Engineering of Immune Cells Inside the Body
The Problem: Engineered cell therapies are complex to make, take weeks to manufacture, can only be administered at a small number of specialized hospital facilities, and can cost up to one hundred thousand dollars to produce one dose. Only a handful of cell therapies have won approval, and none in solid tumors or other immune system disorders.
The Current State: Researchers are working to improve immune-modifying therapies and apply them to broader conditions. Advances in cell-specific targeting and gene delivery could make a more precise type of therapeutic possible. Another approach is substituting patient harvested immune cells with healthy donor ones to create an off-the-shelf cell therapy. Even still, the manufacturing challenges inherent to current cell engineering methods mean the treatment is very expensive, limiting its use to the sickest patients and available only at a few treatment centers.
The Challenge: The Engineering of Immune Cells Inside the Body, or EMBODY, program aims to develop an adaptable, low-cost platform where cells in the body are given instructions to adjust their behavior. By moving the creation of specially trained immune cells out of the lab and in vivo, this platform could eliminate the time, cost, and access hurdles faced by traditional immune cell therapy manufacturing.
Moreover, EMBODY is not limited to a single disease or condition. It aims to employ these new technologies to treat various immune system disorders, including chronic autoimmune diseases, hard-to-treat infections, and solid tumor cancers.
The Solution: To make immune therapies more affordable, easily accessible at any U.S. hospital, and to eliminate the multi-week wait time, EMBODY will focus on two major technical areas. Technical area 1 encompasses cell-specific delivery methods and development of programmable and controllable genetic material. Technical area 2 supports advanced production and validation methods, including reducing manufacturing cost, improving quality control, and developing predictive preclinical models of the human immune system.
Engineering of Immune Cells Inside the Body
The Problem: Patients in need of organ transplants face chronic shortages, long wait lists, and the lifelong risk of transplant rejection. A variety of factors impact organ transplant shortages, including a recipient’s geographic location, the need for blood type matches as well as expensive immunosuppressive drugs, and low donation rates. Someone can wait months to years for a transplant organ, and thousands of patients in the U.S. die annually while awaiting a match.
The Current State: Current matching criteria is done by a national computer list that considers medical urgency, tissue match and blood type, organ size, immune status, and geographic distance. Because of factors like geographic distance and the need for certain, blood type matches, people in rural areas or those from Black, Asian, and minority ethnic groups are less likely to receive an organ. When a compatible organ does become available, the recipient faces a lifetime of immunosuppressive drugs to combat rejection. Transplanted organs last only a decade or two, and complications are common.
The Challenge: The Personalized Regenerative Immunocompetent Nanotechnology Tissue (PRINT) program intends to use state-of-the-art bioprinting technology and a regenerative medicine approach to 3D print personalized, on demand organs that do not require immunosuppressive drugs. The goal is to use patient cells or a biobank to quickly produce immune- and blood type-matched replacement organs, such as kidneys, hearts, and livers. If successful, PRINT technology would decrease donor list wait times, reduce the need and cost for immunosuppressive drugs, and make organs and tissues more widely available for patients across the country.
The Solution: PRINT focuses on three technical areas with the end goal of restoring normal tissue function. The first technical area aims to generate the necessary cell types for organ bioprinting, via blood draw, biopsy, or biobank generation. The second technical area involves large scale manufacturing of cell types. The third technical area focuses on organ biofabrication and testing for safety and efficacy. Performers are encouraged to explore multiple methods for achieving the goals of each technical area.