Expanding functional cells in culture is a challenge, as cells tend to lose some of their functionality, once removed from their natural tissue microenvironment. Additionally, expanding stem cells is normally associated with their accelerated differentiation followed by loss of “stem” characteristics.
Our proprietary technology, which is based on epigenetic regulation by the small molecule NiCotinamide (NAM), has successfully overcome these challenges and is already being applied to several cell types and pipeline products.
The leading application of the NAM technology is in expanding blood forming stem and progenitor cells from the umbilical cord blood to create a highly functional graft for bone marrow transplantation. NK cells are also expanded for immune therapy of cancer.
Bone marrow transplant is a life-saving treatment for patients with high risk blood cancers and genetic diseases such as sickle cell disease and thalassemia. Bone marrow transplantation requires that the patient have a fully matched donor; however, many patients cannot find a match.
Umbilical cord blood (UCB) offers a viable alternative graft source for transplantation as only partial matching is required, making it available to practically all patients in need. However the use of cord blood for transplantation is limited, because a unit of UCB has a low number of stem and progenitor cells, resulting in a higher rate of graft failure, delayed engraftment, increased morbidity, prolonged hospitalization and higher transplant related mortality.
Gamida Cell’s proprietary NAM technology increases the limited population and the quality of stem and progenitor cells in such a way that the end product is enriched with a therapeutic quantity of these highly functional cells.
NAM technology addresses the two limitations of ex vivo expansion of blood forming stem cells for transplantation:
NAM technology has been applied to develop NiCord®, a stand-alone graft for bone marrow transplantation in patients with blood cancer, and CordIn™, a graft for bone marrow transplantation in patients with rare genetic blood conditions such as sickle cell disease and thalassemia; bone marrow failure syndromes such as aplastic anemia and rare genetic metabolic diseases.
Clinical data obtained to date from several clinical studies in US and EU shows that NiCord® and CordIn™ substantially shorten time to recovery from transplantation due to rapid engraftment of the ex vivo expanded grafts. This has translated into a decrease in morbidity, transplant related mortality and duration of hospitalization. The products have also demonstrated to increase the probability of successful engraftment in hard to engraft patients suffering from rare blood genetic diseases.
NAM-NK cells for adaptive immune therapy: The implementation of the technology is not restricted to cord blood derived stem cells. NAM technology was further demonstrated to increase the functionality of additional ex vivo expanded cells. Based on pre-clinical findings, a product for adaptive immune therapy of cancer comprised of natural killer cells (NK cells), a subset of lymphoid cells, was developed for patients suffering from refractory blood and solid cancers. First clinical study will begin at H1 2017 for patients with multiple myeloma and Non-Hodgkin lymphoma.