EpilepsyGTx, a biotechnology company focused on developing gene therapies for refractory epilepsy, has raised $33 million in a Series A financing to advance its lead program, EPY201, into first-in-human clinical trials. The funding will support Phase 1/2a studies designed to evaluate the safety and efficacy of the therapy in patients with focal refractory epilepsy, a severe form of epilepsy that does not respond to existing treatments.
The Series A round included investment from XGEN Venture, the British Business Bank, and a global biopharmaceutical company. The new capital positions EpilepsyGTx to advance its most advanced candidate into the clinic while also laying the groundwork for future financings to expand its broader pipeline of gene therapies targeting refractory epilepsy and other disorders driven by neuronal hyperexcitability.
EPY201 is an adeno-associated viral gene therapy engineered to reduce neuronal hyperexcitability, the underlying driver of seizures in focal refractory epilepsy. Unlike systemic gene therapies, EPY201 is delivered locally to the epileptogenic focus in the brain, minimizing systemic exposure and associated complications. The company says this localized delivery could offer patients seizure freedom with a single minimally invasive intervention, without the need for surgical resection or ablation of brain tissue and without reliance on long-term use of multiple antiseizure medications.
Focal refractory epilepsy represents a significant unmet medical need. Focal epilepsy arises from a specific region of the brain. When seizures persist despite treatment with at least two appropriately selected and tolerated antiseizure medicines, the condition is classified as refractory. The company estimates that focal refractory epilepsy affects approximately 10 million people worldwide, including around 2 million patients across the United States, the United Kingdom, and the European Union. EpilepsyGTx believes its approach has the potential to dramatically improve outcomes, survival, and quality of life for this patient population.
In addition to advancing EPY201 through early-stage clinical development, EpilepsyGTx plans to use future financings to progress additional programs aimed at treating refractory epilepsy and related neurological disorders characterized by abnormal neuronal activity. The company previously raised $10 million in pre-seed and seed funding led by the UCL Technology Fund, managed by AlbionVC in collaboration with UCL Business, with participation from Zcube, the venture capital arm of Zambon.
Support: Taylor Wessing LLP acted as legal advisor to EpilepsyGTx on the financing, while A&O Shearman served as legal advisor to XGEN Venture. Panmure Liberum Cambridge Capital acted as financial advisor on the transaction.
KEY QUOTES:
“Refractory epilepsy is a devastating condition causing unpredictable and life-threatening seizures, and affecting millions of patients worldwide. Our novel gene therapy EPY201 delivered directly to the seizure focus has the potential to stop seizures with a single, minimally invasive administration. In doing so, it will change the way refractory epilepsy has been treated for decades. We are proud to have the support of such high calibre investors as we progress into clinical trials.”
Nicolas Koebel, Chief Executive Officer, EpilepsyGTx
“EpilepsyGTx is pioneering a novel, locally administered gene therapy approach designed to achieve targeted modulation of epileptogenic brain regions. We believe that localized gene delivery offers a powerful avenue for durable and disease-modifying interventions in severe neurological disorders and are excited to support the company as EPY201 progresses toward clinical evaluation.”
Federica Draghi, Managing Partner, XGEN Venture

