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EpilepsyGTx raises $33 million Series A to develop single dose gene therapy for focal refractory epilepsy

  • Investment from XGEN Venture, British Business Bank, and a global biopharmaceutical company will support first-in-human Phase 1/2a clinical trials for lead candidate EPY201 targeting focal refractory epilepsy (FRE)
  • Future financings to advance a pipeline of transformative gene therapies targeting refractory epilepsy and disorders of neuronal hyperexcitability

EpilepsyGTx, a biotechnology company focused on research and development of cutting-edge gene therapies to treat refractory epilepsy, today announced it has raised $33 million in a Series A financing to advance its lead programme EPY201 through Phase 1/2a clinical trials. The round included investment from XGEN Venture, the British Business Bank, and a global biopharmaceutical company.

The funding will enable the delivery of first-in-human Phase 1/2a clinical trials to establish the safety and efficacy of the company’s lead gene therapy programme EPY201 in a broad population of patients with focal refractory epilepsy (FRE). EPY201 is an adeno-associated viral (AAV) gene therapy designed to reduce neuronal hyperexcitability. It is delivered locally to the epileptogenic focus of the patient and therefore avoids the complications of systemic delivery. EPY201 offers FRE patients the prospect of seizure freedom in a single intervention without resection or ablation of brain tissue and without the chronic use of multiple antiseizure medicines.

EpilepsyGTx will also advance a pipeline of transformative gene therapies targeting refractory epilepsy and disorders of neuronal hyperexcitability with future financings.

Focal epilepsy describes a group of disorders in which patients experience seizures that arise from a specific part of the brain. If seizures persist despite trials of at least two tolerated and appropriately chosen antiseizure medicines, epilepsy is deemed refractory. FRE affects about 10 million patients worldwide1, including 2 million patients in the US, UK and EU. EPY201 offers the potential to eradicate seizures in patients with FRE with a single intervention, dramatically improving survival and quality of life.

Nicolas Koebel, Chief Executive Officer of EpilepsyGTx, commented: “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.”

Federica Draghi, Managing Partner of XGEN Venture, added: “EpilepsyGTx is pioneering a novel, locally administered gene therapy approach designed to achieve targeted modulation of epileptogenic brain regions. We believe that localized gene delivery offers a powerful avenue for durable and disease-modifying interventions in severe neurological disorders and are excited to support the company as EPY201 progresses toward clinical evaluation.”

EpilepsyGTx previously raised $10 million in pre-seed and seed funding led by the UCL Technology Fund, managed by AlbionVC in collaboration with UCL Business, the commercialisation company of UCL with participation from Zcube, the venture capital arm of Zambon.

Taylor Wessing LLP acted as legal advisor to EpilepsyGTx on the financing; A&O Shearman acted as legal advisor to XGEN Venture; Panmure Liberum Cambridge Capital acted as financial advisor.

1.      Vaughan et al (2019): https://thejns.org/doi/abs/10.3171/2018.3.JNS171722

About EpilepsyGTx

At EpilepsyGTx our mission is to make patients with refractory epilepsy seizure-free. We are developing a portfolio of cutting-edge gene therapies which are based on pioneering research from the UCL Queen Square Institute of Neurology. For more information, please visit: www.epilepsygtx.com.

About EPY201

EPY201 is an investigational gene therapy targeting focal refractory epilepsy. EPY201, also known as AAV9-CAMK2A-EKC, utilizes an AAV9 capsid, a CAMK2A promoter, and an engineered naturally occurring form of the Kv1.1 potassium channel (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.

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