EXUMA Biotech, Corp. is a clinical-stage molecular immunology and biotechnology company dedicated to discovering and developing next-generation cell and gene therapies for oncological malignancies and autoimmune diseases. Headquartered in West Palm Beach, Florida, with a global research and development footprint, the company focuses on designing genetic vector delivery solutions that bypass the traditional, resource-intensive barriers of Chimeric Antigen Receptor T-cell (CAR-T) manufacturing.
Standard cell therapies depend on costly ex vivo (outside the body) cellular manipulation and toxic preparative lymphodepleting chemotherapy regimes. EXUMA Biotech circumvents these challenges through proprietary platforms capable of targeting, loading, and modifying immune cells directly inside the patient (in vivo) or through ultra-rapid point-of-care workflows. Their therapeutic portfolio aims to transition cellular medicine out of tertiary academic hospital settings and directly into community clinics by generating functional, safe, and rechargeable CAR-T cells entirely within the patient's body.
Core Technology Platforms
EXUMA Biotech’s scientific framework integrates specialized lentiviral vector engineering, synthetic biology, and logic-gated safety receptors to safely exploit and manage the therapeutic potential of cellular treatments.
1. In Vivo GCAR™ Platform
The Global Chimeric Antigen Receptor (GCAR) platform is EXUMA’s foundational approach to true "off-the-shelf" in vivo CAR therapy. Rather than harvesting a patient's cells, modifying them in a lab, and re-infusing them weeks later, the GCAR platform uses a targeted delivery vehicle injected directly into the patient.
* CD3-Targeted Lentiviral Vectors (LVs): These engineered vectors feature a surface element designed to selectively bind, activate, and enter CD3-positive circulating lymphocytes inside the human body. This specialized homing system avoids acute non-specific systemic toxicities while precisely delivering the genetic code to target T cells and Natural Killer (NK) cells.
* FITNESS DRIVER Transgene: Embedded inside the vector payload is a proprietary, synthetic, membrane-bound signaling construct. Discovered via an unbiased screening process of thousands of signaling protein pairs, the FITNESS DRIVER optimizes cellular stemness, proliferation, and persistence. This allows the engineered cells to proliferate and exert cytotoxic anti-tumor actions without needing lymphodepleting pre-treatment chemotherapy.
2. rPOC SC Platform
The rapid Point-of-Care Subcutaneous (rPOC SC) platform provides an intermediate, low-complexity autologous solution. Peripheral Blood Mononuclear Cells (PBMCs) are harvested via a routine blood draw and incubated briefly (approximately 4 hours) ex vivo with EXUMA's CD3-targeted lentiviruses.
* Subcutaneous Delivery: The loaded lymphocytes are administered via a subcutaneous injection rather than an intravenous line.
* Synthetic Lymph Node Formation: Once injected beneath the skin, the formulation naturally stimulates a localized tertiary lymphoid structure (a synthetic lymph node) at the injection site. From this site, the CAR-T cells safely mature, expand exponentially via the internal FITNESS DRIVER, and distribute systematically to combat remote disease sites.
3. Tumor Metabolism Regulated (TMR) CARs
To successfully target solid tumors without triggering severe "on-target, off-tumor" toxicities (where healthy tissue expressing the same antigen is destroyed), EXUMA developed its TMR logic-gating technology. TMR CAR architectures exploit the hostile, acidic, and metabolically distinct environments unique to tumor microenvironments (TME). Binding selectivity is reversibly gated: the CAR receptor remains inactive in normal systemic circulation but becomes highly active when it encounters the distinct conditions of the TME, expanding the therapeutic index for over-expressed solid tumor antigens.
4. Synthetic CAR Ligands (SCL)
To resolve the historical challenge of CAR-T exhaustion or early cell death, EXUMA uses Synthetic CAR Ligands (SCL) delivered via lipid nanoparticle (LNP)-encapsulated mRNA. SCL acts as a controllable, antigen-independent "recharge" mechanism. By administering an off-the-shelf SCL injection, physicians can dynamically stimulate the engineered CAR cells in vivo, modulating the kinetic parameters (Cmax, AUC, and long-term durability) without requiring repeated, complex cellular manufacturing or re-dosing protocols.
Therapeutic Pipeline & Clinical Development
EXUMA Biotech actively channels its integrated platforms into human clinical trials and advanced preclinical workflows targeting liquid tumors, solid tumors, and autoimmune conditions.
Clinical Candidates
CCT303-406 (HER2): An autologous, solid-tumor CAR-T candidate utilizing the company's Tumor Metabolism Regulated (TMR) safety technology to target HER2-overexpressing malignancies. This candidate recently completed an investigator-initiated Phase 1 clinical trial evaluating its safety and activity in patients with advanced gastrointestinal and metastatic solid tumors. Clinical data presented at the 2025 ASCO Gastrointestinal Cancers Symposium demonstrated zero dose-limiting toxicities, zero serious adverse events, and deep, ongoing partial responses (including a 100% reduction in sum of longest diameters [SLD] at 36 weeks in a gastroesophageal patient).
CCT303-ASL-0038 (AXL): A conditionally active autologous CAR-T cell therapy targeting AXL-positive solid malignancies, focusing on indications such as advanced osteosarcoma.
C402-CD19-CAR: A point-of-care autologous CAR-T program in Phase 1 development targeting CD19-positive hematological cancers, including follicular lymphoma and other B-cell leukemias.
Preclinical & Next-Generation Development
rPOC-0752/0754 (CD19 x CD22): A bispecific dual-targeting CAR-T pipeline candidate under preclinical development for advanced B-cell lymphomas, utilizing the rapid 4-hour point-of-care formulation.
In Vivo GCAR™ IND-Enabling Programs: Leveraging successful clinical de-risking from the autologous CCT303-406 study, EXUMA is executing non-human primate (NHP) validation studies combining their solid-tumor and hematologic CAR payloads with the in vivo GCAR delivery platform and SCL mRNA technology to support Investigational New Drug (IND) activities in the United States.
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