Drug intelligence / Profile preview

Ntx-DBI

Development stage
Preclinical
Lead developer
Northwestern University
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intrathecal
01

Overview

Ntx-DBI is a first-in-class therapeutic consisting of a lipid-conjugated small interfering RNA (siRNA) designed to target Diazepam Binding Inhibitor (DBI), also known as Acyl-CoA-binding protein (ACBP). Developed by researchers at Northwestern University, the drug utilizes a novel lipid-conjugate delivery system to distribute the siRNA throughout the central nervous system. DBI is upregulated in epileptogenic foci and glioblastoma cells, where it contributes to seizure activity and tumor progression. By silencing DBI, Ntx-DBI inhibits fatty acid oxidation and promotes catastrophic lipid peroxidation, leading to glioblastoma cell death and reduction of spontaneous seizures. In pre-clinical models, it has demonstrated the ability to eliminate seizure-induced mortality and significantly increase survival in glioma models.

02

Targets

ACBP (Acyl-CoA-binding protein)

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