Leukogene Therapeutics

What Leukogene Therapeutics does

Leukogene Therapeutics, Inc. is a preclinical-stage cancer biotherapeutics company developing MHC class II-targeted immunotherapies under its proprietary M2T™ platform. The platform is a recombinant protein technology that conjugates tumor-associated antigens (TAAs) to a high-affinity MHCII-binding protein, functioning as an antigen-presenting cell engager that activates CD4+ helper T cells and orchestrates both T- and B-cell immune responses against tumor cells expressing the targeted antigen. The stated scientific rationale is to convert immunologically 'cold' tumors into 'hot' tumors by engaging the MHCII-CD4+ axis, which the company positions as complementary to — and synergistic with — incumbent MHCI-CD8+ focused modalities.

The company's pipeline consists of two preclinical candidates. The lead asset, LTI-214 (M2T-CD33), targets CD33-positive leukemic blasts and leukemic stem cells for acute myeloid leukemia (AML); the second, LTI-002 (M2T-Mesothelin), targets mesothelin-expressing solid tumors including pancreatic ductal adenocarcinoma and ovarian cancer. The lead candidate received FDA Orphan Drug Designation in November 2025, and the company has indicated first-in-human clinical evaluation of LTI-214 in AML is imminent. Preclinical data demonstrating anti-leukemic efficacy, CD4+/CD8+ T-cell-dependent mechanism, synergy with anti-PD-1, and absence of toxicity at 40-fold efficacious dose were published in Blood Cancer Journal (Nature portfolio) in November 2025.

Leukogene is a privately held spin-out of the Medical University of South Carolina (MUSC) Hollings Cancer Center, headquartered in Charleston, SC within MUSC's Blue Sky Labs facility. The company has 1–10 employees, is pre-revenue, and has no commercial products; its revenue model will ultimately rely on one-time licensing deals or future product sales post-approval. To date it has been funded by SCRA grants, SC Launch Inc., NCI portfolio status, and was the first portfolio investment of MUSC's newly established $20M 1824 Health Ventures Fund I. The current leadership team, recruited in late 2024, is led by CEO Dr. Sandeep Gupta (ex-Asana BioSciences, 25+ years pharma experience) and Chairman Dr. Lawrence Olanoff (former President/COO of Forest Laboratories).

Name

Leukogene Therapeutics

Legal name

Leukogene Therapeutics, Inc.

Website

https://leukogene.com

Company type

Private

Founded year

2021

Operating status

Operating

Headcount range

1–10 employees

Short description

Leukogene Therapeutics is a preclinical-stage, Charleston, SC-based biotech developing MHC class II-targeted (M2T™) recombinant protein immunotherapies for hard-to-treat cancers, with lead candidate LTI-214 in AML and a second candidate in pancreatic and other solid tumors.

Ownership category

akta.pro rank

Product category

Cancer Immunotherapy Biopharmaceuticals

NAICS

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

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro industry

PRIMARYHLAIAAAD

Immunology & Inflammation Pharmaceuticals

Healthcare & Life Sciences/Pharmaceuticals/Branded (Innovator) Pharmaceuticals

Keywords

• Cancer immunotherapy
• MHC class II targeted
• Acute myeloid leukemia treatment
• Oncology biotherapeutics
• Solid tumor immunotherapy

Headquarters

HQ city

Mount Pleasant

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations

Revenue model

1. Biopharmaceutical Product Revenue: Pre-revenue company. Plans to commercialize M2T™ immunotherapy candidates through eventual product sales or licensing partnerships upon successful clinical development and regulatory approval.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Leukogene Therapeutics develops a proprietary M2T™ immunotherapy platform — a recombinant protein technology that conjugates tumor-associated antigens to MHC Class II (MHCII) binding proteins to mobilize immune cells against cancer. The company's pipeline comprises two preclinical-stage candidates: LTI-214 (M2T-CD33), a lead candidate for acute myeloid leukemia (AML) which received FDA Orphan Drug Designation in November 2025, and LTI-002 (M2T-Mesothelin) targeting solid tumors including pancreatic ductal adenocarcinoma and ovarian cancer. The company is pre-revenue, currently advancing candidates toward first-in-human clinical evaluation.

Product overview

Leukogene Therapeutics operates a platform-plus-products model built around its proprietary M2T™ (Major Histocompatibility Complex Class II-targeted) immunotherapy platform. The M2T™ platform is a recombinant protein that conjugates tumor associated antigens to an MHCII targeting protein, functioning as an antigen presenting cell engager that generates potent T and B cell immune responses. The company's pipeline consists of two clinical development candidates: LTI-214 (M2T-CD33), the lead asset targeting CD33-positive leukemic cells for acute myeloid leukemia, and LTI-002 (M2T-Mesothelin), targeting mesothelin for pancreatic and other solid tumors. Both candidates are in preclinical development with the company preparing for first-in-human clinical evaluation of LTI-214 in AML.

Differentiator

Problem solved

Functional benefit

Products and services

• M2T™ Immunotherapy Platform A novel recombinant, high-affinity Major Histocompatibility Complex Class II (MHCII) binding protein conjugated to tumor-associated antigens. Functions as an antigen presenting cell (APC) engager that directly stimulates MHCII and generates a powerful T and B cell response against chosen antigens, converting immunologically cold tumors into more responsive hot tumors. Targeted at pharmaceutical partners and oncology drug development.
• LTI-214 (M2T-CD33) Leukogene's myeloid-targeted lead immunotherapy designed to selectively eliminate CD33-positive leukemic blasts and leukemic stem cells that drive disease progression in acute myeloid leukemia (AML). Preclinical studies demonstrated robust anti-leukemic efficacy with significant reduction in leukemic burden and prolonged survival, along with a favorable safety profile. Currently in preclinical development with first-in-human clinical evaluation expected in the near term.
• LTI-002 (M2T-Mesothelin) Second M2T™ platform candidate targeting tumor antigen mesothelin, currently in preclinical development for treating pancreatic ductal adenocarcinoma, ovarian cancer, and other solid tumors.

Quantifiable outcome

• Preclinical studies demonstrated robust anti-leukemic efficacy in AML models with significant reduction in leukemic burden and prolonged survival
• +2 more outcomes

Segments2 records

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

One partnership is on record.

• Medical University of South Carolina (MUSC)coreStrategic or Co-development PartnerLeukogene is a spin-out from MUSC. The company originated from research at MUSC Hollings Cancer Center. Dr. Nathan Dolloff serves as Director of Entrepreneurship at MUSC Hollings Cancer Center. Company is located at Blue Sky Labs associated with MUSC.

Scale indicators3 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• Marker Therapeutics: Marker Therapeutics develops TCR-based immunotherapies for hematologic malignancies and solid tumors, including AML. Comparable as a small-cap immuno-oncology company targeting similar hematologic cancer indications with novel T cell-directed approaches.
• Werewolf Therapeutics: Werewolf Therapeutics develops tumor-activated immunotherapies using the PREDATOR platform - engineered proteins conditionally activated in the tumor microenvironment. Directly comparable as a protein-based cancer immunotherapy platform company targeting cold/immune-excluded tumors with novel biological mechanisms.
• Maverick Therapeutics: Maverick Therapeutics (acquired by Takeda) developed conditional T cell engager (CTE) protein therapeutics for solid tumors. Comparable as a developer of engineered protein platforms with novel mechanisms to engage immune cells against tumors.
• Compass Therapeutics: Compass Therapeutics develops novel antibody-based immunotherapies for cancer, including bispecifics that engage immune cells against tumors. Comparable as a clinical-stage immuno-oncology company using engineered protein therapeutics with novel mechanisms to address tumors resistant to conventional immunotherapy.
• Harpoon Therapeutics: Harpoon Therapeutics (acquired by Merck in 2024) developed T cell engagers - engineered protein therapeutics that redirect T cells to tumor antigens. Directly comparable as a recombinant protein immunotherapy platform company developing novel immune-engaging modalities for cancer, including hematologic malignancies.
• Adagene: Adagene uses its POWER platform to discover and engineer antibody-based immunotherapies for cancer. Comparable as a clinical-stage biotech developing novel protein-based immunotherapies with proprietary platforms targeting difficult-to-treat tumors.

Broad incumbents

• Gilead Sciences: Gilead (through Kite and via acquisition of Forty Seven) markets magrolimab (anti-CD47) for hematologic malignancies including AML. Comparable as a major oncology player with established AML franchise and significant R&D investment in next-generation immuno-oncology approaches.
• AbbVie: AbbVie markets Venclexta (venetoclax), the standard-of-care BCL-2 inhibitor for AML, and has a deep hematology/oncology pipeline. Comparable as the dominant AML therapeutics incumbent against whose franchise Leukogene's lead candidate (LTI-214) will eventually compete.
• Bristol-Myers Squibb: BMS markets Opdivo (nivolumab) and Yervoy (ipilimumab) checkpoint inhibitors and has multiple AML/hematology programs. Comparable as the immuno-oncology incumbent whose checkpoint blockade franchises define the standard Leukogene seeks to complement or compete with through CD4+ axis engagement.

Emerging players

• Pyxis Oncology: Pyxis Oncology develops antibody-drug conjugates and immunotherapies for difficult-to-treat cancers. Comparable as an emerging oncology company developing novel protein-based therapies targeting tumors with limited treatment options, including hematologic malignancies.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/leukogene-therapeutics-inc

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles5 records

Funding overview

Funding rounds1 record

Investors2 records

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M&A

Investments

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

Frequently asked questions about Leukogene Therapeutics