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Our Technology

Transfection is a Complex Process.

Transfecting cells is a critical step in producing viral vectors for cell and gene therapies, manufacturing recombinant proteins and monoclonal antibodies for immunotherapies, editing genes through CRISPR, and applying a range of other applications such as silencing mRNA translation. Successful transfection requires navigating through several cellular barriers in a sequence of precisely orchestrated steps including:

  • DNA complexation
  • Cellular binding and uptake
  • Endosomal release and cytoplasmic diffusion
  • Nuclear transport

It’s complex. That’s why single component transfection reagents, whether they are polymers or lipids, are not sufficient to maximize efficiency of the process and retain cell viability.

In contrast to transfection reagents that consist of a single component, our transfection technology include both proprietary polymer and lipid elements. This combination enables both high transfection efficiency and low cellular toxicity.

With our transfection platforms, the polymer, lipid, and your nucleic acid come together to form a lipid polymer nanoparticle complex, which is effective at delivering cargo to nearly any cell type.

Intentional, Iterative Design of Transfection Solutions

Our transfection solutions, such as the VirusGEN® platform, are developed using an intentional, iterative design process that allows us to optimize polymer and lipid components in parallel.

We begin with vast, proprietary libraries of polymers and lipids to screen thousands of combinations of related and novel structures. When we identify promising compounds, we screen related structures in the library. New compounds are also synthesized and screened. Finally, compound hits are tuned by varying the polymer and lipid structure and combinations that balance transfection efficiency and cellular viability that give the best results for the intended application. 

We applied a similar approach to identify our RevIT™ AAV enhancer. When used in combination with our VirusGEN® transfection platform, AAV titers can be increased 2-4x.

 

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