First Patient Successfully Transplanted with Cryopreserved (Frozen) NiCord® in Gamida Cell’s Ongoing Phase I/II Clinical Trial for Blood Cancers. This is the first transplantation to use a graft of ex-vivo expanded, cryopreserved, cord blood progenitor cells

Jerusalem, Israel, January 8, 2015 — Gamida Cell, a leader in cell therapy technologies and products for transplantation and adaptive immune therapy, announced today that the first person has been successfully transplanted with cryopreserved (frozen) NiCord® in the company’s ongoing Phase I/II clinical study for blood cancer patients. This is the first time that a patient has been successfully transplanted with umbilical cord blood stem and progenitor cells, that were expanded (population increased) ex-vivo (outside of the body), and cryopreserved. The company also noted today that after thaw, the cryopreserved product maintained the advantage of NiCord® in demonstrating very rapid engraftment (white blood cell recovery). It is expected that this rapid engraftment will reduce the risk of opportunistic infections, will lower the morbidity associated with cord blood transplantation and shorten hospitalization.

“This is a significant technological milestone in the evolution of the development of NiCord®. Moving forward, in all ongoing and future clinical trials, NiCord® will be used only in its cryopreserved formulation. Until now, NiCord® has been transplanted as a “fresh” product that must be infused into the patient within a limited number of hours from the moment the product is released from the manufacturing site. This timetable restricted the window for transplantation for the patient. It also limited the location of manufacturing sites to the vicinity of participating medical centers and increased the cost of shipping and logistical support required from manufacturing through delivery. To overcome these limitations, Gamida Cell developed a cryopreserved formulation of the product with a long shelf life,” said Dr. Yael Margolin, president and CEO of Gamida Cell.

Dr. Margolin continued, “The cryopreserved formulation of NiCord® provides several key benefits. It enables flexibility in the timing of a transplantation, which can now be determined according to the clinical conditions of the patient. If a delay is required, it is now possible and more easily managed. This flexibility in timetable also means that the product can now be distributed more efficiently and worldwide, enabling the company to expand its clinical trials to all geographic areas. NiCord® can also now be manufactured in a preferable, centralized location, which will significantly reduce the cost of goods.”

NiCord® is currently manufactured and shipped to the US and Europe from Gamida Cell in Jerusalem.

NiCord® is derived from a single umbilical cord blood unit which has been expanded in culture and enriched with stem and progenitor cells using Gamida Cell’s proprietary NAM technology. NiCord® is in development as an investigational therapeutic treatment for blood cancers such as leukemia and lymphoma and for non-malignant hematological diseases. Clinical sites in the US, Spain, Italy, Israel and the Netherlands will collectively enroll 20 patients, with hematologic malignancies following myeloablative therapy, in the Phase I/II study of NiCord®, in a single cord configuration.