NanoGenXb

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Naked genetic material has a short serum half-life due to rapid breakdown by nucleases, resulting in their low bioavailability and entry into target cells. Thus, development of any clinically translatable nucleic acid therapy requires a robust and efficient delivery agent.

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For successful gene delivery, the agent has to overcome several barriers.

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1. DNA and RNA are negatively charged molecules that require stable loading to the delivery agent.

2. The agent has to be taken up by cells through endocytosis or macropinocytosis.

3. Lastly, the agent has to drive DNA delivery to the nucleus.

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Thus, viruses and synthetic material (non-viral) have been explored as gene delivery agents.

However, due to the limitations of the current viral and non-viral agents, there is a need for a robust platform that efficiently delivers nucleic acids to primary cells with low cytotoxicity. 

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Our in vitro and in vivo small animal studies till date indicate that NanoGenXB has the following distinctive properties

 

Improved specificity without additional functionalization with targeting groups such as peptide or antibodies

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NanoGenXB shows cell-specific uptake and release of genetic material in cell’s nucleus to facilitate high transfection efficiencies (>90%) in immortal and primary cells. It is preferentially uptaken into cells that overexpressing Epidermal growth factor receptor (EGFRs). This cell surface protein plays an important role in the division of cells. It is found at abnormally high levels on the surface of many types of cancer cells, so these cells may divide excessively in the presence of epidermal growth factor ligand-independent.

 

Allows increased gene-loading capacity

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NanoGenXB’s large surface area allows high loading concentrations (>50%) of DNA or SiRNA. In contrast, viral agents are limited by the size of genetic material they can carry. They may induce adverse immune responses and viral DNA may integrate into the chromosomal DNA to cause deleterious mutations. Non-viral agents such as liposomal nanoparticles tend to have low transfection efficiency with large kb-sized DNA and induce inflammation. Polycations have better transfection efficiencies and condense large DNA, but are toxic. Further, transfection efficiency of a non-viral vector depends on its ability to escape endosomes and enter the nucleus. Nuclear entry of genetic material is difficult because of the small size of nuclear pores, and typically depends on cell division. Non-viral vectors are sub-optimal for transfection of primary cells.

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Facilitates stimulus-responsive controlled gene release

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NanoGenXB absorbs light at biological relevant near infrared (740–820 nm) wavelengths as well as radio- and microwave frequencies. These broad electromagnetic absorption property can be exploited for to facilitate an “active” stimulus-based release of pharmaceuticals.

 

Enables real-time tracking by multimodal imaging systems

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The optical and magnetic properties of NanoGenXB allows its detection by optical, photoacoustic imaging, and magnetic resonance imaging systems. Contrast-enhanced multimodal imaging employing NanoGenXB with its inherent scalability will provide complementary information of the therapeutic nucleic acid, from imaging of individual blood capillaries or lymph nodes to entire organs.

 

Market

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The global transfection technologies market includes non-viral vectors such as lipofection, Calcium Phosphate, physical methods such as electroporation, nucleofection, magnetofection, gene gun, and viral vectors. The key growth area of transfection products is the application in therapeutic area, where transfection is effectively used in treatment of cancer and other genetic disorders. Growth in gene therapy is also driving the transfection market. The global market for transfection products is expected to reach US $601 million by 2017 (CAGR of 9.32%).