We are developing gene and cell based therapies. Gene therapy is addition, alteration or removal of genes within cells and biological tissues to treat diseases. A correcting genetic agent can be delivered directly to blood stream in vivo, or to the affected tissue in situ, or the targeted cells can be modified ex vivo and injected back to the patients. Cell based therapy involves injecting or implanting intact or genetically modified cells to the patients, where the cells themselves act as a curative agent.
For gene editing, we developed an improved CRISPR/CAS and TARGATT™ technologies to deliver precision genetic editing or gene replacement.
Gene delivery system is a key in gene therapy. Our therapeutics team is working with state-of-the-art systems including recombinant Adeno-Associated Viruses and lipid-nanoparticles.