SUMMARY OF INVENTION
This invention is a novel, in vitro method for generating endoderm cells from human embryonic stem cells (hESCs) and/or human induced pluripotent stem cells (hiPSCs). Endoderm cells have the potential to be further differentiated in vitro into a variety of cell types including pancreatic, liver, lung, and thyroid cells, which can then be implanted in vivo for the study and treatment of various disorders.
Investigators have found that the addition of a single small molecule to hPSCs allows for more efficient differentiation of embryonic stem cells to the endoderm lineage; this reduces differentiation variability among different hPSC cell lines. Researchers were able to differentiate these endoderm cells into functional pancreatic beta cells. Patients with Type 1 diabetes do not have sufficient amounts of pancreatic beta cells, whose primary function is to store and release insulin. This novel protocol can yield functionally active pancreatic beta cells in vivo, enabling the study and treatment of Type 1 diabetes.
This technology streamlines the existing protocol by significantly improving the efficiency of endoderm differentiation to greater than 90%
This enables a significant cost savings by allowing for much lower doses (at least 5-fold) of an expensive component of the protocol to be used
Controlled and efficient in vitro differentiation of endoderm cells is critical for the production of pancreatic beta cells for use as potential therapy in patients with Type 1 diabetes
In the U.S., there are approximately three million people living with Type 1 diabetes, according to the Juvenile Diabetes Research Foundation (JDRF). Of these, ~15% are children. There are an estimated ~15,000 children and ~15,000 adults diagnosed per year with Type 1 diabetes.
AREAS OF APPLICATION
Initial application in Type 1 diabetes
STAGE OF DEVELOPMENT
Ready to use
Provisional application 62/377,363 filed August 19, 2016
Danwei Huangfu, PhD, Laboratory Head, Developmental Biology Program, Sloan Kettering Institute, MSK
Eileen Flowers, PhD, Licensing Manager