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PIPELINE
Driving innovative therapeutics across transplantation
Our proprietary nicotinamide (NAM) technology enhances and expands innate stem cells, offering potentially curative options for patients living with blood cancers and severe aplastic anemia (SAA).
CXCR4 antagonist mobilizes stem cells in patients with Multiple Myeloma who then receive these cells to treat their condition through transplantation. Our CXCR4 antagonist is also being studied to support stem cell mobilization for patients with sickle cell disease who are designated for treatment with gene therapy.
Explore our pipeline below for information on our clinical development candidates and ongoing clinical trials.
The safety and efficacy of motixafortide for stem cell mobilization in Sickle Cell Disease has not been established by the U.S. Food and Drug Administration or any other health authority.
Motixafortide
Motixafortide, already FDA approved for stem cell mobilization in multiple myeloma, is being evaluated in ongoing investigator initiated studies (NCT06442761 and NCT05618301) evaluating CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD).
For Full Prescribing Information on APHEXDA in Multiple Myeloma, please visit aphexda.com.
The safety and efficacy of motixafortide in sickle cell disease has not been established by the U.S. Food and Drug Administration or any other health authority.
NAM Technology
Gamida Cell’s proprietary nicotinamide (NAM) technology expands the number of stem cells while maintaining their intrinsic properties and enhances cellular functionality and phenotype. For stem cells, this may mean improved homing and retention in bone marrow and lymph nodes and increased metabolic fitness. Our technology leverages the properties of NAM, creating allogeneic cell therapy products and candidates that are potentially curative for patients with hematologic malignancies and in Severe Aplastic Anemia.
CXCR4 Antagonist Mechanism of Action
OUR MANUFACTURING
Gamida Cell has a wholly owned, fully licensed GMP manufacturing facility in Israel and have strategic partnership in place for future manufacturing of OMISIRGE® at RoslinCT in Hopkinton, MA.
Selected Publications and Presentations
2025
Improved hematopoietic stem cell mobilization for gene therapy using single agent motixafortide in sickle cell disease.
Blood 2025; 146 (Supplement 1): 2542.
Motixafortide (CXCR4 inhibition) alone and in combination with natalizumab (VLA-4 inhibition) to mobilize hematopoietic stem cells for gene therapy in sickle cell disease
A first-in-human, safety and feasibility study. Blood 2025; 146 (Supplement 1): 5956.
Motixafortide + G-CSF hematopoietic stem cell mobilization in patients with multiple myeloma following quadruplet induction therapy.
Blood 2025; 146 (Supplement 1): 5957.
Outcomes of omidubicel-expanded umbilical cord blood transplantation in patients with severe aplastic anemia.
Blood 2025; 146 (Supplement 1): 5963.
Outcomes of myeloablative allogeneic hematopoietic cell transplantation with omidubicel vs alternative donor sources.
Blood Neoplasia. 2025 2(3):100104.
Omidubicel-onlv Transplantation for Hematologic Malignancies: Results of a Multicenter Expanded Access Program
Transplant Cell Ther. 2025 Jul;31(7):436-447.
2024
2023
Hospitalization and Healthcare Resource Utilization of Omidubicel-Onlv versus Umbilical Cord Blood Transplantation for Hematologic Malignancies: Secondary Analysis from a Pivotal Phase 3 Clinical Trial
Transplant Cell Ther. 2023 Dec;29(12):749.e1-749.e5.
Immune Reconstitution Profiling Suggests Antiviral Protection after Transplantation with Omidubicel: A Phase 3 Substudy.
Transplant Cell Ther. 2023 Aug;29(8):517.e1-517.e12.
Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials
Transplant Cell Ther. 2023 May;29(5):338.e1-338.e6.
Health-Related Quality of Life Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel versus Umbilical Cord Blood
Transplant Cell Ther. 2023 Jan;29(1):52.e1-52.e9.
2022
Gamida Cell Presents New Data Demonstrating Improved and Sustained Health-Related Quality of Life Outcomes for Patients Treated with Omidubicel at the Society of Hematologic Oncology Meeting
Health-Related Quality of Life Following Allogeneic Hematopoietic Stem Cell Transplantation with Omidubicel Versus Standard Umbilical Cord Blood
Presented at the Tenth Annual Meeting of the Society of Hematologic Oncology
2021
Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study
Blood. 2021 Oct 21;138(16):1429-1440.
