EpilepsyGTx Secures $33 Million Series A to Advance Single-Dose Gene Therapy for Focal Refractory Epilepsy
核心洞察
EpilepsyGTx (搜索) raised $33 million in Series A funding from XGEN Venture (搜索), British Business Bank (搜索), and a global biopharmaceutical company to advance EPY201 through Phase 1/2a clinical trials.
EPY201 is an adeno-associated viral gene therapy designed to reduce neuronal hyperexcitability (搜索) through local delivery to the epileptogenic focus (搜索), avoiding systemic complications.
The therapy targets focal refractory epilepsy (搜索) affecting approximately 10 million patients worldwide, offering potential seizure freedom with a single intervention without brain tissue resection.
EpilepsyGTx (搜索), a biotechnology company developing gene therapies for refractory epilepsy (搜索), announced it has secured $33 million in Series A financing to advance its lead program EPY201 through Phase 1/2a clinical trials. The funding round included investment from XGEN Venture (搜索), the British Business Bank (搜索), and a global biopharmaceutical company.
Novel Gene Therapy Approach
EPY201 is an adeno-associated viral (AAV) gene therapy designed to reduce neuronal hyperexcitability (搜索) through local delivery to the epileptogenic focus (搜索) of patients. This targeted approach avoids the complications associated with systemic delivery while offering patients the prospect of seizure freedom through a single intervention without requiring brain tissue resection or ablation.
The therapy represents a departure from traditional treatment approaches by eliminating the need for chronic use of multiple antiseizure medicines. According to Nicolas Koebel, Chief Executive Officer of EpilepsyGTx (搜索), "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 (搜索) has been treated for decades."
Addressing Significant Unmet Medical Need
Focal refractory epilepsy (搜索) affects approximately 10 million patients worldwide, including 2 million patients in the US, UK, and EU. The condition is defined by seizures that persist despite trials of at least two tolerated and appropriately chosen antiseizure medicines. These seizures arise from a specific part of the brain and represent a devastating condition causing unpredictable and life-threatening episodes.
EPY201 offers the potential to eradicate seizures in patients with focal refractory epilepsy (搜索) through a single intervention, which could dramatically improve survival and quality of life for affected patients.
Clinical Development Plans
The Series A funding will enable EpilepsyGTx (搜索) to conduct first-in-human Phase 1/2a clinical trials to establish the safety and efficacy of EPY201 in a broad population of patients with focal refractory epilepsy (搜索). The company plans to use future financings to advance a pipeline of transformative gene therapies targeting refractory epilepsy (搜索) and disorders of neuronal hyperexcitability (搜索).
Federica Draghi, Managing Partner of XGEN Venture (搜索), commented on the investment: "EpilepsyGTx (搜索) 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."
Company Background
EpilepsyGTx (搜索) previously raised $10 million in pre-seed and seed funding led by the UCL Technology Fund (搜索), managed by AlbionVC (搜索) in collaboration with UCL Business, with participation from Zcube (搜索), the venture capital arm of Zambon (搜索). The company received legal advisory services from Taylor Wessing LLP for the Series A financing, while A&O Shearman advised XGEN Venture (搜索) and Panmure Liberum Cambridge Capital served as financial advisor.