Drug intelligence / Profile preview

NJA-312

Development stage
Preclinical
Lead developer
NapaJen Pharma
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

NJA-312 is a murine-specific small interfering RNA (siRNA) therapeutic candidate developed by NapaJen Pharma. It serves as the murine orthologue for the NJA-730 program, designed for use in preclinical proof-of-concept studies. The drug utilizes NapaJen's proprietary delivery technology, which involves complexing the siRNA with schizophyllan (SPG), a β-1,3-glucan that specifically binds to Dectin-1 receptors on antigen-presenting cells (APCs) such as dendritic cells and macrophages. By targeting CD40, a critical co-stimulatory molecule, NJA-312 silences its expression to modulate the immune response. It is primarily investigated for the treatment of acute graft-versus-host disease (GvHD), where it aims to suppress the pathological activation of T cells by inhibiting APC-mediated co-stimulation.

02

Targets

CD40 (Cluster of differentiation 40 receptor)CLEC7A (Dectin-1)

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