To unlock more targeted, effective treatments, genetics researchers are exploring mutations responsible for some of the most common motor neuron diseases. These discoveries may lead to gene therapy treatment routes that do a better job of slowing disease progression than current medications. Much like gene therapy in oncology has brought improvement in cancer regression in recent years, we believe gene therapy—particularly therapy that targets and modulates RNA—is the future of neurology.
It is estimated that the number of PD cases is likely to grow significantly as populations grow older around the world and it is predicted that PD prevalence will at least double by 2030 in the US alone. As a result, it has become increasingly clear that these novel PD treatments and others need to be advancing to reach the clinical realm.