New techniques for replacing, silencing or editing the underlying genetic defects provide new hope. One striking example is gene therapy for spinal muscular atrophy, in which infants who would never have been able to sit up now have the potential to lead relatively normal lives. But the number of successes is small. The challenge is delivering the gene replacement, gene silencing, or gene editing to the right cells in the human body.
Kunwoo and colleagues developed a novel delivery technology. They have published multiple high-profile papers, including one written in collaboration with Nobel laureate Jennifer Doudna, and demonstrated that gene editing can show therapeutic effects in mice with Duchenne muscular dystrophy and autism. NIH Director, Francis Collins, wrote in an article that “this approach is especially exciting because it also holds potential for treating or even curing many other genetic diseases.”
GenEdit has continued to improve the delivery technology. The innovation comes from the NanoGalaxy™ platform, which deploys machine learning to evaluate the results from systematic screening of thousands of unique polymer nanoparticles and create optimized tissue-specific delivery systems. These unique polymer nanoparticles can then be used to deliver the components required for gene replacement, gene silencing, or gene editing to the right tissue, maximizing therapeutic benefits. Kunwoo envisions a world where we can edit out disease and rewrite patient health and to make these new genetic medicines available to patients throughout the world.