PDX Pharmaceuticals has published recent data on PDX-002 in collaboration with the Biomedical Engineering Department of Oregon Health and Science University . PDX-002 is a nanoparticle platform for siRNA delivery designed to treat fibrosis, a complex disease characterized by increased oxidative stress, inflammation, and an excessive accumulation of extracellular matrices mainly consisting of collagen. Notably, nearly 45% of all naturally-occurring deaths in the western world are attributed to some form of fibrotic disease.
The work published in Biomaterials (IF=8.557) highlights the advantages of using a mesoporous silica based nanoparticle siRNA delivery platform to manage the over stimulation of pro-fibrotic genes. A gene silencing strategy against HSP47, a protein integral in collagen accumulation, proved effective in models of dermal fibrosis. More importantly, the work demonstrated the added therapeutic benefit of the mesoporous silica nanoparticle carrier due to its intrinsic antioxidant properties. Together, the work represents a continuing success of the mesoporous silica nanoparticle platform and given the most optimal gene target, identifies a viable combinational treatment strategy that can be extended to fibrotic diseases of other organs as well as inflammatory diseases.