We are a precision medicine company creating new genetically targeted medicines for people with severe genetic disorders. Beyond our internal programs, we also partner with pharmaceutical companies and with foundations in the discovery of new therapeutics. Our initial efforts focus on nervous system disorders including epilepsy, pain, and autism.
Our proprietary platform incorporates an unparalleled suite of technologies and capabilities that power our science:
- Clinical genetic sequencing and analysis through our CLIA-certified laboratory
- Cutting-edge stem cell technologies and genetically faithful human disease models that enable high fidelity prediction of drug efficacy
- All-optical electrophysiology technology, OptopatchTM, that facilitates the study of disease models with 10,000-fold greater cellular measurement throughput than industry standards
- Custom instrumentation that enables novel drug screening approaches and deep analytics to provide unprecedented insights
- Small molecule and antisense oligonucleotides (ASOs) design and discovery expertise that provides a breadth of opportunities for therapeutic intervention
- Partnerships will enable the ability to search and identify patients through existing partnerships for data purposes related to diagnostics, target identification, and clinical trial recruitment