Maryland Patent of the Month – May 2023
Gene therapy is a promising approach for treating a wide range of diseases, including cancer, genetic disorders, and neurological diseases. American Gene Technologies International Inc. has developed a gene transfer vector that offers versatility, control, high expression, stable multiple gene expression, tolerability, and safety, making it a potential tool for effective gene therapies.
Research into disease therapies have explored numerous approaches with varied results. While adenovirus vectors can efficiently deliver therapeutic genes, they are highly immunogenic, and long-term expression in target tissues is not observed. Lentiviruses have the potential for long-term expression, but safety has been a concern, although various strategies have been developed to eliminate their ability to replicate. The use of RNAi agents for disease therapies has also gained immense excitement, with studies reporting on their potential to suppress tumor growth. However, delivery of siRNA for long-term expression in target cells and tissues has been particularly challenging.
With such limitations, research can experience stagnation. American Gene Technologies has developed a platform which harnesses the power of lentivirus vectors to cure diseases. They liken their platform to Apple’s iOS platform which provides a base for other app developers to build. In the same way, American Gene Technologies’ platform will provide the base for other researchers to build on.
Using their lentiviral vector offers the potential for long-term expression and stable multiple gene expression and can be directed for use in the targeted delivery of multiple therapeutic molecules.
A recently granted patent describes the use of safe lentiviral vectors for the targeted delivery of therapeutic molecules. This patent describes a lentiviral transfer system that includes (a) a self-inactivating transfer vector made of multiple gene units and (b) a helper construct. The unique design of the gene units and the helper construct result in a deletion so that the vector produces non-functional env proteins. This method allows researchers to easily combine and regulate the expression and delivery of treatments, while taking effective safety measures.
At present, American Gene Technologies has the following priority areas for clinical drug development:
- A Cure for HIV disease that combines immunotherapy with lentivirus delivery of genetic medicine to reconstitute effective and natural immune control of this dangerous virus;
- A novel strategy for curing phenylketonuria (PKU) – the second most common genetic disease in the U.S. and Europe; and,
- Innovative approaches to immuno-oncology that use genetic medicine to modify tumors so these sites of disease become potent stimulators of the immune system causing tumor destruction, removing the genetic modification and avoiding long-term consequences of altered T cells
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