David Anderson, PhD, CNS Research Lead at Spark Therapeutics talks about the numerous neurological conditions that have been identified and the concern that most of them are rare diseases and most of them are not being properly managed.
“There is something like five and a half thousand diseases that have been described in the CNS and quite frankly, the vast majority those are rare diseases. They’re not Alzheimer’s disease or Parkinson’s disease,” noted Dr. Anderson, adding that most of those rare diseases are not being treated.
Dr. Anderson is hopeful that gene therapy and gene delivery systems may help many of those rare neurological conditions. “We have an opportunity to go in because we can take a gene and we can perhaps augment or replace something that’s missing – we couldn’t do that before with other technologies,” stated Dr. Anderson. “But what we have with our platform is an AAV gene therapy where we can place it into the CNS, possibly even into the one region that’s being directly affected. it gives us an opportunity to really affect the disease in a way that we’ve never had before.”
One advantage that rare neurological conditions have over more common diseases, like Alzheimer’s disease, is that the pathophysiology is often less complicated and that lends itself well to gene therapy.
“If you come back to something like Parkinson’s or Alzheimer’s, or some other big disease, that other drug companies traditionally want to go after – we don’t actually know what causes these diseases. Working in the rare disease space, we mostly have an idea.”
Some of the rare neurologic conditions Spark Therapeutics is developing therapies for include Batten disease and Huntington’s disease. Last year, Spark’s first gene therapy was approved for RPE65 mutation associated retinal dystrophy.
For more information, visit sparktx.com/