At the inception of Targazyme, we discovered that the efficacy of adoptive cell therapy for the treatment of a wide range of diseases is constrained by the impaired homing ability of therapeutic cells to reach diseased sites and engraft effectively in target tissues. Today, it is estimated that less than 5% of intravenously infused therapeutic cells successfully home to the bone marrow, tumor sites, and sites of inflammation or disease, with limited or delayed cell engraftment and penetration in target tissues or diseased organs.
Our proprietary cell efficacy multiplier fucosylation platform technology is founded on a distinctive combination of fucosyltransferase enzymes and small molecules to enhance the surface properties of therapeutic cells, empowering them to navigate more efficiently and effectively through the bloodstream to successfully reach target tissues or disease sites faster and in greater numbers. With precise targeting capabilities for specific diseased tissues and organs, the use of our fucosylation platform technology increases adoptive cell extravasation from the vasculature to the chemokine-expressing diseased sites, enabling preferential targeting to and improved penetration into tumors or organs affected by disease. Additionally, our cell efficacy multiplier platform technology has shown in multiple preclinical and clinical studies to improve adoptive cell engraftment and better intra-tumor/intra-diseased organ penetration, resulting in better cell therapy efficacy outcomes across multiple therapeutic cell types for a wide range of clinical indications.
To better understand the mechanism of action underlying the enhanced delivery of immune cells to the tumor microenvironment, we invite you to watch the attached video. This video provides an insightful visualization of how TZ 102 functions to improve the therapeutic impact of immune cells within the tumor microenvironment.
While Targazyme has been laser-focused on the development of our medicine to cure cancer patients, we have proven that our cell efficacy multiplier platform technology multiplies efficacy outcomes for multiple cell types, such as stem cells, NK cells, T-cells, MSCs, T-regs, and others, used for the treatment of multiple diseases, including cancer, autoimmune diseases, blood disorders, cardiovascular diseases, and regenerative medicine. Data generated at Targazyme and replicated at esteemed institutions, such as MD Anderson Cancer Center, Harvard, OMRF, and the University of Pennsylvania, indicate that fucosylated therapeutic cells exhibit 3-5 times greater delivery to diseased sites, along with a 3-5-fold increase in intra-tumor or intra-diseased tissue penetration compared to untreated cells. These enhancements contribute to better efficacy outcomes in the treatment of various diseases.
Moreover, published studies in esteemed journals such as Blood and Clinical Cancer Research further support the effectiveness of our cell efficacy multiplier fucosylation platform technology in enhancing the potency of therapeutic cells. Fucosylated NK and T cells demonstrate increased effectiveness as cancer tumor killers when compared to their non-fucosylated counterparts. Additionally, fucosylated stem cells display faster and more efficient engraftment within the bone marrow, leading to accelerated hematopoietic recovery post-Hematopoietic Stem Cell (HSC) treatment in comparison to non-fucosylated stem cells. This is important to help prevent the incidence of side effects associated with standard of care cancer therapies, such as infections, engraftment failure, and hemorrhaging. Fucosylated Regulatory T-cells and MSCs have also been shown to home more effectively to auto-immune disease sites, such as GVHD, with improved engraftment, increased residence time, and better disease control.
These scientific findings highlight the significant impact of our fucosylation cell efficacy multiplier platform technology in enhancing the capabilities and effectiveness of therapeutic cells across various applications and its potential to transform the whole field of cell therapy and patient outcomes. Targazyme’s commitment to rigorous research and innovation drives our mission to transform patient outcomes and revolutionize the field of cell-based therapies.
While adoptive cell therapy (ACT) utilizing autologous or allogeneic therapeutic cells holds immense potential to revolutionize treatment options for patients with blood cancers, solid tumors, blood disorders, autoimmune diseases, and regenerative medicine, significant unmet medical needs still persist. These unmet needs include the limited success of cancer immunotherapy in effectively treating patients with solid cancer tumors. Curative cell therapies such as hematopoietic stem cell transplantation and CAR-T cell therapies continue to be associated with major life-threatening side effects. The high cost of goods of these curative therapies prevents the majority of the world’s population from receiving these life-saving treatments, and terminally ill cancer patients have to endure extended wait times for these therapies while their disease progresses.
While other cell therapy companies have primarily directed their efforts towards expanding and engineering cells to better target tumors and diseased organs, we have chosen a radically different path to accomplish our mission of developing life-saving medicine for cancer patients. We have chosen to advance multiple products leveraging our cell efficacy multiplier platform technology, aimed at overcoming deficiencies in cell homing/trafficking and cell engraftment/intra-tumor and intra-diseased organ penetration. These are key constraints that limit the efficacy of cell therapy.
Data generated by Targazyme and replicated at OMRF, MD Anderson Cancer Center, Harvard, and the University of Pennsylvania have demonstrated that this strategy, in one broad strategic stroke, has been able to address multiple unmet medical needs described above simultaneously. Firstly, our FucoTIL products have shown the potential to transform efficacy outcomes for late-stage cancer patients with solid cancer tumors. Secondly, TZ 101 has demonstrated the potential to prevent or reduce the incidence of major side effects associated with standard-of-care curative cancer therapies. Thirdly, our platform technology has the potential to be leveraged by the entire industry to reduce the cost of goods, enabling reduced cell doses while achieving target efficacy outcomes. This makes cell therapy medicine more affordable for billions of patients worldwide and reduces patient wait times.