On September 28, 2018 KSQ Therapeutics reported that the company has secured an $80 million Series C financing to advance oncology drug candidates generated from the company’s proprietary CRISPRomics drug discovery engine into clinical studies (Press release, KSQ Therapeutics, SEP 28, 2018, View Source [SID1234529649]). With the financing, KSQ will advance its first drug program into the clinic within the next 18 months and up to three additional oncology drug programs into 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.

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In the past 12 months, utilizing its CRISPRomics platform, KSQ has initiated and advanced 12 drug discovery programs across three oncology drug categories: adoptive T-cell therapies, immuno-oncology, and targeted therapies. In addition, KSQ is continuing to expand CRISPRomics for use in other therapeutic areas, including immunology and rare diseases. The Series C financing includes new investors Baillie Gifford, Cowen Healthcare Investments, Invus, and Lilly Asia Ventures, as well as full participation from its founding investors Flagship Pioneering and Polaris Partners, as well as existing investors ARCH Venture Partners and Alexandria Equities.

"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," said David Meeker, MD, Chief Executive Officer of KSQ. "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."

"KSQ has made impressive progress in demonstrating the power of its pioneering CRISPRomics discovery engine," said Jim Gilbert, Senior Partner at Flagship Pioneering and Chairman of the Board at KSQ. "We see a compelling opportunity for KSQ’s high-confidence drug development approach to identify untapped opportunities and improve productivity in developing innovative medicines."

Using the genome-scale analysis of its CRISPRomics technology, KSQ has uncovered the most relevant therapeutic targets while ruling out thousands of less relevant targets at the outset. To date, KSQ has applied this high-confidence drug development approach in two areas: a tumor-genome platform for targeted cancer therapies and a T-cell genome platform for immuno-oncology monotherapies.

With the tumor-genome platform, KSQ has interrogated the function of all 20,000 human genes across more than 600 cancer models – a massive data base which allowed the company to pinpoint the optimal therapeutic targets and patient selection biomarkers for multiple cancer types.
With the T-cell genome platform, KSQ has comprehensively mined the function of all genes in the T cell in vivo, allowing identification of adoptive T-cell enabling targets and the next generation of monotherapy targets in immuno-oncology.
Dr. Meeker continued, "Our CRISPRomics engine has exceeded expectations in its performance and productivity, as it has systematically pinpointed cancer targets across two platforms – one for targeted therapies and another for immuno-oncology – and shown extraordinary scale, precision and speed in generating our pipeline of more than a dozen drug programs. With this financing and strong syndicate of investors, we are well positioned to take the next steps in realizing the potential of CRISPRomics to advance medicines that will have meaningful impact for patients."

About CRISPRomics

KSQ Therapeutics has built a genome-scale, functional-genomics drug discovery engine, called CRISPRomics, to pinpoint therapeutic nodes that directly correlate genomic function to disease with unprecedented certainty and speed. Using CRISPRomics, KSQ is elucidating 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, targets 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.