Biotechnology firm EpilepsyGTx has secured $33m in a Series A funding round to progress its adeno-associated viral (AAV) gene therapy, EPY201, for focal refractory epilepsy (FRE), through Phase I/IIa trials.

The round saw participation from XGEN Venture, the British Business Bank, and a biopharma company.

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The newly raised capital will support the first-in-human Phase I/IIatrials to assess the efficacy and safety of the therapy in a wide population of those diagnosed with FRE.

According to the company, EPY201 is intended to decrease neuronal hyperexcitability. It is administered to the epileptogenic focus locally, thereby avoiding complications associated with systemic delivery.

This approach is designed to provide patients with FRE the possibility of seizure freedom via single intervention, eliminating the need for brain tissue resection or ablation and long-term use of antiseizure drugs.

The company has also outlined plans to progress a pipeline of gene therapies that target refractory epilepsy and other disorders linked to neuronal hyperexcitability, with additional fundings.

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EpilepsyGTx noted that focal epilepsy refers to a group of conditions where seizures begin in a certain area of the brain.

When seizures continue despite trials of at least two well-tolerated and appropriately selected antiseizure medicines, the condition is classified as refractory.

EPY201, also known as AAV9-CAMK2A-EKC, uses an AAV serotype 9 (AAV9) capsid, a calcium/calmodulin-dependent protein kinase II alpha (CAMK2A) promoter, and an engineered naturally occurring form of the Kv1.1 potassium channel (EKC).

The therapy is delivered directly to the targeted seizure focus within the brain via intraparenchymal administration.

EpilepsyGTx CEO Nicolas Koebel said: “Refractory epilepsy is a devastating condition causing unpredictable and life-threatening seizures, and affecting millions of patients worldwide.

“Our novel gene therapy EPY201 delivered directly to the seizure focus has the potential to stop seizures with a single, minimally invasive administration. In doing so, it will change the way refractory epilepsy has been treated for decades. We are proud to have the support of such high calibre investors as we progress into clinical trials.”

Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.

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