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SmartPharm Therapeutics is a Cambridge, MA-based gene therapy company applying the next generation of non-viral DNA and RNA technologies to treat serious human diseases.
SmartPharm's stated goal is to replace recombinant protein therapeutics with gene-encoded therapeutics (GETs). The company is focusing on three main areas:
- Enzyme replacement therapy with an initial focus on Gaucher disease type 1, and other lysosomal storage diseases
- Gene-encoded biologics for cell and tissue restoration, with an initial focus on restoring heart tissues after an acute myocaridal infarction (heart attack).
- Monoclonal antibodies as a potential prophylactic measure for preventing infection from SARS-CoV-2.
SmartPharm says that it is using its non-viral gene therapy platform to develop a preventive treatment for SARS-CoV-2. The company said that it is using a custom DNA vector which will produce monoclonal antibodies which could be protective against SARS-CoV-2.
The company says it has formed strategic partnerships "with a number of organizations," including Sorrento Therapeutics, to identify neutralizing antibodies against SARS-CoV-2.
SmartPharm says it "will define a DNA that can express these antibodies in human skeletal muscle and use a delivery technology designed to allow the DNA to be delivered directly into the muscle." The company says that, after delivery into skeletal muscle via injection with a hypodermic needle, its novel DNA is designed to produce antibodies in muscle tissue within hours.
The company says that "[within] a day or two—a much faster time than a typical vaccine—protective levels of antibody could be produced, resulting in what we refer to as 'pop up-immunity': a rapidly-established window of immune protection against the virus."
