Drug intelligence / Profile preview

OXB-401

Development stage
Discontinued
Lead developer
Oxford BioMedica
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intrathecal
01

Overview

OXB-401, also known as MoNuDin, is a preclinical-stage gene therapy candidate developed by Oxford Biomedica for the treatment of Amyotrophic Lateral Sclerosis (ALS). It utilizes the company's proprietary LentiVector platform to deliver the gene for Vascular Endothelial Growth Factor (VEGF) directly to the central nervous system. The therapeutic rationale is based on the neuroprotective properties of VEGF, which has been shown to promote the survival of motor neurons and protect them from degeneration. Reduced levels of VEGF have been linked to the pathogenesis of motor neuron diseases, and OXB-401 was designed to restore these levels to slow or halt the progression of ALS. Although the program showed promise in preclinical models, it has not advanced to clinical trials and is no longer listed in Oxford Biomedica's active development pipeline.

Brand names
MoNuDin
Other names
MoNuDin
02

Targets

PAH (Phenylalanine hydroxylase)

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