Lead Development Program: EXO-NG01

EXO-NG01 is an engineered exosome-based therapeutic candidate designed to deliver shRNA targeting NANOG mRNA to glioblastoma cells. The objective is to reduce NANOG expression, targeting a transcription factor associated with cancer stemness and treatment resistance.By downregulating NANOG, EXO-NG01 is designed to initiate a targeted approach against biological mechanisms that may help glioblastoma cancer stem cells maintain their aggressive and therapy-resistant characteristics.

NANOG is associated with stem-like characteristics in glioblastoma cancer stem cells, including properties linked to self-renewal and resistance to conventional treatment. Reducing NANOG expression is intended to suppress cancer stemness and molecular mechanisms associated with treatment resistance. This approach targets the underlying cancer stem cell population that may contribute to tumor persistence and resistance to therapy.

As cancer stemness and resistance mechanisms are suppressed, the viability of glioblastoma cancer stem cells may be reduced.Exousia Bio's preclinical program has reported reduced glioblastoma stem cell viability following its NANOG-targeted therapeutic approach. Targeting this treatment-resistant cell population is intended to address an important biological contributor to tumor persistence and recurrence.

By targeting NANOG-associated cancer stemness and treatment resistance, EXO-NG01 is designed to enhance the sensitivity of glioblastoma cells to temozolomide (TMZ), a standard chemotherapy used in glioblastoma treatment. This combination approach is designed to complement established therapy rather than replace it. In Exousia Bio's preclinical program, enhanced sensitivity to temozolomide and a significant reduction in tumor burden were reported with combination treatment.