One of the great limitations of therapies based on stem cells derived from umbilical cord blood is the small number of stem/progenitor cells available in any given cord blood unit. This small number severely limits its use in bone marrow transplantation in adolescents and adults.
To overcome this limitation, Gamida Cell has developed different proprietary platform technologies to increase the number of stem cells in culture with limited differentiation. This means that most of the new stem cells are identical to those in the original population. Gamida Cell's epigenetic* technologies for expansion of populations of functional hematopoietic (blood) progenitor cells (HPC) utilize small molecules to modulate differentiation, homing and the engraftment abilities of cultured cells.
Gamida Cell is applying these expansion technologies to develop a pipeline of products with therapeutic doses of effective stem/progenitor cells for bone marrow transplantation and regenerative medicine. Gamida Cell is the only company to demonstrate, in the laboratory and the clinic, significant expansion of populations of stem cells in culture. The results of the Phase I/II study of the first developed technology, TEPA, prompted the registration ExCell study, a global, multi-center, pivotal phase II/III study, and its further investigation of the safety and efficacy of StemEx®, Gamida Cell's flagship product.
Gamida Cell's enabling technologies are simple, reversible, do not involve genetic interference, and are easily scalable. They are additionally cost effective and efficient for commercial production.
Gamida Cell's technologies and products are protected by more than 20 granted patents worldwide and numerous patent applications.
*In biology, the term epigenetics refers to changes in phenotype (appearance) or gene expression caused by mechanisms other than changes in the underlying DNA sequence, hence the name epi- (Greek: over; above) -genetics. Wikipedia
Rationale
Successful in vitro (outside the living body and in an artificial environment) expansion of hematopoietic (blood) stem/progenitor cells (HPC) depends on the ability of these cells to multiply while maintaining their basic characteristics. Most protocols for ex vivo expansion utilize various combinations of cytokines that support extensive proliferation of cultured cells. Nonetheless, in vitro proliferation is tightly coupled with commitment and differentiation, thereby lessening the clinical utility of the cultured cells. Thus, culture conditions attenuating in vitro differentiation have been employed to support a more successful expansion of HPC populations and to increase their clinical applicability.
Gamida Cell's proprietary technologies for in vitro expansion of populations of HPC utilize low molecular weight compounds, which regulate the physiological pathways of cell differentiation. Two of the four technologies have been presented in scientific forums. | 
