NAM Technology

Gamida Cell’s proprietary NAM technology is uniquely designed to provide a curative approach to treating a wide range of cancers and genetic diseases.

NAM technology is based on epigenetic regulation by the small molecule, Nicotinamide (NAM), and seeks to overcome challenges associated with expanding cells in a culture while preserving their characteristics and functionality, which typically decrease in culture conditions. This technology addresses these two limitations of ex vivo expansion by:

• Inhibiting rapid cell differentiation in culture, resulting in expansion of a higher number of progenitor and stem cells, including CD34+ and CD34+CD38-Lin-, respectively
• Diminishing the changes in cell characteristics, therefore enhancing the functionality of CD34+ cells expanded in culture conditions, including their migration, homing and engraftment in the bone marrow

NAM Technology in Action

Gamida Cell has applied NAM technology to develop its lead clinical program by expanding blood forming stem and progenitor cells from umbilical cord blood to create highly functional, stand-alone grafts that serve as a universal bone marrow transplant solution.

Why use cord blood?

Given several limitations associated with bone marrow transplants, Gamida Cell developed its clinical assets using umbilical cord blood, a viable alternative graft source in which:

• There is worldwide availability from public cord blood banks
• Full tissue matching is not required, and as a result, cord blood can be used in nearly all patients in need
• There tends to be low incidence of severe Graft-Versus-Host-Disease (GvHD)
• Graft vs. leukemia effect is high

Current cord blood limitations: The use of standard cord blood for transplantation is limited because a unit of cord blood has a low number of stem and progenitor cells, resulting in delayed engraftment, a higher rate of graft failure, increased morbidity, prolonged hospitalization and higher transplant-related mortality.

Gamida Cell has successfully harnessed its NAM platform technology to leverage the advantages of cord blood and overcome its limitations by increasing the number of CD34+ stem and progenitor cells, while enhancing their homing and engraftment efficiency.

Gamida Cell has robust, cost-effective manufacturing capabilities, applying the proprietary NAM technology to create a large number of functional CD34+ cells for NiCord®.