KSQ Therapeutics emerged with $76 million in financing and a world-class executive team to advance the development of drugs empowered by the unique biological insights of its proprietary CRISPRomics™ drug discovery engine. With CRISPRomics, KSQ is pioneering high-confidence drug development utilizing a suite of CRISPR-based technologies that industrialize, for the first time, a high-throughput, systematic way to elucidate the precise function that each human gene plays across a multitude of diseases. Based on this deep and comprehensive approach, KSQ is advancing a pipeline of oncology and immunology drug development programs against novel therapeutic nodes to create a new set of disease-tailored medicines. KSQ was founded in 2015 by David Sabatini, MD, PhD, of the Whitehead Institute and MIT, William Hahn, MD, PhD, of the Broad Institute and Dana-Farber Cancer Institute, Jonathan Weissman, PhD, of UCSF, and Tim Wang, PhD, of MIT, along with founding investors Flagship Pioneering and Polaris Partners.

KSQ also announced today the appointment of David Meeker, MD, as Chief Executive Officer. Dr. Meeker most recently served as Head of the Specialty Care Business Unit of Sanofi-Genzyme and previously was Chief Executive Officer and President of Genzyme, a Sanofi Company. He brings more than 20 years of industry leadership experience, from leading-edge R&D programs to global operational roles.

“We have a clear goal with CRISPRomics: empower the drug development process to strategically focus on high-confidence, patient-tailored, novel drug candidates,” said Dr. Meeker. “There is a compelling need to improve the quality of drug targets and to identify patients most likely to respond because our industry and our health care systems are challenged by the sheer volume of potential new medicines. With our proprietary CRISPRomics engine, KSQ is positioned to play a leading role in shortening drug development timelines, increasing the rate at which meaningful medicines can reach patients, and ultimately, improving the sustainability of our health care systems.”

KSQ also announced Frank Stegmeier, PhD, as Chief Scientific Officer and George Golumbeski, PhD, as Executive Advisor and Board Member. Dr. Stegmeier is a leading expert in industrialized functional genomics who led Oncology Target Discovery at Novartis prior to joining KSQ. He has led KSQ’s platform development and R&D efforts since the company’s formation in 2015, demonstrating the early productivity of CRISPRomics. Dr. Golumbeski is a business development executive with an outstanding track record of success in the pharmaceutical industry. During his more than 25-year career, he has identified and evaluated innovative technologies and drug candidates from early-stage biotech companies and accelerated their drug innovation potential through creative partnerships.

“The human genome harbors more than 20,000 genes, however for most diseases we have yet to discern which genes represent the best therapeutic targets. For the first time, CRISPRomics allows us to systematically pinpoint the optimal nodal targets of disease with extraordinary precision and speed,” said Dr. Stegmeier. “The genome-scale insights that are possible with CRISPRomics are enabling KSQ to most efficiently focus our drug discovery efforts on the development of medicines with the greatest potential to intervene in disease and, therefore, impact the lives of patients.”

KSQ’s financing is led by Flagship Pioneering; Polaris Partners, ARCH Venture Partners, and Alexandria Equities also participated. Since the company’s formation in 2015, KSQ has deployed its CRISPRomics engine to complete the functional assessment of each gene in more than 600 tumor and immune models. The company has compiled new insights in disease biology to identify novel drug targets and new drug combinations designed to elicit maximal impact on disease. This approach has produced KSQ’s initial pipeline of drug development programs, and will serve as a product engine across a wide range of disease areas for the company and future collaborators. KSQ has grown to a team of more than 40 employees.

“KSQ is a pioneering company, unleashing high-confidence drug development for the first time — a step change in the way drugs are discovered and developed,” said David Berry, General Partner, Flagship Pioneering. “Led by proven industry leaders, KSQ is uniquely positioned to translate its breakthrough biological insights into therapeutics that can impact the core of many important diseases.”

High-Confidence Drug Development
Powered by CRISPRomics, KSQ has elucidated the function that each human gene plays in a multitude of diseases providing a unique and more comprehensive understanding of disease biology. The quality of these insights enables KSQ to identify a multitude of high-confidence, patient-tailored, novel programs for drug development and rapidly rule-out thousands of less relevant targets from the outset; thereby, focusing R&D investment on the development of medicines with the greatest potential to meaningfully impact the treatment of human disease.