BioPharma, Startups

KSQ Therapeutics closes $80M Series C for cell therapy developed with CRISPR-based platform

The company aims to get its lead program, developed using its in-house “CRISPRnomics” platform, into the clinic in the next 18 months.

A company developing adoptive T-cell, immuno-oncology and targeted therapies using CRISPR-Cas9 technology has raised $80 million in a Series C financing round.

Cambridge, Massachusetts-based KSQ Therapeutics said Friday that the round included Cowen Healthcare Investments, Invus, Lilly Asia Ventures and Baillie Gifford as new investors, with founding and existing investors Flagship Pioneering, Polaris Partners, Alexandria Equities and ARCH Venture Partners participating.

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The company plans to use the funding to advance its first program into the clinic within the next year and a half, a tumor-infiltrating lymphocyte, or TIL therapy – a type of adoptive T-cell therapy – that it has thus far tested in animal models of PD-1 immune checkpoint resistance. The company also plans to conduct IND-enabling studies of three additional oncology programs.

According to its website, KSQ’s therapeutic focus comprises oncology, immuno-oncology and two undisclosed programs. It has used its platform, dubbed CRISPRnomics, to initiate or advance 12 drug-discovery programs over the last 12 months across three categories, namely adoptive cell therapies, immuno-oncology and targeted therapies.

The company’s website describes CRISPRnomics as using a suite of proprietary CRISPR-Cas9 tools to enable “high confidence” drug development, combining the gene-editing technology with large-scale functional genomics.

Cell therapies are one of the many potential applications of gene therapy, particularly allogeneic CAR-T therapies that would not require use of patients’ own T cells. CRISPR Therapeutics, based in Switzerland, is an example of a company that is using CRISPR-Cas9 technology to develop allogeneic CAR-Ts. CAR-Ts are one form of adoptive cell therapy, which also includes TILs and T-cell receptor therapies.

Earlier this month, the US Court of Appeals for the Federal Circuit ruled in favor of the Broad Institute of the Massachusetts Institute of Technology and Harvard University in a long-running patent interference lawsuit between it and the University of California Berkeley. A panel of three judges ruled that the Broad’s patent on CRISPR-Cas9 did not interfere with Berkeley’s patent application for its own version of the technology.

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