EpilepsyGTx has partnered with ClearPoint Neuro to support the targeted delivery of its lead gene therapy candidate, EPY201, as the biotechnology company prepares to begin its first-in-human Phase 1/2a clinical trial in patients with focal refractory epilepsy.

Under the agreement, EpilepsyGTx will use ClearPoint’s MRI-guided navigation platform and SmartFlow cannula to deliver EPY201 directly to the seizure focus during the clinical study. The technology is designed to provide precise, image-guided delivery within the brain while allowing clinicians to monitor administration in real time.

Focal refractory epilepsy affects patients whose seizures continue despite treatment with at least two appropriately selected anti-seizure medicines. By delivering gene therapy directly to the area of the brain where seizures originate, EPY201 aims to reduce abnormal neuronal activity while limiting effects on surrounding healthy tissue.

Unlike surgical procedures that involve removing or destroying brain tissue, the investigational therapy has been designed as a minimally invasive approach that targets the seizure focus without resection or ablation.

Nicolas Koebel, chief executive officer of EpilepsyGTx, said: “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.”

ClearPoint’s navigation platform is already used at more than 100 clinical sites worldwide for the delivery of gene and cell therapies targeting disorders of the central nervous system. The collaboration is intended to provide the delivery technology required for EPY201’s upcoming clinical evaluation.

Jeremy Stigall, chief business officer and general manager of biologics and drug delivery at ClearPoint Neuro, said: “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.”

Gene therapies targeting neurological disorders present unique delivery challenges because they must reach precise locations within the brain. MRI-guided stereotactic navigation has become an increasingly important technology for investigational treatments that require accurate placement while minimising the risk of damage to surrounding tissue.

The partnership will support EpilepsyGTx’s planned Phase 1/2a trial as the company advances EPY201 into clinical testing for patients with focal refractory epilepsy.