What KSQ Therapeutics does

KSQ Therapeutics is a clinical-stage biotechnology company founded in 2015 and headquartered in Lexington, Massachusetts, that applies its proprietary CRISPRomics® genome-scale functional genomics platform to identify and validate therapeutic targets in oncology and immunology. The platform conducts unbiased, in vivo CRISPR screening across more than 20,000 human genes and 600+ cancer and immune cell models, and has been used to nominate first-in-class targets including SOCS1, Regnase-1, and USP1. KSQ's lead pipeline consists of CRISPR/Cas9-engineered Tumor Infiltrating Lymphocyte (eTIL®) therapies — KSQ-001EX (single SOCS1 knockout) and KSQ-004EX (dual SOCS1/Regnase-1 knockout) — both manufactured through the proprietary 22-day ExPRESS™ process and currently in Phase 1/2 trials across multiple solid tumor indications including melanoma, head and neck squamous cell carcinoma, non-small cell lung cancer, colorectal cancer, pancreatic cancer, and cervical cancer. KSQ-4279, a first-in-class USP1 small-molecule inhibitor, has been out-licensed worldwide to Roche, and multiple research-stage DDR programs have been acquired by Ono Pharmaceutical.

The business model is a partnership-driven biotech model rather than a commercial product model. KSQ generates revenue from upfront payments, development and commercialization milestones, and royalties on partnered programs — with disclosed potential milestone exposure of more than $1 billion across the Takeda collaborations (2021 and 2023 expansion), Roche agreement (2023), and Ono transaction (2023). Equity capital raised totals approximately $156 million ($76M founding financing in October 2017 and $80M Series C in September 2018) from Flagship Pioneering, Polaris Partners, ARCH Venture Partners, Alexandria Equities, Baillie Gifford, Cowen Healthcare Investments, Invus, and Lilly Asia Ventures. The company has built an operational ecosystem comprising a clinical collaboration with MD Anderson Cancer Center for the eTIL trials, a manufacturing partnership with CTMC (a National Resilience/MD Anderson joint venture), a non-exclusive cross-license with CRISPR Therapeutics for editing IP, and a cell engineering platform agreement with MaxCyte.

Name

KSQ Therapeutics

Legal name

KSQ Therapeutics, Inc.

Website

https://ksqtx.com

Company type

Private

Founded year

2015

Operating status

Operating

Headcount range

51–100 employees

Short description

KSQ Therapeutics is a clinical-stage biotechnology company using its proprietary CRISPRomics® functional genomics platform to discover and develop CRISPR/Cas9-engineered Tumor Infiltrating Lymphocyte (eTIL®) therapies and targeted oncology drugs for advanced solid tumors.

Ownership category

akta.pro rank

Product category

Oncology Biotechnology / Cell Therapy

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414), Pharmaceutical Preparation Manufacturing (325412)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Oncology Specialty Pharmaceuticals (HLAIACAA)

akta.pro secondary industry

Personalized Therapeutic Design (neoantigen vaccines, individualized cell/gene therapies) (HLAAANAK)

Keywords

• Functional genomics
• Cell therapy oncology
• CRISPR drug discovery
• Tumor infiltrating lymphocytes
• Engineered cell therapies

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

Personnel, Technology or R&D, Operations, Infrastructure, Others

Revenue model

1. Licensing and Milestone Payments: Upfront payments, development and commercialization milestone payments, and royalties on net sales from strategic partnerships. Takeda deal: potential for more than $100M upfront and preclinical milestones, up to $400M+ per program in additional milestones plus royalties. Takeda expansion: upfront and investment in double-digit millions, up to $510M in future payments plus royalties. Roche: upfront and additional milestone and royalty payments.
2. Equity Financing: Series financing rounds. $76M financing at founding in October 2017. $80M Series C financing in September 2018. Investors include Flagship Pioneering, Polaris Partners, ARCH Venture Partners, Baillie Gifford, Cowen Healthcare Investments, Invus, Lilly Asia Ventures.
3. Program Acquisitions: ONO Pharmaceutical acquired multiple research-stage DDR programs with double-digit million upfront payment, near-term milestones, potential long-term value into triple-digit millions plus royalties on net sales

Go-to-market motion2 records

Distribution channels1 record

Marketing channels4 records

Core offering

KSQ Therapeutics is a clinical-stage biotechnology company that discovers and develops oncology therapeutics using its proprietary CRISPRomics® genome-scale functional genomics platform. The company is advancing a pipeline of CRISPR/Cas9-engineered Tumor Infiltrating Lymphocyte (eTIL®) therapies, including KSQ-001EX and KSQ-004EX, manufactured via its proprietary ExPRESS™ process, and has out-licensed the small-molecule USP1 inhibitor KSQ-4279 to Roche.

Product overview

KSQ Therapeutics is a clinical-stage biotechnology company developing a portfolio of cancer therapies powered by its proprietary CRISPRomics® functional genomics platform. The company's core offerings include two CRISPR/Cas9-engineered Tumor Infiltrating Lymphocyte (eTIL®) therapies—KSQ-001EX (single SOCS1 gene inactivation) and KSQ-004EX (dual SOCS1/Regnase-1 inactivation)—both manufactured using the company's proprietary ExPRESS™ process. Additionally, KSQ-4279, a first-in-class USP1 small-molecule inhibitor for DNA damage response, was out-licensed to Roche. The CRISPRomics platform enables genome-scale, in vivo validated target discovery, while the ExPRESS process enables efficient manufacturing of edited cell therapies from both surgical resections and core biopsies.

Differentiator

Problem solved

Functional benefit

Products and services

• CRISPRomics® Platform Proprietary genome-scale functional genomics platform using CRISPR/Cas9 technology to systematically identify and validate therapeutic targets across cancer and immune cells. Screens over 20,000 genes across more than 600 cancer lines and immune cell types (T cells, NK cells, T regulatory cells) for unbiased, in vivo validated target discovery.
• ExPRESS™ Manufacturing Process Proprietary next-generation manufacturing process (Ex Vivo, Potent, Economical, Streamlined, Simple) for producing single and multiplex CRISPR/Cas9-edited eTIL therapies. Generates a cryopreserved product with maximized cancer-killing potential from surgical resections and core biopsies using a 22-day manufacturing cycle across 10+ solid tumor types.
• KSQ-001EX Investigational CRISPR/Cas9-engineered Tumor Infiltrating Lymphocyte (eTIL®) therapy in which the SOCS1 gene is inactivated, manufactured using the ExPRESS™ process. In Phase 1/2 clinical study for advanced solid tumors including melanoma, head and neck squamous cell carcinoma, and non-small cell lung cancer.
• KSQ-004EX Dual-edit eTIL investigational therapy in which both the Regnase-1 and SOCS1 genes are inactivated by CRISPR/Cas9 gene editing, manufactured using the ExPRESS™ process. In Phase 1/2 clinical study for advanced solid tumors including melanoma, head and neck squamous cell carcinoma, non-small cell lung cancer, colorectal cancer, pancreatic cancer, and cervical cancer.
• KSQ-4279 (RO7623066/RG6614) First-in-class, potent, and selective small-molecule inhibitor of USP1 (Ubiquitin-specific processing protease 1), a protein regulating DNA damage response (DDR). Licensed worldwide to Roche for development and commercialization; previously studied in Phase 1 clinical trial in patients with advanced solid tumors including BRCA-mutant and HRD cancers.

Quantifiable outcome

• 10-fold increase in anti-tumor activity in vivo with CT-1 edited T cells
• +2 more outcomes

Named customers1 record

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature6 records

Partnerships

Eight partnerships are on record, tiered key and core.

• The University of Texas MD Anderson Cancer CenterkeyStrategic or Co-development Partner · 24 September 2024Clinical collaboration for KSQ-004EX Phase 1/2 study. MD Anderson provides clinical trial site and principal investigator Dr. Rodabe Amaria (professor of Melanoma Medical Oncology). Also collaborates on KSQ-001EX study.
• CTMC (Cell Therapy Manufacturing Center)coreStrategic or Co-development Partner · 6 September 2023Strategic collaboration with CTMC, a joint venture between National Resilience, Inc. and MD Anderson Cancer Center. CTMC provides full process development and manufacturing support for KSQ's lead eTIL programs KSQ-001EX and KSQ-004EX, advancing toward clinical studies for solid tumors. Manufacturing uses KSQ's ExPRESS process.
• RochecoreOEM/ Whitelabel/ Licensing Partner · 13 July 2023Worldwide license and collaboration agreement for KSQ-4279, a first-in-class USP1 inhibitor for cancer. Roche assumes development responsibilities including full clinical development from 2024. KSQ receives upfront payment and eligible for milestone and royalty payments. KSQ-4279 is in Phase 1 clinical trial for advanced solid tumors.
• Takeda Pharmaceutical Company (Expansion)coreStrategic or Co-development Partner · 17 May 2023Expanded collaboration focused on novel tumor-intrinsic target discovery and validation. Takeda provides upfront payment and investment in double-digit millions. KSQ eligible for up to $510M in future payments if all milestones achieved, plus royalties on net sales. Expansion adds work on tumor-intrinsic targets to ongoing NK and T-cell collaboration.
• Ono Pharmaceutical Co., Ltd.keyOEM/ Whitelabel/ Licensing Partner · 25 January 2023Ono Pharmaceutical acquired multiple research-stage DNA damage response (DDR) programs identified using KSQ's CRISPRomics platform. All programs are novel with potential for first-in-class therapies. ONO provided double-digit million upfront payment with near-term milestones expected and potential long-term value into triple-digit millions plus royalties on net sales.
• Takeda Pharmaceutical CompanycoreStrategic or Co-development Partner · 13 January 2021Broad strategic collaboration to research, develop and commercialize novel immune-based therapies for cancer. KSQ granted Takeda exclusive worldwide royalty-bearing license for cell and non-cell therapy products modulating targets identified using CRISPRomics. Deal includes two T-cell programs and NK cell target discovery. Potential for $100M+ upfront and preclinical milestones, up to $400M+ per program additional milestones plus royalties. KSQ retains U.S. cost/profit-sharing option on one product.
• MaxCytekeyTechnology or Integration · 4 December 2019Development and commercialization agreement using MaxCyte's Flow Electroporation technology and ExPERT instruments for KSQ's eTIL programs. KSQ obtains non-exclusive clinical and commercial use rights to MaxCyte's cell engineering platform for multiple adoptive cell therapies. MaxCyte eligible for milestone payments and licensing fees.
• CRISPR TherapeuticskeyTechnology or Integration · 15 October 2019License agreement for mutual IP access. CRISPR Therapeutics gains non-exclusive access to KSQ IP for editing certain novel gene targets in its allogeneic oncology cell therapy programs. KSQ gains non-exclusive access to CRISPR Therapeutics IP for editing novel gene targets in its autologous eTIL cell programs.

Scale indicators9 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Beam Therapeutics: Beam is a clinical-stage precision genetic medicines company using base editing, a CRISPR-derived technology. Comparable platform-driven biotech with similar oncology focus and partnership approach (Pfizer, Verve).
• Intellia Therapeutics: Intellia is a clinical-stage CRISPR/Cas9 platform company developing both ex vivo (cell therapy) and in vivo gene editing programs. Similar platform-technology profile and partnership-driven business model with comparable Big Pharma validation (Novartis, Regeneron).
• Iovance Biotherapeutics: Iovance is the most direct peer - the leader in TIL therapy with FDA-approved lifileucel for melanoma. KSQ's eTIL programs compete head-to-head in melanoma and other solid tumors, and Iovance's commercial experience directly benchmarks KSQ's opportunity and challenges.
• Adaptimmune: Adaptimmune develops engineered T-cell receptor (TCR) therapies for solid tumors, with approved Tecelra for synovial sarcoma. Direct competitor in the solid tumor cell therapy space where KSQ's eTIL programs also operate.
• Editas Medicine: Editas is a clinical-stage CRISPR gene editing company developing edited cell therapies for oncology and genetic diseases. Comparable platform-technology approach and similar partnership-driven development model.
• CRISPR Therapeutics: CRISPR Therapeutics is the most direct CRISPR platform peer, with its Cas9-based allogeneic CAR-T programs. KSQ has a mutual license with CRISPR Therapeutics (2019) and competes for the same gene-edited cell therapy opportunity, with overlapping target space.

Emerging players

• Cellectis: Cellectis develops TALEN-based gene-edited allogeneic CAR-T cell therapies. Comparable gene-edited cell therapy approach but earlier stage in clinical development and focused on allogeneic platform.
• Allogene Therapeutics: Allogene is a clinical-stage allogeneic CAR-T company with multiple programs in lymphoma and other hematologic malignancies. Comparable cell therapy platform approach and Big Pharma backing (Pfizer, Cellectis founder).
• Caribou Biosciences: Caribou is a clinical-stage CRISPR genome-edited allogeneic CAR-T and NK cell therapy company. Comparable gene-edited cell therapy technology profile but with allogeneic (off-the-shelf) rather than KSQ's autologous approach.
• Fate Therapeutics: Fate develops iPSC-derived off-the-shelf NK and T-cell cancer immunotherapies. Comparable cell therapy modality for oncology with multiple Big Pharma partnerships (J&J, Ono), analogous to KSQ's licensing model.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/KSQ_TX
• LinkedInlinkedin.com/company/10414448

Revenue estimate

Valuation estimate

Number of profiles

Profiles11 records

Funding overview

Funding rounds2 records

Investors8 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A1 record

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about KSQ Therapeutics