EXP05: MSC-EV Therapy for POI/DOR and Gynecologic Tissue Repair

EXORPHIA is a biotechnology company with an integrated platform for extracellular vesicle (EV) manufacturing, purification, and quality control. Next-generation IVF technologies are our core business in assisted reproductive technology, while therapeutic assets are advanced through external partnering, co-development, and licensing.

EXP05 is an umbilical cord-derived MSC-EV therapeutic candidate for premature ovarian insufficiency (POI) and diminished ovarian reserve (DOR). It is designed as a non-hormonal, cell-free tissue-repair approach that aims to restore the ovarian microenvironment affected by inflammation, fibrosis, and tissue injury. Rather than replacing hormones or supporting ART procedures alone, EXP05 seeks to address ovarian tissue dysfunction upstream of oocyte retrieval.

In a chemotherapy-induced POI mouse model, MSC-EV administration significantly increased primary follicle counts and showed a favorable litter-size signal, supporting further evaluation of ovarian function recovery. The current in vivo proof-of-concept was obtained in the POI model; DOR is included in the proposed development scope based on the shared ovarian-function and microenvironment rationale.

Umbilical cord-derived MSC-EVs were selected based on comparative omics signals related to reproductive biology. The program is supported by EXORPHIA’s integrated upstream, downstream, and quality-control platform, including potency-linked QC concepts and CDMO-supported GMP/CMC readiness activities.

POI and DOR are the initial focus. Potential expansion into gynecologic tissue dysfunction, including endometrial atrophy, intrauterine adhesions/Asherman’s syndrome, endometriosis, and adenomyosis, will be prioritized with partners based on strategic fit, disease-model availability, and development feasibility. These expansion indications remain development hypotheses and do not yet have established proof-of-concept.

Related EXP02 ex vivo embryo-culture findings are presented only as supportive evidence for the biological activity of the related umbilical cord-derived EV platform. EXP02 itself is outside the scope of this partnering opportunity.

A staged development pathway may include investigator-initiated clinical research in Japan after GMP clinical-batch manufacturing and release/QC, providing an early decision point for option exercise or licensing.

We seek pharmaceutical or business partners for early option-based partnering, co-development, or staged licensing of EXP05. EXORPHIA would lead nonclinical data-package generation, GMP/CMC readiness, and QC/potency development. Following option exercise or licensing, the partner would lead IND-enabling studies, regulatory development, company-sponsored clinical development, and commercialization.