The Ligandal Approach
Gene therapy requires delivery. Ligandal has developed a breakthrough nanotechnology for the precise and high-efficiency delivery of nucleic acids (CRISPR, TALEN, and RNA) to specific cells and organelles without the limitations of viruses, enabling a new standard for genetic medicine. Ligandal's gene editing efficiencies are unprecedented.
What does this mean for gene therapy?
Permanent gene therapy using viruses cannot be repeatedly administered without the immune system clearing and responding to a virus, and many viral gene therapies carry oncogenic risks. Ligandal's non-viral technology circumvents these issues. We attain extremely high efficiencies with site-specific "remove-and-replace" and "insertion" mutations, including in the most difficult to transfect cell types.
For temporary gene delivery of RNA, we can create gene expression lasting for as much as a month—far longer than the several days enabled by leading commercial technologies.
Similarly to a virus, our nanotechnology targets specific cells and delivers a payload to where it counts—the nucleus of the cell. When used for performing gene insertion and timed gene release inside of cells, Ligandal's proprietary platform is unprecedented and does not carry the risks or limitations of viral gene delivery.
Advanced Multistage Assemblies