Cell therapy weekly: gene therapy for epilepsy
This week: EpilepsyGTx (London, UK) raised US$33 million in Series A funding to advance its gene therapy candidate for focal refractory epilepsy to clinical trials, Cell and Gene Therapy Catapult (CGT Catapult; London, UK) established a consortium to develop universal tests to increase the safety and efficiency of stem cell therapies, and Syntax Bio (IL, USA) received an award under Breakthrough T1D’s (NY, USA) Industry Discovery and Development Partnership to develop a pancreatic beta cell therapy for type 1 diabetes.
The news highlights:
- $33 million to develop an epilepsy gene therapy
- Universal safety test for stem cell therapies
- Advancing pancreatic beta cell therapy
$33 million to develop an epilepsy gene therapy
EpilepsyGTx has secured $33 million in Series A funding to advance its lead candidate, EPY201, through Phase I/IIa clinical trials to evaluate its safety and efficacy in patients with focal refractory epilepsy.
EPY201 is an AAV gene therapy designed to reduce neuronal hyperexcitability by targeting the epileptogenic focus directly, avoiding systemic delivery complications. It offers the potential for seizure freedom through a single intervention, eliminating the need for brain tissue resection, ablation or chronic antiseizure medications.
CEO of EpilepsyGTx Nicolas Koebel commented: “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.”
The financing round included contributions from XGEN Venture (Milan, Italy), the British Business Bank (Sheffield, UK) and an unnamed global biopharmaceutical company.
Universal safety test for stem cell therapies
CGT Catapult has established ReCell, a consortium of 13 partners aiming to develop universal safety tests to detect undifferentiated residual pluripotent stem cells (PSCs) in therapy products. These residual PSCs pose a risk of uncontrolled growth and tumor formation, which can compromise product purity and patient safety.
The consortium will evaluate two droplet digital PCR methods to detect residual PSCs across eight different therapy products, including neuronal, cardiac and hematological cell types. One test was developed by CGT Catapult in collaboration with the Health and Environmental Sciences Institute (DC, USA), while the other is a commercially available solution created by Sistemic (Glasgow, UK).
The goal of the consortium is to demonstrate how universal tests for residual PSCs can be applied across various therapy products, reducing costs, accelerating development timelines, and ensuring patient safety. By standardizing the testing process, therapy developers can avoid duplication, streamline development and bring life-changing therapies to patients more quickly.
Advancing pancreatic beta cell therapy
Syntax Bio has been awarded up to $856,250 by Breakthrough T1D, a leading global organization focused on type 1 diabetes research and advocacy. This funding, provided through Breakthrough T1D’s Industry Discovery and Development Partnership program, will support Syntax Bio’s efforts to develop a pancreatic beta cell therapy for type 1 diabetes using its proprietary Cellgorithm platform technology.
The Cellgorithm platform is a CRISPR-based system that automates the process of gene activation, guiding precursor cells to mature into specific cell types, such as insulin-producing pancreatic beta cells.
“Pancreatic cell therapy has been constrained for decades by the inability to consistently produce functional beta cells at scale,” said Ryan Clarke, co-founder of Syntax Bio. “Syntax’s Cellgorithms are designed to solve that problem by programming differentiation like software, with sequential precision, repeatability, and consistent quality. We are grateful for Breakthrough T1D’s support and collaboration, which strengthens our ability to advance this work toward a therapy that can make a meaningful difference for people with type 1 diabetes.”