Gamida Cell identified sirtuins (SIRTs), which are type-III lysine deacetylases of histones and of numerous transcription factors, as new stem cell targets. Gamida Cell's pre-clinical studies demonstrate that controlling the function of SIRTs with small molecules modulates stem cell fate, extends their ability to replicate and increases the number of blood-building stem cells known as hematopoietic stem cells.
Based on these findings, Gamida Cell has developed a novel propriety technology for expansion of functional hematopoietic stem cells in in vitro cultures. The lead molecule of this technology is Nicotinamide (NAM), a Vitamin B3, a potent modulator of SIRT1 activity as well as a potent inhibitor of NAD+ -dependent ADP ribosyl transferase enzymes.
Pre-clinical studies show the tremendous potential of this technology to increase stem cell migration, homing and engraftment efficacy, to rescue lethally irradiated primary and secondary recipients and to enhance myeloid and lymphoid reconstitution by maximizing the full therapeutic potential of bone marrow transplantation, tissue regeneration and gene therapy.
To view pre-clinical data obtained with two models for bone marrow transplantation click here.
In December 2006 and 2008, Gamida Cell addressed the American Society of Hematology in oral and poster presentations on the recent pre-clinical study results of its NAM technology. Currently, the company is focused on applying this technology in the development of new products including NiCord.
To view the abstracts of this study, please click here.
*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. |