£24.8m Series A backs first-in-human AAV gene therapy trials to stop seizures
This article covers EpilepsyGTx, a biotech startup, which has raised £24.8m in a Series A to advance EPY201, a locally delivered AAV gene therapy intended as a single-dose treatment for focal refractory epilepsy. The funding will support first-in-human Phase 1/2a trials and targets patients with focal refractory epilepsy, with implications for clinicians and investors in neurologics and gene therapy.
EpilepsyGTx, a biotech startup, has raised £24.8m ($33m) in a Series A to advance EPY201, a locally delivered AAV gene therapy intended as a single-dose treatment for focal refractory epilepsy (FRE). The funding will support first-in-human Phase 1/2a trials and the company says it plans further rounds to expand a pipeline addressing disorders of neuronal hyperexcitability.
Why it matters
Focal refractory epilepsy describes seizures originating from a specific brain region that do not respond to at least two appropriately chosen antiseizure medicines. The condition affects an estimated 10 million people worldwide, including around 2 million across the US, UK and EU. Current options for patients who are not medication-responsive include surgical resection or ablation; a safe, locally delivered gene therapy that reduces hyperexcitability could offer an alternative that avoids removal of brain tissue and long-term polypharmacy.
The scale of unmet need and the prospect of a one-time intervention make EPY201 notable for neurologics and gene therapy investors, and for clinical teams looking for new interventions in drug-resistant epilepsy.
Inside the product
EPY201 is described as an adeno-associated viral (AAV) gene therapy designed to reduce neuronal hyperexcitability. It is intended for local delivery to the epileptogenic focus, which the company says should limit systemic exposure compared with whole-body dosing. EpilepsyGTx positions EPY201 as a potential single-administration approach aimed at reducing or eliminating seizures without surgical resection.
The Series A is specifically earmarked to enable first-in-human Phase 1/2a trials that will assess safety and efficacy across a broad population of patients with focal refractory epilepsy. EpilepsyGTx also says it will use future financings to advance a broader pipeline targeting refractory epilepsy and related neuronal hyperexcitability disorders.
Who are the investors?
The round included XGEN Venture, the British Business Bank, and an unnamed global biopharmaceutical company. EpilepsyGTx previously raised around £7.5m ($10m) in pre-seed and seed funding led by the UCL Technology Fund, managed by AlbionVC in collaboration with UCL Business, with participation from Zcube, the venture arm of Zambon. Those earlier investors supported the company’s formation and preclinical work.
Taylor Wessing LLP acted as legal adviser to EpilepsyGTx on the Series A. A&O Shearman advised XGEN Venture. Panmure Liberum Cambridge Capital acted as financial adviser.
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.
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Founder perspective
Nicolas Koebel, Chief Executive Officer of EpilepsyGTx, 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.
Koebel’s comment underscores the company’s clinical focus and the immediate aim of translating preclinical findings into early human studies. Leadership and prior academic partners — notably UCL Business and the UCL Technology Fund — point to an academic spinout pathway common in UK biotech.
The bigger picture
The financing highlights continued investor interest in neurological gene therapies within the UK and Europe, a space where clinical complexity, manufacturing constraints and regulatory scrutiny remain real hurdles. Public involvement through the British Business Bank is notable; it reflects a growing trend of blended public and private funding for translational biotech that aims to move complex biologics from lab to clinic.
EpilepsyGTx’s path will test several ecosystem factors: recruitment for a defined patient population, validation of a locally applied AAV approach, and the ability to scale manufacturing for CNS-directed gene therapies. If EPY201 reaches its clinical endpoints, it could shift treatment paradigms for some patients with refractory focal epilepsy and attract further attention from academic centres and investors active in gene therapy and neurology.
The deal also ties into a wider pattern of UK academic spinouts and specialist funds targeting high-barrier therapeutic areas. Success or setbacks in this programme will feed into UK and European conversations around clinical translation, regulatory frameworks for gene therapy, and the kinds of public-private funding mixes needed to bring complex biologics into the clinic.
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