EpilepsyGTx is Developing a Single-Dose Gene Therapy for Focal-Refractory Epilepsy

EpilepsyGTx, a biotechnology company focused on research and development of cutting-edge gene therapies to treat refractory epilepsy, led by Nicolas Koebel, Prof. Dimitri Kullmann, Dr. Joanne Broadhead, and Dr. Luiz Almeida Silva, has secured $33 million in a Series A financing from XGEN Venture, the British Business Bank, and a global biopharmaceutical company. Taylor Wessing LLP advised EpilepsyGTx on the round, A&O Shearman advised XGEN Venture, and Panmure Liberum Cambridge Capital served as financial advisor.

The new funding will advance first-in-human Phase 1/2a trials for EPY201, an AAV gene therapy targeting focal refractory epilepsy (FRE). Delivered directly to the epileptogenic focus, EPY201 reduces neuronal hyperexcitability without the risks of systemic delivery. The therapy aims to offer FRE patients potential seizure freedom through a single, non-resective intervention that avoids brain tissue removal and long-term dependence on multiple antiseizure medications.

EpilepsyGTx plans to expand its pipeline of gene therapies for refractory epilepsy and other neuronal hyperexcitability disorders with future funding. Focal epilepsy involves seizures originating from a specific brain region, and when seizures continue despite at least two suitable antiseizure medications, it is classified as refractory. FRE affects roughly 10 million people worldwide, including about 2 million across the US, UK, and EU. EPY201 has the potential to eliminate seizures with a single procedure, offering major improvements in survival and quality of life.

Nicolas Koebel, CEO of EpilepsyGTx, said refractory epilepsy remains a severe, unpredictable condition, and EPY201, a minimally invasive, single-dose gene therapy delivered directly to the seizure focus, has the potential to transform long-standing treatment approaches. He noted the company’s pride in having strong investor backing as it moves into clinical trials.

Federica Draghi, Managing Partner at XGEN Venture, said EpilepsyGTx is advancing a pioneering, locally delivered gene therapy designed to precisely modulate epileptogenic brain regions. She added that targeted gene delivery could enable durable, disease-modifying treatments for serious neurological disorders, and expressed support as EPY201 advances toward clinical testing.

EpilepsyGTx previously secured $10 million in pre-seed and seed funding led by the UCL Technology Fund, managed by AlbionVC with UCL Business, alongside participation from Zcube, Zambon’s venture arm.

Taylor Wessing LLP advised EpilepsyGTx on the round, A&O Shearman advised XGEN Venture, and Panmure Liberum Cambridge Capital served as financial advisor.