UCB and Q-State Biosciences (Q-State) have entered into a multi-year therapeutics discovery collaboration. The joint programme will employ a precision-medicine approach to the development of novel therapeutics for epilepsy, and particularly genetically defined subtypes of childhood epilepsy that present with substantial morbidity, taking advantage of Q-State’s proprietary optogenetics platform.

Financial terms are not being disclosed, nor are the genetic targets that are the subject of the research. The programme includes research support, as well as prospective preclinical and clinical milestones and royalties.

The impact of genetic changes on neuronal function is not well understood, and targeted therapies for epilepsy patients suffering from such monogenic diseases are not currently available.

The collaboration calls for Q-State to build disease models using stem cell-derived neurons from epilepsy patients that can be employed with Q-State’s proprietary Optopatch technology. Optopatch permits the fine interrogation of neuronal networks by optical stimulation and recording and identifies the deficits in neuronal activity caused by genetic mutations. By coupling this platform with neuronal cell lines derived from epilepsy patients, the collaboration aims to characterize neuronal and network dysfunction with unparalleled precision. It also seeks to identify new therapies addressing the specific deficit unique to each epilepsy.

This alliance involves the application of a combined UCB/Q-State small molecule chemical library and collaborative medicinal chemistry effort. The goal is to identify and bring to clinical development therapeutics that can efficiently treat seizures in patients with genetic mutations.

“We are excited at the opportunity to make a real difference for such a devastating set of disorders, and to be doing it with the global leader in epilepsy therapeutics” said Jonathan Fleming, Q-State’s president and chief executive officer “Genetically defined indications such as these represent an ideal target for the Q-State approach, and we have high hopes that the combined efforts of our two companies will yield new treatments. These patients and their families are in desperate need of help, and innovative approaches based directly on these patients’ neurons are likely to be the best way to get it to them.”

“This collaboration is core to our genetics strategy at UCB which is to move from high unmet needs of specific patient sub-populations to providing transformational medicines to these sub populations using innovative science ”, said Ismail Kola, UCB’s chief scientific officer. “By coupling our epilepsy expertise with Q-State’s scientific excellence and technology platforms, we hope to accelerate the search of a new treatment for this severe disease.”