EpilepsyGTx partners with ClearPoint Neuro to support targeted delivery of lead gene therapy in epilepsy
- ClearPoint Neuro’s system enables precise delivery to the seizure focus, ensuring best possible efficacy and safety of EpilepsyGTX’s investigational gene therapy
- Partnership will support first-in-human Phase 1/2a clinical trials of EPY201 in patients with focal refractory epilepsy
London, UK, and CALIFORNIA, USA, 14 JULY 2026: EpilepsyGTx, a biotechnology company developing cutting-edge gene therapies to treat refractory epilepsy, and ClearPoint Neuro (“ClearPoint”), a global therapy-enabling company offering precise navigation and delivery to the brain, today announced a partnership to support the targeted delivery of EpilepsyGTx’s lead gene therapy candidate, EPY201, for focal refractory epilepsy (FRE).
Under the agreement, EpilepsyGTx will use the ClearPoint SmartFlow Cannula and the ClearPoint Neuro Navigation system to administer EPY201 in its upcoming first-in-human Phase 1/2a clinical trial for FRE. ClearPoint’s MRI-guided stereotactic navigation and delivery platform is designed to support sub-millimetric accuracy for delivery to defined targets in the brain, enabling intraprocedural visualisation of delivery to the intended seizure focus. The ClearPoint Navigation System is installed in over 100 active clinical sites globally for the delivery of gene and cell therapies to the central nervous system within the scope of preclinical studies, approved clinical trials, and commercial procedures.
Focal epilepsy describes a group of disorders in which seizures arise from a specific part of the brain. When seizures persist following trials of at least two tolerated and appropriately chosen antiseizure medications, epilepsy is deemed refractory, or pharmacoresistant. FRE affects approximately 10 million patients worldwide1, including two million patients in the US, UK, and EU. EPY201 is designed to be delivered directly into the seizure focus, with the aim of reducing neuronal hyperexcitability locally while limiting exposure beyond the target region in the brain. If successful, this approach will offer patients a single, minimally invasive option that does not require resection or ablation of brain tissue
Nicolas Koebel, Chief Executive Officer of EpilepsyGTx, commented: “Accurate delivery to the seizure focus is central to the potential of EPY201 in FRE. ClearPoint’s guided navigation system provides the precision and intraprocedural visualisation we need as we prepare to evaluate EPY201 in first-in-human trials. We look forward to working with ClearPoint to support safe, targeted administration of EPY201 for people living with focal refractory epilepsy.”
Jeremy Stigall, Chief Business Officer & General Manager of Biologics and Drug Delivery at ClearPoint Neuro, stated: “EpilepsyGTx is developing a targeted gene therapy approach for focal refractory epilepsy, a devastating condition affecting millions of patients. We are pleased to partner with them as the team advances this important programme into clinical evaluation.”
1. Vaughan et al (2019): https://thejns.org/doi/abs/10.3171/2018.3.JNS171722.
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Nicolas Koebel
EpilepsyGTx
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About EpilepsyGTx
EpilepsyGTx is a biotech company developing cutting-edge gene therapies for epilepsy. Its technology offers patients with focal refractory epilepsy (FRE), the prospect of seizure freedom in a single procedure, delivered directly to the seizure focus with minimal damage to surrounding brain tissue.
The Company has generated substantial evidence of potential anti-seizure activity and tolerability in translatable preclinical models of FRE for a variety of seizure focus locations. The Company’s lead programme, EPY201, is administered to the seizure focal point via intraparenchymal delivery, reducing neuronal activity, and preventing seizures. EPY201 is entering first-in-human clinical trials for FRE in 2026.
EpilepsyGTx was founded in 2021 based on pioneering research from University College London Institute of Neurology, with its founders bringing many years of unique experience in synaptic research. For more information, please visit : www.epilepsygtx.com.
About EPY201
EPY201 is an investigational gene therapy targeting focal refractory epilepsy. EPY201, also known as AAV9-CAMK2A-EKC, utilizes an AAV9 capsid, a CAMK2A promoter, and an engineered naturally occurring form of the Kv1.1 potassium channel (referred to as “EKC”). The therapy is administered directly to the target seizure focus, the specific region of the brain responsible for seizures, via intraparenchymal delivery.
About ClearPoint Neuro
ClearPoint Neuro is a device, cell, and gene therapy-enabling company offering precise navigation to the brain and spine. The Company uniquely provides both established clinical products as well as preclinical development services for controlled drug and device delivery. The Company's flagship product, the ClearPoint Neuro Navigation System, has FDA clearance and is CE-marked. ClearPoint Neuro is engaged with healthcare and research centers in North America, Europe, Asia, and South America. The Company is also partnered with the most innovative pharmaceutical/biotech companies, academic centers, and contract research organizations, providing solutions for direct central nervous system delivery of therapeutics in preclinical studies and clinical trials worldwide. To date, thousands of procedures have been performed and supported by the Company's field-based clinical specialist team, which offers support and services to our customers and partners worldwide. For more information, please visit www.clearpointneuro.com.