#USA #gene therapy #San Sebastián #Viralgen #EpilepsyGTx

Collaborative Advances in Gene Therapy for Refractory Epilepsy

EpilepsyGTx, a pioneering biotechnology firm focused on developing innovative gene therapies, has announced a strategic partnership with Viralgen, a prominent contract development and manufacturing organization (CDMO) specializing in adeno-associated virus (AAV) gene therapies. This collaboration is set to significantly propel the development of EPY201, a novel gene therapy aimed at treating patients with refractory focal epilepsy, currently in preparation for its first clinical trial in humans.

The collaboration is particularly notable as it leverages Viralgen's proprietary Aava™ manufacturing platform, known for its reliability and scalability in producing high-yield AAV serotypes. This technology has already successfully facilitated the production of over 1,500 AAV batches, highlighting its efficiency and effectiveness in supporting gene therapy advancements.

Understanding Refractory Focal Epilepsy

Refractory focal epilepsy is characterized by seizures that originate from a specific region within the brain, and it affects approximately 10 million individuals globally, including around 2 million in the United States, the United Kingdom, and the European Union. When a patient's seizures persist despite having tried at least two well-tolerated antiepileptic drugs tailored to their needs, they are diagnosed with refractory epilepsy.

The EPY201 therapy is designed to be administered directly to the seizure focus, with the aim of locally reducing neuronal hyperexcitability while minimizing exposure to other brain areas. If clinical trials yield positive results, this therapeutic approach could provide a unique and less invasive option for patients suffering from refractory focal epilepsy, potentially allowing them to live seizure-free without the need for tissue resection or ablation.

Expert Insights on the Collaboration

Nicolas Koebel, CEO of EpilepsyGTx, expressed enthusiasm regarding the partnership, stating, “Viralgen's AAV manufacturing expertise and their platform are vital as we prepare to move EPY201 into clinical development. Their infrastructure, knowledge, and regulatory experience make them an exceptional manufacturing partner, helping us advance our innovative gene therapy approach into clinical application.”

Jimmy Vanhove, CEO of Viralgen, also emphasized the importance of this collaboration, remarking, “We are proud to partner with EpilepsyGTx on this critical program. Our Aava platform is specifically designed to efficiently develop AAV programs at scale, and our manufacturing expertise will support EpilepsyGTx in making EPY201 available to patients with refractory focal epilepsy as quickly as possible.”

A Closer Look at EPY201

The experimental gene therapy EPY201, also designated as AAV9-CAMK2A-EKC, employs an AAV9 capsid, a CAMK2A promoter, and a modified version of the naturally occurring potassium channel Kv1.1 known as “EKC.” This cutting-edge treatment allows for direct administration into the targeted seizure focus via an intraparenchymal method, effectively reducing neuronal activity and preventing seizures.

Clinical trials for EPY201 are expected to commence in the first half of 2027, marking a significant advancement in the quest for effective treatments for refractory focal epilepsy.

About EpilepsyGTx

Founded in 2021, EpilepsyGTx emerged from groundbreaking research conducted at the Institute of Neurology at University College London. The founders bring extensive experience in synaptic research, focusing on providing patients with the hope of seizure relief through a minimally invasive intervention directly targeting the seizure focus.

About Viralgen

Established in 2017 as a subsidiary of AskBio Inc., part of Bayer AG, Viralgen specializes in AAV-based cell and gene therapies. They provide comprehensive support, from early-phase development to commercial-scale production. Their state-of-the-art facility in San Sebastián, Spain, boasts cGMP certified suites equipped for high-yield production, ensuring swift delivery of transformative gene therapies for patients worldwide.

For more information on EpilepsyGTx and their groundbreaking therapies, visit EpilepsyGTx. Similarly, for details about Viralgen and their services, head to Viralgen.