Gene therapy for severe epilepsy moves forward with EpilepsyGTx’s €28 million funding

Dec 10, 2025 - 18:00
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Gene therapy for severe epilepsy moves forward with EpilepsyGTx’s €28 million funding

EpilepsyGTx, a Cambridge-based BioTech startup focused on research and development of  gene therapies to treat refractory epilepsy, today announced it has raised €28 million ($33 million) in 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 (who invested €12 million (£10.5 million)), and a global biopharmaceutical company.

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.”

For context: European funding activity in 2025 shows sustained investor interest in neurological and CNS-focused therapeutics, with companies such as EG 427 securing €27 million to advance pinpoint genetic medicines for chronic neurological conditions, Aerska raising €17 million to develop RNAi therapeutics for brain diseases, TRIMTECH Therapeutics collecting €28.6 million to progress CNS-penetrant degrader therapies, and Augustine Therapeutics raising €77.7 million to develop treatments for neurodegenerative and neuromuscular diseases.

Together, these rounds represent around €150 million of new capital entering the sector this year.

Within this landscape, EpilepsyGTx’s Series A aligns with a wider European push towards precision neurology and advanced genetic or molecular technologies. The presence of another UK-based company in this group (TRIMTECH Therapeutics) highlights the country’s growing concentration of CNS-focused biotech R&D, situating EpilepsyGTx’s progress within an active and well-funded 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’s mission is to make patients with refractory epilepsy seizure-free. We are developing a portfolio of cutting-edge gene therapies which are based on research from the UCL Queen Square Institute of Neurology.

EpilepsyGTx raised €8.5 million ($10 million) last year 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.

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 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.

FRE describes a group of disorders in which patients experience seizures that arise from a specific part of the brain, leading to recurrent disabling events, cognitive impairment and psychosocial burden. This reportedly affects about 10 million patients worldwide, 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. This offers FRE patients the prospect of seizure freedom without resection or ablation of brain tissue and without the chronic use of multiple antiseizure medicines.

EpilepsyGTx will also advance a pipeline of gene therapies targeting refractory epilepsy and disorders of 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.

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