Technology Invented in UCSF Lab has Many Other Applications for Human Cell Therapy
Encellin, a San Francisco–based biotechnology company, has obtained exclusive worldwide rights from UC San Francisco for a proprietary cell encapsulation technology aimed at improving physicians’ ability to perform cell transplants without the need for immunosuppressive drugs.
Based on ongoing preclinical trials in animal models, the technology – in the form of a pouch approximately the diameter of a quarter, made of an ultrathin nanoporous membrane – represents a significant advance towards the ability to transplant donated cells without danger of immune rejection or harmful fibrosis at the transplant site, while also ensuring that transplanted cells cannot infiltrate other parts of the body.
Encellin first aims to apply this technology – originally developed in the laboratory of Tejal Desai, PhD, chair of the Department of Bioengineering and Therapeutic Sciences in UCSF’s schools of Pharmacy and Medicine – to treat type 1 diabetes, an autoimmune disease affecting over 1 million Americans, with over 9,000 young people newly diagnosed each year.
Type 1 diabetes is caused by the loss of the islet cells of the pancreas, which normally secrete the hormone insulin to coordinate the body’s use of blood glucose. The transplantation of functional, insulin-producing pancreatic islet cells from a donor has shown clinical efficacy as a treatment for some people with type 1 diabetes, but – like most transplantation techniques – this treatment requires lifelong immunosuppression to prevent patient immune systems from destroying the donor cells. However, these immunosuppression drugs also make patients susceptible to heightened risk of infection, cancer, and organ damage.
Encellin’s device will encapsulate glucose-sensitive, insulin-producing islet cells in a pouch that can be implanted just under the skin. Following this minimally invasive procedure, the encapsulated islet cells can communicate chemically with the bloodstream through the nanopore membrane, letting them respond to changes in glucose and provide insulin to the recipient, while at the same time protecting the transplant from the recipient’s immune system.
“We are thrilled to have this technology from UCSF, and to move our therapy toward the clinic where it can benefit patients with type 1 diabetes. Our goal is to significantly reduce the burden of glucose management for these patients without the need for lifelong immunosuppression,” said Crystal Nyitray, PhD, CEO and co-founder of Encellin, and the co-inventor of the cell encapsulation technology in the Desai lab.
"Given the success of this approach in the lab, we are excited for the potential of this technology to impact diabetes treatment,” said Desai, who directs the Therapeutic Microtechnology and Nanotechnology Laboratory at UCSF and is an acknowledged world leader in developing microscale and nanoscale drug delivery technologies to solve major health problems.
“I am extremely pleased to see that technology developed in Tejal Desai’s group is getting to the point that we can explore this for therapeutic purposes,” said Matthias Hebrok, PhD, the director of the Diabetes Center at UCSF and a member of Encellin’s scientific advisory board. “Encapsulation and protection of islet cells remains a critical hurdle that needs to be overcome before cell therapy becomes a reality in type 1 diabetes.”
Nyitray hopes that when the technology proves effective in treating patients with type 1 diabetes, it will also find applications in other types of cell therapy, where physicians hope to transplant healthy cells from donors, stem cell–derived therapies, or even genetically modified cells derived from patients themselves, to treat diseases such as genetic metabolic disorders.
About Encellin: Encellin is a San Francisco–based biotechnology company developing an implantable cell delivery platform to address the unmet clinical need of type 1 diabetes. Encellin’s technology provides a vehicle for cell therapy and transplantation that both protects donor cells (eliminating the need for immunosuppression), and cell recipients (the cells can be removed or exchanged). Encellin has demonstrated and published proof of concept in small animals, and is looking to accomplish a first-in-human study in the next year to prepare for entry into the diabetes transplant market. From there, Encellin will move into the larger type 1 and type 2 diabetes markets as well as other indications. Encellin has a team of experts committed to the success of this technology and has received recognition through a number of awards, including a 2016 Rosenmann Institute Innovation Award and 2017 Medtech Innovators title for top Northern California startup.
UC San Francisco (UCSF) is a leading university dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care. It includes top-ranked graduate schools of dentistry, medicine, nursing and pharmacy; a graduate division with nationally renowned programs in basic, biomedical, translational and population sciences; and a preeminent biomedical research enterprise. It also includes UCSF Health, which comprises three top-ranked hospitals, UCSF Medical Center and UCSF Benioff Children’s Hospitals in San Francisco and Oakland, and other partner and affiliated hospitals and healthcare providers throughout the Bay Area.