Therapy Design

Leveraging internal technologies for enhanced drug discovery

 

In the drug discovery process, some diseases are best evaluated therapeutically with human models, including derived neurons. At Q-State, we excel in the design and discovery of genetically-targeted medicines for these kinds of nervous system disorders. Our platform has the greatest power for diseases where one can expect a cell-autonomous or synaptic phenotype. We primarily focus on antisense oligonucleotides (ASOs) and small molecules, while also building early-stage efforts in gene therapies.

Genetic Screens

In our genetic screens, we ascertain the effects of therapeutic candidates on gene expression and specialized functional studies. This approach is crucial for ASOs and gene therapies that directly impact how a protein is made.

Phenotypic Screens

In our phenotypic screens, we search for the most robust functional signature conferred by a disease model, and then apply therapeutics to try to correct the specific cellular aberrations. This approach is especially valuable in the study of a complex target or genetic disorder where identification of a consistent pathological phenotype is particularly challenging. Our proprietary OptopatchTM technologies are some of the most powerful tools for phenotypic screens because they provide high-content data from which we can identify the most salient cellular disease markers. In therapeutic screens, our robust analytics quantify drug response to these parameters in both individual and collective assessments.