New Partnership to Test Advanced Gene Therapy for Hard-to-Treat Epilepsy

EpilepsyGTx, a company working on new treatments for epilepsy, has teamed up with ClearPoint Neuro to use a specialized tool for delivering a gene therapy called EPY201 directly to the area of the brain where seizures start. This approach is being prepared for the first human trials, which will focus on people with focal refractory epilepsy—meaning their seizures continue even after trying at least two different epilepsy medications that should work for them.

To deliver EPY201, the team will use the ClearPoint SmartFlow Cannula and ClearPoint Neuro Navigation system. This method uses MRI guidance to place the therapy with extreme precision—down to less than a millimeter—and allows doctors to see exactly where the medication is going in real time during the procedure. ClearPoint’s system is already used in over 100 medical centers around the world for gene and cell therapies in research studies, approved clinical trials, and even some approved treatments.

Focal epilepsy happens when seizures begin in a specific part of the brain. When seizures don’t improve with standard medications, the condition is called refractory epilepsy. This type affects about 10 million people worldwide, including 2 million in the United States, United Kingdom, and European Union. EPY201 is designed to be given directly to the area of the brain causing seizures. The goal is to calm overactive brain cells in that spot while keeping the treatment from spreading to other parts of the brain. If this works, it could provide a single, less invasive treatment option that doesn’t require removing or destroying brain tissue.

Nicolas Koebel, CEO of EpilepsyGTx, said: “Precisely delivering EPY201 to the exact spot where seizures start is key to its potential success. The ClearPoint system gives us the accuracy and real-time feedback we need as we get ready to test this therapy in people for the first time.”

He also added: “We’re excited to work with ClearPoint to help safely and accurately give EPY201 to people living with focal refractory epilepsy.”

Jeremy Stigall, Chief Business Officer and General Manager of Biologics and Drug Delivery at ClearPoint Neuro, said: “EpilepsyGTx is creating a targeted gene therapy for focal refractory epilepsy, a very challenging condition that affects millions. We’re happy to partner with them as they move this important treatment into human trials.”

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