EpilepsyGTx raises $33m for focal refractory epilepsy gene therapy
Esme Needham | December 10, 2025 | News story | Research and Development | EpilepsyGTx, Neurology, epilepsy
EpilepsyGTx, a biotechnology company researching and developing gene therapies for the treatment of refractory epilepsy, has announced $33m raised in Series A financing to move its lead programme through clinical trials.
EPY201, EpilepsyGTx’s lead programme, is an investigational gene therapy designed for the treatment of focal refractory epilepsy (FRE). It is administered directly to the brain region responsible for seizures, bypassing the potential complications of systemic exposure. EPY201 could provide patients with freedom from seizures without the necessity of long-term antiseizure medicine use.
FRE affects around ten million patients globally, including two million in the UK, the US and the EU. It is a refractory form of focal epilepsy, in which seizures arise from a specific part of the brain, and occurs when seizures persist despite treatment with two or more tolerated and appropriate antiseizure treatments.
The funding round included investments from XGEN Venture, the British Business Bank and a global biopharmaceutical company. EpilepsyGTx previously raised $10m in pre-seed and seed funding led by the University College London Technology Fund.
Federica Draghi, managing partner of XGEN, said: “EpilepsyGTx is pioneering a novel, locally administered gene therapy approach designed to achieve targeted modulation of epileptogenic brain regions. We believe that localised gene delivery offers a powerful avenue for durable and disease-modifying interventions in severe neurological disorders.”
The funding will allow first-in-human phase 1/2a clinical trials of EPY201 to begin. Future funding will also allow EpilepsyGTx to advance other gene therapies.
“Refractory epilepsy is a devastating condition causing unpredictable and life-threatening seizures, and affecting millions of patients worldwide,” Nicolas Koebel, CEO of EpilepsyGTx, said. “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.”
Related Content
UCB presents positive results for fenfluramine in CDKL5 deficiency disorder
UCB has announced positive results from its phase 3 GEMZ study, showing that fenfluramine can …
Rare and severe epilepsies – current treatments and advance therapeutics
The current forms of immunotherapy, how T cell therapy works and what the future holds
Epilepsy Research Institute opens 2025-26 grant round with new awards
The Epilepsy Research Institute has opened its 2025-26 Grant Round, offering a wide range of …
