Concepts and vision

Eukarÿs’ paradigm: safe, efficient, well-tolerated, economically viable, synthetic non-viral gene therapy for rare monogenic and common complex disorders.

 

The synthetic gene therapy developed by Eukarÿs relies on artificial double-stranded DNA molecules, which are produced artificially. Synthetic DNA contains both the C3P3 gene, plus one or several target genes under control of the enzyme. Once the C3P3 enzyme is expressed in the targeted organ, the enzyme self amplifies in the cytoplasm, and then transcribes the genes of interest and generates fully processed mRNA molecules of interest.

Synthetic gene therapy sharply differs from other existing gene therapy approaches:

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Eukarÿs’ synthetic gene therapy relies on the artificial C3P3 technology, which produces large amounts of target mRNA(s) – a critical drawback that hampers the uses of most non-viral gene therapies.
Synthetic gene therapy is being developed to prevent insertional mutagenesis of the artificial double-stranded DNA in the host-cell genome, a safety issue that exposes to the risk of development solid or blood cancers – a key limitation of viral gene therapy
Artificial DNA is entirely devoid of non-methylated CpG dinucleotides – an important driver of the innate immune response that can hamper the tolerance of therapeutic DNA and its efficacy when repeatedly administered
Without limitation in the length of genes – a significant issue of some viral vectors, especially AAV. Synthetic gene therapy makes it therefore possible to express and/or inhibit several genes at the same time by the C3P3 enzyme. This feature makes possible the development of treatments for rare simple monogenic, as well as common complex disorders.