Headquartered in Jerusalem, Israel, Gamida Cell is best known for its innovative approaches to hematopoietic stem cell therapeutics. Although a small company at the time, Gamida Cell broke onto the global scene in 2003 when it announced a Phase I trial of its StemEx™ product for the treatment of leukemia and lymphoma patients.
StemEx™ was Gamida Cell’s first generation product for theex vivo (“outside the body”) expansion of cord blood stem cells, which can be utilized in hematopoietic stem cell transplantation (HSCT) to treat high risk cancers. Although StemEx™ did not make it beyond Phase II/III trials, this expertise in ex vivo expansion of cord blood lead to the development of Gamida Cell’s current flagship product, NiCord®, and established Gamida Cell as a global technology leader for functional expansion of cells.
Gamida Cell’s lead product, NiCord®, offers patients in need of HSCT an alternative to the existing approach of bone marrow transplant by using ex vivo expansion of cord blood. NiCord® has an orphan drug designation. NiCord®, CordIn™ and other products under development by Gamida Cell, all utilize the company’s proprietary NAM platform technology to expand cells while preserving, and surprisingly, even enhancing their functionality.
Cord blood has many advantages over the use of peripheral blood for HSCT, including that it does not require a full tissue matching, it is widely available through a global network of public cord blood banks, and it exhibits low rates of graft versus host disease (GVHD). However, the small quantity of blood obtained from an umbilical cord, usually about 50-60mL, and the small number of cells, results in delayed engraftment of neutrophils and platelets.
While double cord blood transplantation is being used as an alternative, the use of multiple units is very expensive, it can increase GvHD incidence rates, and the approach does not meaningfully improve transplant outcomes, including time to neutrophil and platelet engraftment.
NiCord® is “an ex vivo expanded cell graft utilizing Gamida Cell’s proprietary NAM platform technology to expand cells from the umbilical cord blood.” In clinical trials so far, NiCord® has demonstrated rapid engraftment and clinical outcomes that appear comparable to transplantation of peripheral blood.
Therefore NiCord® has the potential to introduce a paradigm shift to HSCT by providing a transplantation option that could be available to practically all patients in need.
NiCord® could become a transplant of choice for patients who do not have an appropriate graft. These include patients that cannot find a matched bone marrow donor, patients that cannot wait 3-4 months to find an unrelated bone marrow match, and patients that are choosing experimental approaches because they have no other viable options.
These groups represent approximately 60-80% of the total patient population requiring a HSCT each year for high risk cancers.
To learn more about Gamida Cell’s technology, partnerships, and intellectual property portfolio, click here to read my recent interview with CEO of Gamida Cell, Dr. Yael Margolin.