Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, announced that the first subject has been transplanted in an investigator-initiated study of Cordin for patients with severe aplastic anemia (AA) or hypoplastic myelodysplastic syndrome (MDS) who have no available matched donor.

The study is being conducted by Dr. Richard W. Childs, Clinical Director of the National Heart, Lung, and Blood Institute’s (NHLBI) Division of Intramural Research in the National Institutes of Health (NIH) and Assistant United States Surgeon General.

“Severe aplastic anemia and myelodysplastic syndrome are life-threatening bone marrow disorders with few optimal treatment options. Many patients with these diseases fail conventional therapy. Amongst those with severe AA who respond to conventional treatment, up to 30% will suffer relapse or evolve to myelodysplastic syndrome or leukemia which is often fatal,” said Dr. Childs. “Promising preclinical and clinical data have shown the efficacy of Gamida Cell’s ex-vivo hematopoietic stem cell technology. Based upon exciting prior data, we are now conducting a clinical trial at the NHLBI testing whether umbilical cord blood transplantation using CordIn can be used to improve the results of conventional cord blood transplantation for patients with these life-threatening conditions who lack an available matched donor,” said Dr. Childs.

“The study, announced today, represents a significant milestone for Gamida Cell, as we continue our important mission of expanding access to curative transplantation in patients for which engraftment has been historically difficult,” said Gamida Cell President and CEO Dr. Yael Margolin. “We highly value our collaboration with the NIH and with Dr. Childs, who is one of the world experts in the field, and we look forward to evaluating the potential of CordIn in patients with life-threatening hematologic diseases.”

The study is titled Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome. The primary endpoint of the Phase 1/2 trial is prompt and durable cord engraftment in patients transplanted with CordIn. Additional information can be accessed here on the Clinicaltrials.gov website.