EpilepsyGTx and Viralgen collaborate to advance AAV gene therapy program for epilepsy
Collaboration with AAV-specialist CDMO will support scalable production of EPY201 for focal refractory epilepsy affecting 10 million people
1 Oct 2026
EpilepsyGTx, a biotechnology company developing gene therapies for refractory epilepsy, and Viralgen, a contract development and manufacturing organization (CDMO) specializing in recombinant adeno-associated virus (AAV) gene therapies, have announced a partnership to support production of EpilepsyGTx’s lead investigational gene therapy program targeting patients with focal refractory epilepsy (FRE), EPY201, ahead of first-in-human clinical trials.
As part of the collaboration, Viralgen will use its proprietary Aava™ manufacturing platform to provide a reliable, efficient solution for production of EPY201. The platform enables high-yield, scalable manufacturing across AAV serotypes, and has already supported the production of 1,500 AAV batches.
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.
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, offering patients with FRE the prospect of seizure freedom.
Nicolas Koebel, Chief Executive Officer of EpilepsyGTx, commented, said, “Viralgen’s AAV manufacturing expertise and platform designed to support programmes from early development through to commercial scale will be critical as we prepare to advance EPY201 into clinical development. Viralgen’s infrastructure, know-how, and regulatory track record make it an excellent manufacturing partner as we work to bring our novel gene therapy approach closer to the clinic.”
Jimmy Vanhove, Chief Executive Officer of Viralgen added, “We are proud to partner with EpilepsyGTx on this important programme. Our Aava platform is purpose-built to scale AAV programmes efficiently, and our manufacturing expertise positions us to help EpilepsyGTx advance EPY201 toward patients with focal refractory epilepsy as rapidly as possible.”
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What is EPY201, EpilepsyGTx’s gene therapy for focal refractory epilepsy?
EPY201 is EpilepsyGTx’s lead investigational gene therapy for focal refractory epilepsy (FRE). It is designed for direct delivery into the seizure focus to reduce neuronal hyperexcitability locally while limiting exposure elsewhere in the brain. If successful, it could provide a single, minimally invasive option without resection or ablation of brain tissue.
How will Viralgen support EPY201 production before first-in-human clinical trials?
Viralgen will use its proprietary Aava™ manufacturing platform to produce EPY201 ahead of first-in-human clinical trials. The platform supports high-yield, scalable manufacturing across recombinant adeno-associated virus (AAV) serotypes and has already supported 1,500 AAV batches. Viralgen’s infrastructure, expertise, and regulatory track record can support programmes from early development through commercial scale.
How many people are affected by focal refractory epilepsy worldwide?
Focal refractory epilepsy affects approximately 10 million patients worldwide, including two million across the US, UK, and EU. Focal epilepsy involves seizures arising from a specific brain region. It is considered refractory, or pharmacoresistant, when seizures persist after trials of at least two tolerated and appropriately selected antiseizure medications.