / tissue-repair applications.