HORIZON-EIC-2023-PATHFINDEROPEN-01-01: Hungarian biotech SME is looking for partners in the field of electrodes applied in neurosurgery or in vivo neuronal transfection/transduction for the development of chemogenetic therapeutics for various CNS diseases

Short summary Hungarian biotech SME is developing a novel class of CNS therapies using chemogenetics. The company would like to apply for EIC Pathfinder Open 2023 call. The therapy will consist of a single microinjection and a subsequent pharmacotherapy. It is looking for partner companies manufacturing and developing electrodes to be applied in neurosurgery or devices for microinjection or electroporation. Companies developing solutions for in vivo neuronal transduction or transfection are also awaited.

Full description Since neuronal receptors are usually expressed in various central nervous system (CNS) regions, even highly selective CNS pharmacotherapies show subideal safety/efficacy profile. There are several CNS disorders that do not have acceptable pharmacotherapy, despite the fact that their etiology is quite well known. In academic CNS research, the activity of predifined neurons can be modified in a reliable and repeatable manner using chemogenetics. In clinical practice, there are only two solutions providing targeted effect on predefined neuronal populations: deep brain stimulation (DBS) and local lesions. Although DBS offers a fine-tuned effect, it requires the implantation of an electrode and carrying a device. In contrast, chemogenetics requires a single microinjection and the effect can subsequently be exerted with pharmacotherapy, also providing the opportunity to fine-tune the effect size. The aim of the HORIZON-EIC-2023-PATHFINDEROPEN-01-01 project is to develop a chemogenetic tool that can be registered as Cell and Gene Therapy or Advanced Therapy Medicinal Product. This tool could be the first member of a new therapeutic class providing precise targeting of neuronal populations defined by their spatial and neurochemical attributes. It would offer safe and effective treatment for various debilitating CNS diseases, such as Parkinson’s, epilepsy, chronic pain. The leader of the future consortium, the Hungarian SME, possesses sound expertise in modification of peptides/proteins of natural origin using its own platform. This platform combines directed evolution, next-generation sequencing and in silico modeling. The grant application is yet to be prepared. The EOI deadline to contact to leader of the consortium is 17 February 2023. The following partners are invited: - Companies or research institutes manufacturing and developing electrodes to be applied in neurosurgery or devices for microinjection or electroporation. The task of the partner would be to develop a tool that can be used for precise targeting of microinjection with detection of electrophysiological currents and for performing the microinjection. - Companies or research institutes developing solutions for in vivo neuronal transduction or transfection. The task of the partner would be to develop a solution for the safe an effective delivery of the genetic information to neurons.

Advantages and innovations

• - This project is dedicated to creating a new class of therapeutic product for debilitating CNS diseases.
• - It would provide a safe and effective treatment for various CNS diseases without an implanted device.
• - No implanted device needed
• - Effect can be fine-tuned individually or even stopped by modifying the dose and administration frequency of the pharmacotherapy
• - It can provide precisely targeted effect on predefined neuronal populations, resulting in exceptional safety / efficacy profile.
• - Conventional CNS pharmacotherapy exerts effect on various neuronal populations, resulting in poor safety/efficacy profile
• - Deep brain stimulation requires an implanted electrode, not able to make distinction between neurochemically different neuronal populations and can’t be applied in spinal cord
• - Lesion surgeries are not adjustable or reversable, can’t be used for excitation and not able to make distinction between neurochemically different neuronal populations

Contact / source: NEXT EEN Widgets (europa.eu)