First Pediatric Sickle Cell Disease Patient Receives NiCord® Stem Cell Transplantation in Gamida Cell Pilot Study at Duke University

erusalem, Israel, November 12, 2012 — Gamida Cell Ltd., a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine, announced today that the first patient, enrolled in its pilot study of NiCord® as an investigational treatment for sickle cell disease (SCD), has been transplanted at Duke University in North Carolina.
Gamida Cell CEO Dr. Yael Margolin said, “The NiCord® study is a first step in broadening Gamida Cell’s pipeline of cell therapies to treat patients who suffer from severe non-malignant diseases with a very large unmet clinical need.”
SCD is a group of inherited red blood cell disorders where red blood cells become hard and sticky and look like a C-shaped farm tool called a ‘sickle’.   According to statistics, SCD affects 90,000 to 100,000 in the US alone, mainly African-Americans and Hispanic-Americans. Symptoms range in type and severity.  SCD can be fatal and to date, the only cure for SCD is stem cell transplantation from a family related matched donor.
Dr. Margolin continued, “Sickle cell disease can be cured with a successful bone marrow transplantation, especially from a family related fully matched donor.  Most patients do not pursue this option, since they do not have the suitable donor.  NiCord® is intended to reverse this situation and provide a readily available cure.”

NiCord® is an expanded cell graft derived from an entire unit of umbilical cord blood and enriched with stem cells. NiCord was developed based on Gamida Cell’s proprietary NAM technology. 
The official name of the study is Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Combination With a Second, Unmanipulated Cord Blood Unit in Patients With Sickle Cell Disease.   A total of 10 patients, ages 2 – 21, will be enrolled in the NiCord study, a single center, single arm trial evaluating the safety and efficacy of transplanting NiCord® together with a second un-manipulated cord blood unit in patients with SCD following myeloablative therapy.  The study will also assess transplant-related mortality, event-free survival and overall survival at 100, 180 and 365 days, respectively.