NTT Biopharma PIPELINE

Platform Technologies

Brain Delivery Ocular Delivery (Eyedrop Nanoformulation) Nucleic Acid Delivery Oral Peptide Delivery Active Targeting Nanoparticle

Brain Disease

NTT-001 DNA Plasmid (IV Injection for PD)

Cancer Therapy

NTT-002

Ocular Disease

NTT-003 (Eyedrop Formulation for Wet-AMD) ASO (Eyedrop Formulation for Wet-AMD) Therapeutic Peptide (Eyedrop Formulation for Dry-AMD)

DNA Plasmid (IV Injection for PD)

Summary

• MSN nanoformulations have the potential to deliver plasmid DNA across the blood-brain barrier, enabling gene expression in brain tissue following intravenous administration.

• MSNs with specific modifications allow for controlled biodistribution and targeted transgene expression in the brain and peripheral organs.

• A DNA plasmid encoding the AADC gene, delivered via MSN nanoformulation, is currently under development for the treatment of Parkinson’s disease.

DNA-eGFP Plasmid Brain Delivery