OUR CORE TECHNOLOGY
The key discovery leading to our patented core technology was that muscle tissues can take up polynucleotide genetic material, such as DNA or RNA, directly, without the use of viral components or other delivery vehicles, and subsequently express the proteins encoded by the genetic material for periods ranging from weeks to more than a year. We often describe our approach as "DNA delivery technology" because it typically involves designing and constructing closed loops of DNA called plasmids, or pDNAs. These pDNAs contain a DNA segment encoding the protein of interest, as well as short segments of DNA that control protein expression. Plasmids can be manufactured using uniform methods of fermentation and processing. This could result in faster development times than technologies that require development of product-specific manufacturing processes.
Since the initial discovery of our DNA delivery technology, our researchers have improved the design of our plasmids to provide increases in efficiency of gene expression and immunogenicity. In addition, we are developing other formulation and delivery technologies, including the use of lipid molecules, synthetic polymers called poloxamers, and other approaches, to enhance DNA expression or increase the immune response in DNA vaccine applications. We own broad rights in the United States and in other key markets to certain non-viral polynucleotide delivery technologies through our series of patents. Benefits of our DNA delivery technologies may include the following, which may enable us to offer novel treatment alternatives for diseases that are currently poorly addressed:
- Broad Applicability. Our DNA delivery technologies may be useful in developing vaccines for infectious diseases, in which the expressed protein induces an immune response; novel therapies for cancer, in which the expressed protein is an immune system stimulant or tumor suppressor; and therapeutic protein delivery, in which the expressed protein is a therapeutic agent,
- Convenience. Our DNA-based biopharmaceutical product candidates are intended to be administered on an outpatient basis,
- Safety. Our product candidates contain no viral components that may cause unwanted immune responses, infections, or malignant and permanent changes in the targeted cells' genetic makeup,
- Repeat Administration. Our product candidates contain no viral components that may preclude multiple dosing with a single product or use in multiple products,
- Ease of Manufacturing. Our product candidates are manufactured using uniform fermentation and purification procedures, and
- Cost-Effectiveness. Our DNA delivery technologies may be more cost-effective than other approaches. They may also cause fewer potential side effects, which itself may reduce per patient treatment costs.
Why DNA Vaccines?
Vaccines are a powerful tool in the effort to prevent or treat infectious diseases. Our DNA delivery technology may offer both technical and economic advantages that would allow development of vaccines not feasible with conventional vaccine approaches. Made up of DNA that encodes certain proteins associated with a target pathogen, rather than the pathogen itself, these DNA vaccines can induce potent antibody and T-cell immune responses. Critically, our vaccines contain no viral particles, are non-infectious, and can be administered on repeat basis without unwanted immune responses. Additionally, our DNA vaccines have the potential to achieve proof of concept more quickly and cost-effectively than conventional vaccines. This is especially important because it enables the rapid and economical development of vaccines for small populations and for new threats, such as those posed by bioterrorism.
In fact, we believe infectious disease vaccines represent our most promising independent product development opportunities. Already, our technology is being harnessed to develop a new approach to vaccines against diseases such as CMV, influenza, HIV, Ebola, West Nile virus and SARS. We have several novel product candidates in development with major pharmaceutical partners. We are leveraging that expertise, together with vaccine industry leadership, an expanded research staff and manufacturing capabilities, to fuel the growth of our independent vaccine programs.
For an overview of how our DNA vaccines work, click on the link below.