Our panel of models allow for more accurate prediction of human responses, reduce the risk of late-stage failures and provide a thorough understanding of drug mechanisms.

Figure 1. Visual summary of primary neuronal cells used in electrophysiological recordings. A. Rodent dorsal root ganglion (DRG) neuron preparation for manual patch-clamp studies and a bright-field image of dissociated cells seeded on a coverslip. B. DRG action potential responses to low and high prolonged current injections.

A range of assay methodologies is vital in drug discovery because it ensures a thorough evaluation of drug candidates from multiple perspectives. This approach increases the likelihood of identifying effective and safe drugs, reduces the risk of late-stage failures, and supports the overall success of the drug development process. By leveraging diverse assays, researchers can optimize drug candidates, address challenges as they arise, and ultimately bring better therapies to market. Examples of the assays we offer include:

Screening of your compounds in a neuroscience setting is primarily driven by the fact that they have been designed to modulate CNS or peripheral nervous system targets and signalling pathways, including but not limited to ion channels. Our neuroscience experts can carry out this screening using heterologous cell lines expressing human or rodent neuronal proteins, as well as offering human iPSC-derived neurons and primary rodent cells.

Get insight to selectivity and safety profiling of lead compounds and IND candidates, either as part of an existing screening cascade or because in vivo testing revealed a neurological signal. We have developed and validated an industry-standard rat cortical CNS neuron assay that can reliably detect the seizure-inducing liability of a wide range of reference compounds (as well as serve as a model to profile anti-epileptic drugs). We also have deep expertise with rodent dorsal root ganglion (DRG) sensory neurons which can be used to assess peripheral neurotoxicity.

Eliana is a two-year-old from Canada with a de novo mutation (V434L) in her KCNC1 gene which encodes for the Kv3.1 channel in central nervous system neurons such as cerebellar neurons and GABAergic interneurons. The mutation manifests as a variety of neurological disorders which can include myoclonic epilepsy and ataxia, developmental epileptic encephalopathy (DEE), or hypotonia, depending on the specific variant.

The KCNC1 Foundation collaborated with Perlara, who approached Metrion Biosciences, where manual and automated (Qube) patch-clamp techniques and Fluorescent Imaging Plate Reader (FLIPR) high-throughput screens (HTS) against the mutant channel were performed to identify hit compounds.