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.