Orphan drug pipeline
EXO-NG01
NANOG-targeted shRNA exosome therapy
An engineered exosome-based therapeutic candidate designed to deliver shRNA targeting NANOG mRNA to glioblastoma cells. By downregulating NANOG expression, EXO-NG01 targets cancer stemness and treatment resistance, with the goal of enhancing glioblastoma sensitivity to temozolomide (TMZ) and reducing glioblastoma stem cell viability.
Indication
Malignant glioma / GBM
Modality
Engineered exosome
Target
NANOG mRNA
Stage
Preclinical
Designations
FDA ODD · EMA COMP
Mechanism of action
Step
Title
Body
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.
Key preclinical results
- Reduced glioblastoma stem cell viability.
- Enhanced sensitivity to temozolomide.
- Significant reduction in tumor burden with combination therapy.
- Marked reduction in tumor burden observed in the combination-treatment group in a preclinical mouse model.
Regulatory milestones
United States
FDA Orphan Drug Designation
Granted for shRNA targeting NANOG mRNA for the treatment of malignant glioma. Orphan Drug Designation does not constitute FDA approval.
European Union
EMA COMP positive opinion
Positive opinion from the European Medicines Agency’s Committee for Orphan Medicinal Products.