Non-Viral Gene Therapy
Gene therapy generally refers to the delivery of DNA into a patient’s cells to treat a target disease. The two major classes of gene therapy are viral, which uses recombinant viruses to promote long-term or permanent genomic modification, and non-viral, which uses a naked DNA plasmid to promote short-term therapeutic protein expression locally at the site of drug administration. While viral vectors also have the potential to promote localized gene expression, the permanent modification of the genome, complex manufacturing process and immunogenic response associated with viral delivery systems make them less attractive than a non-viral strategy to induce SDF-1 expression.
We believe there are several advantages to using a non-viral gene therapy strategy for the delivery of SDF-1, including:
- Localized drug delivery. A non-viral DNA plasmid is delivered directly to the site of injured tissue creating a localized homing signal at the site of delivery, with no significant expression of the therapeutic protein observed outside the targeted organ.
- Optimized duration of expression. A single administration of a non-viral gene therapy produces a prolonged window of therapeutic protein expression and does not depend on a patient complying with a dosing regimen that could span weeks or months.
- Potential for repeat administration. The body’s immune response to viral gene therapies may limit or prevent their use beyond, or even before, the initial application. Non-viral gene therapies have been shown to be non-immunogenic and thus may be administered repeatedly. We believe that the potential ability to repeat dose our gene therapy is an important product characteristic for targeting chronic, progressive diseases that may not be fully treated with a single drug administration.
- Safety profile. Non-viral DNA plasmids are not integrated into the genome and do not cause a permanent genetic modification. We believe this mitigates the potential for long-term complications and infections that can be associated with viral gene therapy.
- Cost-effective manufacturing. The manufacturing process for non-viral DNA plasmids is highly scalable and cost-effective. This simplified process results in products exhibiting greater stability than their viral counterparts, a longer shelf life and a lower overall cost of goods.