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UK spinout EpilepsyGTx gets $33M to target seizure hotspots with AAV gene therapy

EpilepsyGTx
Image credits: EpilepsyGTx

Millions of people living with focal refractory epilepsy have limited treatment options, and many continue to suffer seizures even after trying multiple medications. Focal epilepsy describes a group of disorders in which patients experience seizures that arise from a specific part of the brain.

If seizures continue after trying two appropriate and tolerated seizure medications, the epilepsy is considered refractory. The condition affects around 2 million people across the US, UK and EU, often leaving patients and families with few places to turn.

Kent-based biotechnology company EpilepsyGTx is working to change this by developing targeted gene therapies that directly treat the part of the brain causing seizures.

The UK company raised $33M in Series A funding round to advance its lead therapy, EPY201 through Phase 1/2a clinical trials.

The round included investment from XGEN Venture, the British Business Bank, and a global biopharmaceutical company.

EPY201 explained

Led by Nicolas Koebel, EpilepsyGTx was founded as a spin-out company with support from UCL’s commercialisation company, UCL Business Ltd (UCLB). The company is developing a portfolio of cutting-edge gene therapies which are based on pioneering research from the UCL Queen Square Institute of Neurology.

EPY201 is a gene therapy that targets and reduces overly active neurons in the brain.

It is given directly to the area causing seizures, which helps avoid problems that can arise from delivering treatment throughout the body.

For patients with focal refractory epilepsy (FRE), EPY201 offers a chance for seizure freedom with just one treatment, without needing to remove any brain tissue or rely on many different seizure medications.

Nicolas Koebel, Chief Executive Officer of EpilepsyGTx, comments, “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.”

What’s next?

The funds will be used to complete preclinical studies for the company’s lead gene therapy program, EPY201, and to prepare a groundbreaking first-in-human Phase 1/2a clinical study of EPY201 in focal refractory epilepsy.

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 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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