EpilepsyGTx

EpilepsyGTx is a UK-based, late preclinical-stage biotechnology company founded in 2021, operating at the forefront of genetic medicine to address focal refractory epilepsy (FRE). The company was established as a spinout from University College London (UCL) with support from UCL's commercialisation arm, UCL Business Ltd (UCLB), building upon foundational research from the esteemed UCL Queen Square Institute of Neurology. This research was cultivated by a team of scientists and clinicians, including Professor Gabriele Lignani, who brought his biotechnology and neurophysiology expertise to UCL's epilepsy program in 2014 and serves as a co-founder and scientific advisor. His journey reflects a crucial shift from basic research to a translational approach, emphasizing early-stage patenting and clinical application.

The company's core business is the development of a portfolio of advanced gene therapies designed to offer a definitive treatment for patients whose seizures are not controlled by conventional antiseizure medications. This market includes an estimated two million people in the U.S., UK, and EU. In June 2024, EpilepsyGTx secured $10 million in a seed funding round led by the UCL Technology Fund and Health Technology Holding. This capital is allocated to advancing its lead candidate, EPY201, through the final stages of preclinical studies and preparing for a first-in-human Phase 1/2a clinical trial.

EpilepsyGTx's lead product, EPY201, is an investigational gene therapy that targets the specific area of the brain where seizures originate. It utilizes an AAV9 viral vector to deliver an engineered potassium channel gene (Kv1.1) under the control of a CAMK2A promoter. This is designed to make the neurons in the seizure focus less excitable, thereby preventing seizures. The treatment involves a single, minimally invasive surgical procedure where a small volume of the gene therapy is delivered directly into the seizure focus. This highly targeted intraparenchymal administration avoids the widespread side effects associated with oral medications that affect the entire brain and body. Furthermore, unlike resective surgery or laser ablation, this method does not require the permanent destruction of brain tissue.

Keywords: gene therapy, focal refractory epilepsy, neurology, biotechnology, AAV9 vector, potassium channel, preclinical studies, clinical trials, UCL spinout, seizure treatment, neuro-therapeutics, translational neuroscience, intraparenchymal administration, EPY201, genetic medicine, neurological disorders, antiseizure therapy, UCL Queen Square Institute of Neurology, targeted drug delivery, central nervous system disorders