Q-State Biosciences was birthed in 2014 when we realized that human stem cell models combined with all-optical electrophysiology and functional genomics enables us to find new drugs for devastating disorders of the nervous system and to find evidence that a particular drug may be effective for an individual patient.
One in ten people suffer from one of more than 7,000 known genetic diseases. Although most of these disorders are rare, genetic disease is, in the aggregate, common. Genetically mediated disorders of the nervous system are particularly damaging. Few of these diseases are well-understood or easily treated, making it difficult for patients and their physicians to know how to best move forward. The path toward treatment often begins with genetic sequencing. However, finding a likely disease-causing genetic variant in a patient’s genome is just the first step in the process. It is often necessary to characterize the functional impact of a genetic variant. Q-State can perform clinical grade diagnostic sequencing in its CLIA laboratory and can then use patient-derived stem cell models to evaluate the functional impact of a patient’s gene variation. The patient’s own cells are used to create a model of disease that can then be used to identify new or existing drugs that may be effective in vivo. Q-State co-founders Adam Cohen, Kevin Eggan, and I envisioned a company that could provide these services and offer to individuals a path forward in their search toward treatment for a serious condition.
Today, our suite of service offerings focus on diagnostic, functional, and interventional genomics, with the goal of not only making a diagnosis, but also uncovering new information about the disorder. We leverage our proprietary stem cell and OptopatchTM technologies with our expertise in antisense oligonucleotide (ASO) design as part of this platform and take pride in knowing that we are the first company to offer a full spectrum of precision medicine services for rare genetic central nervous system conditions. As part of our process, we work closely with leading investigators in major academic medical centers in well-designed clinical studies focused on single individuals or small cohorts of patients.
We hope our work ultimately provides individuals and their physicians with greater knowledge and improved outcomes for individuals living with a genetic condition.
Thank you for your interest in our company.
David Margulies, MD
Chairman, Q-State Biosciences