Spark in the brain
How Spark is expanding its gene therapy focus to rare neurological disorders
Spark Therapeutics Inc. is expanding beyond eye and blood diseases with its first foray into the brain for the rare neuronal disorder Batten disease, using a gene therapy delivered to the ependymal cells in the CNS. The company believes the new technology - which it licensed from an undisclosed party - could offer a permanent solution for enzyme deficiencies in the brain and spinal cord, making the approach a better option than enzyme replacement therapies for a broad range of neurodegenerative disorders.
On Nov. 11, Spark announced its SPK-TPP1 program, slated to start IND-enabling studies this year, which is based on findings from co-founder Beverly Davidson at the Children's Hospital of Philadelphia (CHOP). The same week, Davidson's group published data in Science Translational Medicine showing a gene therapy based on adeno-associated virus serotype 2 (AAV2) can delay symptom onset and progression in a naturally occurring dog model of Batten disease...