GenEdit's delivery vehicle
Our proprietary Polymer nanoparticle can deliver CRISPR protein and gRNA. Despite CRISPR in a protein form has many advantages, delivery has been a challenge limiting its application. To deliver CRISPR protein, encapsulation of CRISPR ribonucleoprotein (RNP) and efficient uptake by target cell need to be achieved. GenEdit solves the challenge with its proprietary polymer nanoparticle platform.
GenEdit's platform enables broad product opportunity
Various different types of tissues require delivery technology that is specifically tailored to the target tissue. That is why GenEdit developed a library of polymer nanoparticles. The polymer library is used to screen a best polymer nanoparticle that encapsulates CRISPR RNP and also other therapeutic proteins. The delivery efficiency of polymer nanoparticle is tested in a target cell and animal systems. Our recent publications demonstrate efficient gene editing in target tissues: muscle in Duchenne muscular dystrophy mouse (Nature Biomedical Engineering, 2017) and brain in Fragile X syndrome mouse (Nature Biomedical Engineering, 2018). In addition, the polymer library proved the versatility by delivering a next generation CRISPR, Cpf1 in muscle (Nature Communications, 2018).
Each component of CRISPR system can be engineered to enhance functionality or delivery. We have identified several gRNA and donor DNA chemical modifications that enhance cell delivery of CRISPR/Cas9 therapeutics, and also enable enrichment of gene edited cells. The chemical engineering of gRNA and donor has numerous applications to enhance the delivery efficiency and tissue specificity of CRISPR. Moreover, the approach can deliver donor DNA together with CRISPR just like one molecule so that HDR efficiency can be enhanced