CereVasc shared data from a study demonstrating that its eShunt system may help deliver gene therapies to the central nervous system (CNS).
eShunt, a minimally invasive system, includes an endovascularly implantable cerebral spinal fluid shunt and delivery components. CereVasc designed the system to enable transvenous-transdural access to the CNS to treat communicating hydrocephalus (CH) without invasive surgery.
In an ovine model study, a standard gene therapy delivery vector — an adeno-associated virus — was successfully distributed to the brain and spinal cord. By targeting the cerebellopontine angle (CPA) cistern at the base of the brain, the location accessible by the eShunt System in ongoing clinical trials for hydrocephalus, the vector ultimately reached a host of key targets within the CNS.
After delivery, the virus reached the cerebral cortices, striatum, thalamus, midbrain, cerebellum and spinal cord. This method demonstrated minor liver distribution compared with the alternative delivery via cisterna magna, a route of administration accompanied by potential risks. Data showed comparable rates of biodistribution between the two routes of administration. The company said this suggests that the endovascular CPA approach could offer a clinically safer and minimally invasive delivery system for CNS gene and cell-based therapies.
CereVasc said the authors concluded that eShunt could provide a minimally invasive alternative for these therapies that need to reach the CNS.
“To successfully treat patients with neurological disorders, many gene therapies must have the ability to cross the blood-brain barrier. Currently available methods for accomplishing this come with obstacles, including invasiveness, procedure-related complications and limited effectiveness,” said Dan Levangie, CereVasc chair & CEO. “We’re very excited about these promising preclinical results, which suggest that the eShunt System delivery approach could provide biopharmaceutical and biotechnology organizations with a minimally invasive method for delivering gene therapies directly to the cortex and brain.”
