KSQ Therapeutics


Biotechnology company applying genome-scale CRISPR/Cas9 functional genomic screening to nominate therapeutic targets and develop oncology candidates, with programs in engineered autologous tumor-infiltrating lymphocytes (TIL) and small-molecule DNA damage response inhibitors. Core activities include large-scale in vivo and in vitro CRISPR screens, CRISPR-based engineering of autologous T cells for adoptive cell therapy, feeder-free clinical-scale TIL manufacturing, preclinical tumor-model testing, early-phase clinical development, regulatory interactions, and partnerships for process development and clinical manufacturing. The company maintains an external scientific advisory board and a board of directors with experience in immunology, cell therapy, manufacturing sciences, and finance.

Industries

Biopharma
Biotechnology
Pharmaceutical
Therapeutics
Pharmaceutical Manufacturing

Nr. of Employees

medium (51-250)

KSQ Therapeutics

Lexington, Massachusetts, United States


Patents

Compositions of substituted pyrazolopyrimidines and uses thereof

US-12624040-B2

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NR4A super-repressors and methods of use thereof

US-12590312-B2

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Gene-regulating compositions and methods for improved immunotherapy

US-12280111-B2

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Guide RNA combinations and methods of use

US-12188045-B2

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Gene-regulating compositions and methods for improved immunotherapy

US-12123021-B2

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Gene-regulating compositions and methods for improved immunotherapy

US-12084682-B2

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Products

KSQ-001EX (SOCS1-inactivated engineered TIL)

Autologous engineered TIL therapy with CRISPR/Cas9-mediated inactivation of SOCS1, manufactured using an accelerated feeder-free process and evaluated in a Phase 1/2 trial for multiple solid tumors.

KSQ-004EX (Regnase-1 and SOCS1 dual-inactivated engineered TIL)

Autologous engineered TIL product with dual CRISPR/Cas9 inactivation of Regnase-1 and SOCS1, manufactured using an accelerated feeder-free process and cleared for Phase 1/2 study by regulatory authorities.

KSQ-4279 (USP1 inhibitor small molecule)

Small-molecule inhibitor targeting USP1, identified using targeted functional genomic approaches and evaluated preclinically and in early clinical studies, with a licensing collaboration for further development.

ExPRESS

A proprietary, next-generation process to manufacture single and multiplex CRISPR/Cas9-edited eTIL therapies from surgical resections and core biopsies.

KSQ-004EX

Lead engineered tumor-infiltrating lymphocyte (eTIL) program advancing toward clinical studies for the treatment of solid tumors.

KSQ-004

An autologous, dual-gene edited tumor-infiltrating lymphocyte (TIL) cell product wherein Cas9/sgRNAs targeting the Regnase-1 and SOCS1 genes are electroporated into TIL to enhance anti-tumor potency and persistence against solid tumors.

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Services

Collaborative discovery programs and target identification using genome-scale CRISPR screening in cancer and immune models.

Third-party manufacturing partnerships for process development, scale-up, and clinical manufacturing of engineered TIL therapies.

Expertise Areas

  • Functional genomics and target discovery
  • CRISPR-based gene editing for cell therapies
  • Adoptive cell therapy development and clinical translation
  • Cell therapy manufacturing and CMC strategy
  • Show More (8)

Key Technologies

  • CRISPR/Cas9 gene editing
  • Genome-scale functional CRISPR screening
  • In vivo CRISPR screening
  • Engineered tumor-infiltrating lymphocyte (TIL) therapies
  • Show More (7)

News & Updates

Announcement that the first patient was dosed in the Phase 1/2 clinical study evaluating the dual-edited engineered TIL therapy.

Press release announcing regulatory clearance to initiate a Phase 1/2 clinical study of the dual-edited engineered TIL therapy.

Corporate announcement of appointment to CMO role.

Peer-reviewed article describing CRISPR screen-informed design of a SOCS1-edited TIL therapy.

Regulatory clearances to initiate Phase 1/2 clinical studies for engineered TIL candidates developed using CRISPR-based target discovery and gene editing.

Entered a worldwide license and collaboration agreement for development and commercialization of a USP1 inhibitor.


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