EpilepsyGTx Taps Viralgen's Aava Platform to Manufacture AAV Gene Therapy EPY201 Ahead of First-in-Human Trial
核心洞察
EpilepsyGTx (搜索) and CDMO Viralgen (搜索) have partnered to manufacture EPY201, an investigational AAV gene therapy for focal refractory epilepsy (搜索), ahead of first-in-human clinical trials.
Viralgen (搜索) will deploy its proprietary Aava platform, which enables high-yield, scalable AAV manufacturing across serotypes and has supported production of 1,500 AAV batches.
EPY201 (AAV9 (搜索)-CAMK2A-EKC) is delivered directly into the seizure focus via intraparenchymal administration to reduce local neuronal hyperexcitability while limiting exposure beyond the target region.
EpilepsyGTx (搜索), a biotechnology company developing gene therapies for refractory epilepsy, has entered a partnership with contract development and manufacturing organization (CDMO) Viralgen (搜索) to support production of its lead investigational gene therapy program, EPY201, ahead of first-in-human clinical trials. Viralgen, a specialist in recombinant adeno-associated virus (AAV) gene therapies, will apply its proprietary Aava manufacturing platform to produce the candidate for patients with focal refractory epilepsy (搜索) (FRE).
Manufacturing Platform Built for Scale
Under the collaboration, Viralgen (搜索) will use the Aava platform to provide what the companies describe as a reliable, efficient production solution for EPY201. The platform enables high-yield, scalable manufacturing across AAV serotypes and has already supported the production of 1,500 AAV batches. Viralgen was founded in 2017 as a subsidiary of AskBio Inc. within the Bayer AG group and delivers end-to-end support from early development to large-scale commercial manufacturing.
"Viralgen (搜索)'s AAV manufacturing expertise and platform designed to support programs from early development through to commercial scale will be critical as we prepare to advance EPY201 into clinical development," said Nicolas Koebel, chief executive officer of EpilepsyGTx (搜索). "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 (搜索), said the company is "proud to partner with EpilepsyGTx (搜索) on this important program," adding that the Aava platform "is purpose-built to scale AAV programs efficiently, and our manufacturing expertise positions us to help EpilepsyGTx advance EPY201 toward patients with focal refractory epilepsy (搜索) as rapidly as possible."
Targeting the Seizure Focus Directly
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 worldwide, including two million patients in the US, UK, and EU.
EPY201, also known as AAV9 (搜索)-CAMK2A-EKC, is an investigational gene therapy that utilizes an AAV9 capsid, a CAMK2A promoter, and an engineered naturally occurring form of the Kv1.1 potassium channel (搜索), 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.
The design aims to reduce neuronal hyperexcitability locally while limiting exposure beyond the target region in the brain. If successful, the companies state, this approach would 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.
Clinical Timeline
According to the companies, the first-in-human clinical trial of EPY201 for focal refractory epilepsy (搜索) is expected to begin in the first half of 2027. The collaboration is positioned within Bayer's broader commitment to advancing gene therapies through its AskBio subsidiary, with EPY201 framed as a potential option for patients living with drug-resistant epilepsy worldwide.