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EpilepsyGTx

Company Overview

A UK-based gene therapy spinout from UCL's Queen Square Institute of Neurology targeting focal refractory epilepsy with a single-dose AAV9 approach aimed at patients for whom all antiseizure drugs have failed. EpilepsyGTx is building around the premise that focal refractory epilepsy — where seizures are drug-resistant and often ineligible for resection — represents a defined, underserved population suited to a precise, one-time genetic intervention. The company's pipeline centers on neuronal hyperexcitability disorders broadly, with EPY201 as the lead asset advancing toward first-in-human studies.

Headquarters and Global Presence

EpilepsyGTx is headquartered at Camburgh House, Canterbury, Kent, in the United Kingdom. The company's scientific roots lie at UCL's Queen Square Institute of Neurology in London, one of Europe's leading centers for neurological research, which underpins both its academic credibility and its access to clinical networks in epilepsy.

Founding and History

EpilepsyGTx was founded as a spinout from the UCL Queen Square Institute of Neurology. In December 2025, the company closed a $33 million Series A financing round to fund the development of EPY201 toward clinical trials. The company has since moved rapidly to build out its leadership team, appointing a Chief Scientific Officer in April 2026 and a Chief Medical Officer in July 2026.

Therapy Areas and Focus

The company is focused exclusively on refractory epilepsy — specifically focal seizures that persist despite multiple lines of antiseizure medication — and broader neuronal hyperexcitability disorders. Around one-third of people with epilepsy do not achieve seizure control with drugs, and many are not candidates for surgical resection of the seizure focus. Gene therapy applied directly to the seizure focus offers a mechanistically distinct option: not suppressing symptoms systemically, but correcting the electrical dysfunction at source.

Technology Platforms and Modalities

EPY201 is built on an AAV9 viral vector chassis, selected for its efficient neuronal transduction. The construct uses a CAMK2A promoter to restrict expression to excitatory neurons — the cell population most implicated in seizure generation — and delivers an engineered Kv1.1 potassium channel (the EKC element) that acts to dampen neuronal hyperexcitability. Administration is by intraparenchymal delivery directly to the seizure focus, a surgical approach analogous to electrode placement for deep brain stimulation and designed to limit off-target expression. The single-dose ambition is commercially and clinically important: unlike chronic drug regimens, a one-time intervention removes adherence as a variable.

Key Pipeline and Programs

EPY201 — formally designated AAV9-CAMK2A-EKC — is EpilepsyGTx's sole disclosed pipeline asset and the company's entire near-term clinical focus. It is an AAV9 gene therapy encoding an engineered Kv1.1 potassium channel under the neuron-selective CAMK2A promoter, delivered intraparenchymally to the seizure focus in patients with focal refractory epilepsy. The program is progressing toward a Phase I/IIa first-in-human trial; no trial registration number has yet been disclosed in available sources. The target patient population — adults with focal seizures refractory to multiple antiseizure drugs and not amenable to resective surgery — represents a group with very limited remaining options, which supports both the regulatory rationale and the commercial case for a single-dose product.

Recent Developments

In December 2025, EpilepsyGTx raised a $33 million Series A round backed by XGEN Venture, the British Business Bank, and an undisclosed global biopharmaceutical company, providing the capital base to carry EPY201 into clinical trials. Professor Dimitri Kullmann was appointed Chief Scientific Officer in April 2026, bringing the academic expertise behind the UCL research program formally into the executive team. In July 2026, Dr Teresa Nunes joined as Chief Medical Officer, tasked specifically with leading EPY201 through its first-in-human studies — a hire that signals the company's transition from platform-building to active clinical execution.

Key Personnel

Nicolas Koebel serves as Chief Executive Officer, leading the company's strategic direction. Professor Dimitri Kullmann serves as Chief Scientific Officer, appointed April 2026; Kullmann is the UCL neuroscientist whose research on potassium channel gene therapy in focal epilepsy forms the scientific foundation of EPY201. Dr Teresa Nunes serves as Chief Medical Officer, appointed July 2026; she brings over twenty years of CNS and epilepsy drug development experience, including leading the approval of antiseizure medicine Zebinix at Bial, and most recently headed medical and pharmacovigilance functions at Allucent.

Strategic Partnerships

The $33 million Series A closed in December 2025 was co-led by XGEN Venture and supported by the British Business Bank, alongside participation from an unnamed global biopharmaceutical company whose involvement hints at potential downstream licensing interest in the program. The UCL Queen Square Institute of Neurology remains the foundational academic partner, providing the research heritage underpinning EPY201's design.

FAQ Section

Focal refractory epilepsy is defined by treatment failure — patients have typically exhausted multiple antiseizure drugs and are not surgical candidates, leaving them with few options and high seizure burden. The focal nature of the condition is also a practical advantage for gene therapy: a single anatomically defined seizure focus can be targeted by direct intraparenchymal delivery, limiting the need for widespread CNS distribution. This precision narrows both the safety profile challenge and the regulatory path.

Potassium channels are key regulators of neuronal excitability — when they are dysfunctional or underexpressed, neurons fire aberrantly and seizures propagate. Kv1.1, a voltage-gated potassium channel, is implicated in controlling action potential firing rates in excitatory neurons. EpilepsyGTx's engineered Kv1.1 (the EKC component of EPY201) is designed to restore inhibitory tone specifically in the seizure-generating tissue, addressing the pathophysiology rather than blunting neural activity system-wide as conventional antiseizure drugs do.

EPY201 combines three specificity layers: an AAV9 vector for neuronal tropism, a CAMK2A promoter that restricts expression to excitatory neurons (the seizure drivers), and intraparenchymal delivery directly to the seizure focus. The CAMK2A promoter element is particularly important — it avoids off-target expression in inhibitory neurons or non-neural cells, reducing the risk of disrupting normal circuit function. The single-dose design is also a meaningful clinical differentiator against lifelong drug regimens in a population already defined by drug failure.

EPY201 is pre-clinical at present, progressing toward a Phase I/IIa first-in-human trial. The appointment of Dr Teresa Nunes as CMO in July 2026, specifically tasked with steering EPY201 into that study, indicates the clinical submission and site activation work is now underway. No trial start date or regulatory filing timeline has been publicly disclosed, but the $33 million Series A raised in December 2025 is intended to fund this transition into clinical stage.

The company's stated focus covers focal refractory epilepsy and neuronal hyperexcitability disorders more broadly, suggesting the CAMK2A-EKC platform could have applications beyond the lead indication. At present, EPY201 for focal refractory epilepsy is the only disclosed program. The underlying UCL research heritage in potassium channel biology and cortical hyperexcitability may point to future programs in other channelopathy-related conditions, though no additional candidates have been announced.

EpilepsyGTx is a pre-clinical stage company at the point of transition into first-in-human studies, with its Series A financing in place and its clinical leadership team now assembled. The critical near-term milestones are Phase I/IIa trial initiation and initial safety and tolerability data from the first human subjects treated with EPY201. The company is at a stage where execution risk — particularly around surgical delivery logistics and patient selection — is the dominant variable.

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