EpilepsyGTx Raises $33 Million Series A To Develop Single Dose Gene Therapy For Focal Refractory Epilepsy

The round was backed by XGEN Venture, the British Business Bank which contributed €12 million (£10.5 million) and a global biopharmaceutical partner.

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

European funding activity in 2025 continues to show strong investor interest in neurological and CNS-focused therapeutics. Companies such as EG 427 (€27M for precision genetic medicines), Aerska (€17M for RNAi therapies targeting brain diseases), TRIMTECH Therapeutics (€28.6M for CNS-penetrant degraders), and Augustine Therapeutics (€77.7M for neurodegenerative and neuromuscular treatments) have collectively raised roughly €150 million this year.

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Against this backdrop, EpilepsyGTx’s Series A fits squarely within Europe’s growing momentum toward precision neurology and advanced genetic or molecular technologies. With another UK-based player TRIMTECH Therapeutics featured among major rounds the UK is emerging as a key hub for CNS-focused biotech R&D, positioning EpilepsyGTx within a dynamic and well-capitalised innovation ecosystem.

Leandros Kalisperas, Chief Investment Officer, British Business Bank, adds: “The UK’s life sciences sector continues to produce world-class innovation, and the Bank is committed to building that momentum. Through cornerstone commitments to leading sector funds and by being the UK’s most active late-stage investor, the Bank is helping IP-rich companies access the capital they need to scale in the UK.”

Founded in 2021, EpilepsyGTx is dedicated to developing gene therapies that aim to make patients with refractory epilepsy seizure-free. Its therapeutic portfolio is built on pioneering research from the UCL Queen Square Institute of Neurology.

The company previously raised €8.5 million ($10 million) in pre-Seed and Seed funding, led by the UCL Technology Fund managed by AlbionVC in partnership with UCL Business with additional participation from Zcube, the venture arm of Zambon.

Federica Draghi, Managing Partner of XGEN Venture, adds: “EpilepsyGTx is pioneering a novel, locally administered gene therapy approach designed to achieve targeted modulation of epileptogenic brain regions. We believe that localiwed 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.”

The new funding will enable EpilepsyGTx to initiate first-in-human Phase 1/2a clinical trials to assess the safety and efficacy of its lead 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 and is administered directly to the patient’s epileptogenic focus, avoiding the complications associated with systemic delivery.

This single-intervention approach offers the potential for seizure freedom without brain tissue resection, ablation or long-term reliance on multiple antiseizure medications. FRE encompasses disorders in which seizures originate from a specific brain region, leading to recurrent disabling episodes, cognitive decline and significant psychosocial burden. An estimated 10 million people worldwide are affected, including around 2 million across the US, UK, and EU.

EPY201 has the potential to eliminate seizures and dramatically improve survival and quality of life. In parallel, EpilepsyGTx will continue advancing a broader pipeline of gene therapies targeting refractory epilepsy and other disorders driven by neuronal hyperexcitability.

Carmine Circelli, Investment Director, British Business Bank, says: “Epilepsy GTx is built on world leading expertise from UCL, pursuing an innovative approach to modulating neurons in epilepsy and backed by compelling preclinical data. If this mechanism is validated in clinical trials, the company has the potential to be category-defining in epilepsy treatment, addressing substantial unmet patient need and presenting a significant commercial opportunity.”

About EpilepsyGTx

EpilepsyGTx is a late preclinical-stage biotech developing advanced gene therapies to transform the treatment of focal refractory epilepsy (FRE). Building on pioneering neurological research the company’s mission is to make FRE patients seizure-free through precise targeted therapies that address the underlying causes of neuronal hyperexcitability.