Drug intelligence / Profile preview

TMEM100 plasmid

Development stage
Preclinical
Lead developer
University of Arizona
Modality
Gene Therapies, Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

TMEM100 plasmid is a non-integrating gene therapy candidate designed to express Transmembrane Protein 100 (TMEM100), a proangiogenic factor essential for lung vascular and alveolar development. In experimental models of Bronchopulmonary Dysplasia (BPD) and Pulmonary Hypertension (PH) induced by antenatal inflammation (chorioamnionitis), the delivery of this plasmid via lung endothelial-specific nanoparticles has been shown to preserve lung growth, improve alveolarization, and prevent right ventricular hypertrophy. TMEM100 acts as a downstream mediator of the BMP9/BMP10 signaling pathway, and its enhancement is being investigated as a novel therapeutic strategy to preserve angiogenesis and lung structure in premature infants at risk for chronic lung disease.

Other names
non-integrating TMEM100 plasmid
02

Targets

TMEM100 (Transmembrane protein 100)

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