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Exousia Bio

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

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.