EpilepsyGTx lands €28M to test one-shot gene therapy for severe epilepsy

EpilepsyGTx lands €28M to test one-shot gene therapy for severe epilepsy - Professional coverage

According to EU-Startups, EpilepsyGTx, a Cambridge-based biotech startup, has raised €28 million (about $33 million) in a Series A funding round. The investment, which includes €12 million from the British Business Bank and backing from XGEN Venture and a global biopharma company, will fund Phase 1/2a clinical trials for its lead gene therapy program, EPY201. CEO Nicolas Koebel stated the novel therapy is designed to be delivered directly to the seizure focus in the brain with a single, minimally invasive administration. Founded in 2021 based on research from the UCL Queen Square Institute of Neurology, the company’s mission is to make patients with refractory epilepsy seizure-free. This latest round follows an €8.5 million seed funding in 2024 led by the UCL Technology Fund.

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The one-shot ambition

Here’s the thing about refractory epilepsy: current treatments are a constant battle. Patients often juggle multiple antiseizure medications that come with brutal side effects and, by definition, don’t fully work. Surgical options exist, but they involve removing or destroying brain tissue. That’s a massive, irreversible step. What EpilepsyGTx is proposing with EPY201 is fundamentally different. It’s a localized gene therapy. Basically, they use a viral vector (an AAV) to deliver genetic instructions directly to the specific, misfiring part of the brain. The goal? Calm the hyperexcitability of those neurons with a single intervention. No daily pills, no brain resection. If it works as hoped, it could be genuinely disease-modifying. That’s a huge “if,” of course, but the potential impact on quality of life is staggering for the roughly 10 million patients worldwide with focal refractory epilepsy.

Part of a bigger neuro wave

This funding isn’t happening in a vacuum. The article points out that EpilepsyGTx is riding a significant wave of European investment into neurological and CNS-focused therapeutics. We’re talking about €150 million flowing into similar companies just this year. There’s EG 427, Aerska, TRIMTECH Therapeutics, and Augustine Therapeutics all raising big rounds. So what’s the trend? It’s a concerted push into precision neurology. For decades, treating brain diseases has been notoriously hard—the blood-brain barrier is a formidable gatekeeper, and systemic drugs often cause widespread side effects. The new wave, which includes gene therapy and RNAi, is about ultra-targeted interventions. And the UK, with companies like EpilepsyGTx and TRIMTECH, seems to be establishing itself as a real hub for this kind of R&D. The British Business Bank’s investment is a clear signal they want to keep that momentum going.

The long road ahead

Now, let’s be real. A €28 million Series A is substantial, but in the world of clinical-stage biotech, especially for gene therapy, it’s a fuel tank for the first leg of a very long journey. This money gets EPY201 into Phase 1/2a trials to establish initial safety and look for signs of efficacy. That’s a critical milestone, but it’s just the beginning. Gene therapy for the brain carries unique challenges—delivery precision, immune response, long-term expression. The company mentions advancing a broader pipeline, which is smart, but all resources will be laser-focused on this lead program for now. The presence of an unnamed global biopharmaceutical company in the round is interesting, too. It could be a pure financial play, or it might hint at future partnership potential if the early data looks promising. For patients and the field, all eyes will be on that first-in-human data. Can a single injection really silence a seizure focus? We’re about to start finding out.

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