~ Proprietary CRISPRomics discovery engine has yielded 12 oncology programs in just 12 months ~
Advances in genome editing have dramatically transformed the scientific landscape and opened up significant new avenues for drug discovery and disease treatment. The editing tools known as CRISPR (clustered regularly interspaced short palindromic repeats) and a successor known as CRISPR/Cas9 (CRISPR-associated protein 9) have revolutionized the science and enabled faster, more accurate and less expensive methods of editing the human genome. CRISPR/Cas9, adapted from a naturally occurring editing system found in bacteria, involves a short RNA sequence designed to bind to a specific region of target DNA in mammals, e.g., in a virus, cutting it apart and disabling its function. (We would direct readers who desire a more thorough discussion of advances in genome editing to peruse an excellent overview by Rajat M. Gupta, MD, and Kiran Musunuru, MD, PhD, MPH, of the Department of Stem Cell and Regenerative Biology at Harvard and the Division of Cardiovascular Medicine at Brigham and Women’s Hospital.
Cambridge, Massachusetts, based KSQ Therapeutics has developed a proprietary drug discovery engine called CRISPRomics that, using CRISPR/Cas9 tools, has yielded 12 product programs in 12 months across three oncology drug categories: adoptive T-cell therapies (infusion of tumor-specific T cells), immuno-oncology monotherapies and targeted therapies. The company announced today that it has secured $80 million in Series C financing from new investors Baillie Gifford, Cowen Healthcare Investments, Invus and Lilly Asia Ventures, and including full participation from its founding investors Flagship Pioneering and Polaris Partners as well as from existing investors ARCH Venture Partners and Alexandria Equities.
KSQ announced itself just one year ago with a $76 million raise. Proceeds from this Series C will be used to advance the company’s first drug program into the clinic within the next 18 months and up to three additional oncology drug programs into investigation new drug (IND)-enabling studies. The company’s first drug program is a modified adoptive T-cell immunotherapy which has shown efficacy in multiple animal models of PD-1 resistance. The inhibition of PD-1 (programmed cell death protein 1) has been shown to increase the ability of T cells to kill cancer cells.
Source: KSQ Therapeutics
KSQ has used its CRISPRomics technology to identify relevant therapeutic targets and biomarkers while ruling out thousands of others:
- Tumor-genome platform. Interrogated the function of all 20,000 human genes across more than 600 cancer models
- T-cell genome platform. Comprehensively mined the function of all genes in the T cell in vivo, enabling identification of adoptive T-cell enabling targets and the next generation of monotherapy targets in immuno-oncology.
KSQ has made remarkable progress in the past 12 months taking an unbiased, whole-genome approach to target identification with the goal of changing the probabilities of drug discovery and development. This approach has rapidly generated a broad pipeline of cancer programs and positions us to create new medicines with higher success rates and better outcomes for patients. The power of our platform is evident in our first drug program, a modified adoptive T-cell immunotherapy with strong activity in PD-1 resistant solid tumors.
– David Meeker, MD, CEO, KSQ Therapeutics
CEO David Meeker, MD, was formerly the President and CEO of Genzyme, a Sanofi
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.
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