ERUSALEM, Israel – Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, announced today that the first patient with sickle cell disease (SCD) has been transplanted with CordIn™. The transplant took place at UCSF Benioff Children’s Hospital Oakland. Mark Walters, MD, Director of the Blood and Marrow Transplantation (BMT) Program is the Principal Investigator.
CordIn is an experimental curative treatment for rare non-malignant diseases where bone marrow transplantation is the only currently available cure. These include hemoglobinopathies such as SCD and thalassemia, bone marrow failure syndromes such as aplastic anemia, genetic metabolic diseases and refractory autoimmune diseases. This unmet medical need represents a multi billion dollar market potential.
“The successful enrollment and transplantation of our first SCD patient with CordIn as a single graft marks an important milestone in our clinical development program. We are eager to demonstrate the potential of CordIn as a transplantation solution to cure SCD and to broaden accessibility to patients with rare genetic diseases in need of bone marrow transplantation,” said Gamida Cell CEO Dr. Yael Margolin. “In the first Phase 1/2 study with SCD, the expanded graft was transplanted along with a non-manipulated umbilical cord blood unit in a double graft configuration. In the second phase 1/2 study we updated the protocol from our first Phase 1/2 study so that patients would be transplanted with CordIn as a standalone graft, which is expanded from one full umbilical cord blood unit and enriched with stem cells using the company’s proprietary NAM technology.