Our Science: Overview
For patients suffering from debilitating genetic diseases, Code Bio’s novel non-viral gene delivery platform offers new hope for meaningful gene therapy solutions, across numerous indications, that are titratable, have the potential to be re-dosable, and able to address diseases with mutations in large genes.
Our 3DNA platform stands apart because of its ability to overcome serious limitations and liabilities of current gene therapies, by enabling the delivery of large genes, improving cellular targeting, reducing immunogenicity, enabling re-dosability, and simplifying manufacturing. The unique 3DNA vector has the potential to deliver genes of all sizes, resulting in unparalleled utility across a broad range of genetic disorders.
The 3DNA vector is a multivalent structure with 36 binding sites to which targeting molecules and DNA constructs containing therapeutic genes can be attached. This property enables rapid optimization of both binding avidity and therapeutic potency.
Delivery of Large Gene Constructs
Our proprietary 3DNA vector design is not only capable of efficient delivery of gene therapies to cells, but also eliminates the size constraints imposed by a number of viral vector technologies. The 3DNA vector has already been shown to be capable of delivering genes approaching 10kb and we have yet to reach the upper limit for gene size that can be delivered.
Our 3DNA delivery strategy improves cellular targeting and helps minimize off-target toxicities through a dual targeting approach. First, targeting molecules, such as antibodies, peptides or small molecules, which bind to cell surface proteins expressed on the target cells, are attached to the 3DNA vector. After binding to the target cell, the 3DNA vector and cargo are internalized into the cells of interest via a receptor-mediated process. By exploiting and withstanding the endocytotic process, the platform delivers gene sequences from outside the cell to inside the cell and cell nucleus. The dual targeting process is complete once a tissue specific promoter drives the expression of the delivered gene construct, minimizing off-target cell expression.
The 3DNA vector has been designed to eliminate sequence specific elements that trigger immune responses such as those initiated by toll-like receptors (TLR’s).
Manufacturing of Code Bio’s 3DNA vector is simple, reproducible, and scalable. The 3DNA vector can be made in bulk and stored. Generating final gene therapy product is a relatively straightforward process of taking the “off the shelf” 3DNA vector scaffold and attaching the relevant targeting molecules and the DNA constructs containing the therapeutic gene.
Code Bio has extensive preclinical data demonstrating targeting, safety and efficacy of its novel 3DNA delivery platform.