TargaZyme is an award-winning, phase 3 ready biopharmaceutical company developing and commercializing a cell efficacy multiplier platform technology and a family of novel, proprietary fucosyltransferase enzyme drug candidates with the potential to transform the efficacy, safety, affordability and accessibility of potentially curative blood, cell and gene therapies. These novel enzymes serve as the ‘GPS’ of therapeutic cells, improving their delivery to where they are needed most, i.e. diseased sites, with multi-fold efficiency and improved precision while also enabling longer residence time and improved therapeutic potency to more effectively treat diseases. As a result, our enzymes overcome a key technical constraint constraining the efficacy and affordability of the whole field of blood, cell and gene therapies: specifically, the inefficient trafficking/delivery of therapeutic cells (T, B, NK, Treg, MSC, HSC, EPCs) to diseased tissues where today, less than 1–2 percent of cells traffic effectively to diseased organs/tissues.
While TZ 101’s proven MOA has the potential to transform the whole field of blood and cell therapies, we have been laser focused since the company’s inception on developing cancer medicine, driving our cancer programs from discovery stage to phase 3 readiness and an FDA concurred registration program. TZ 101, our off-the-shelf, Phase 3 ready enzyme drug candidate is proven clinically in phase 2 clinical trials to improve the safety and the curative potential of blood transplant therapies for terminally ill cancer patients. Compelling Phase 2 trial data led to an FDA Phase 3 Special Protocol Assessment/Breakthrough Designation award enabling a derisked regulatory pathway towards Marketing Authorization as well as an FDA orphan drug designation. FDA concurred label of ‘Accelerating Hematopoietic Recovery Post Hematopoietic Stem Cell Transplantation’ enables major competitive differentiation/advantage for TZ 101 since unlike competitors that offers ‘piecemeal’ solutions to addressing the myriad of unmet medical needs with this medical procedure, TZ 101 offers the ‘complete solution’ by overcoming the root cause of the HSCT failure enabling both myeloid and lymphoid cell (T, NK, Treg cell) recovery required to prevent the various morbidities and disease relapse. This FDA concurred target label also positions TZ 101 for sale with the HSCT medical procedure to treat multiple diseases including serious auto-immune/neurodegenerative diseases, blood disorders, immune disorders and genetic diseases and for both autologous and allogeneic transplants, post FDA Marketing approval.
In recent years, we have augmented our translational research and development focus on cancer with neurodegenerative/auto-immune diseases due to compelling data showing TZ 101 transforming regulatory T cell therapy outcomes for treatment of diseases such as Multiple Sclerosis and Parkinson’s. A phase 2 clinical trial is currently planned for TZ 101 autologous blood transplant for Relapsing MS patients to improve NEDA outcomes (No evidence of disease activity).
Targazyme has been awarded 35+ worldwide patents with more than 20 patent applications pending to enable patent protection till 2043. The technology has been validated through multiple independent peer-reviewed prestigious medical and scientific publications such as Blood, Nature, Clinical Cancer Research and is a recipient of $60M+ of prestigious medical and scientific awards for the advancement of the fucosylation platform technology not only for its initial clinical indication of cancer but also for treatment of auto-immune, neurodegenerative, cardiovascular, diabetic retinopathy, cardiovascular diseases and for regenerative medicine. Furthermore, strategic partnerships are in place with leading cancer centers such as MD Anderson Cancer Center, Harvard, University of Pennsylvania Medical Center, Yale Medical Center, Duke Medical Center, Cleveland Clinic, University of California, corporate partners such as Kyowa Hakko Kirin, VidaCord of Europe, Asahi Kasei, ThermoFisher, George Clinical.
As we continue to advance medical innovation, we remain committed to pushing the frontier of medicine and science of what is possible, to accomplish our mission-impossible mission. Our cutting-edge products represent a transformative approach that promises to multiply the efficacy, safety, affordability and accessibility of potentially curative blood and cell therapies to successfully treat, cure and even prevent diseases.