We’re enabling the next generation of cancer cell therapy.
We are developing a wholly-owned pipeline of advanced cell therapies with the potential to redefine standards of care across a range of diseases where there is an urgent medical need. Explore our pipeline below for information on our clinical development candidates and ongoing clinical trials.
About Omidubicel
Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU.
Development status
Omidubicel has been studied in an international, multi-center, randomized Phase 3 clinical study evaluating its safety and efficacy compared to standard umbilical cord blood transplant.
The Phase 3 data also show that during the first 100 days of treatment, omidubicel-treated patients had significantly shorter durations of hospitalization, intensive care unit time, consultant visits, procedures, and transfusions than the control arm. These data provide further evidence of the clinical benefit associated with the more rapid hematopoietic recovery in patients treated with omidubicel and the corresponding reduction in healthcare resource utilization.
Full safety and efficacy data from this Phase 3 study have been published in Blood.
In February 2022, we initiated the Biologics License Application (BLA) rolling submission process for omidubicel with the U.S. Food and Drug Administration. We are on track to complete the BLA submission in the second quarter of 2022. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia, a rare and life-threatening blood disorder.
About Omidubicel
Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU.
Clinical Trial Information
Omidubicel is being evaluated in an ongoing investigator-sponsored Phase 1/2 study in patients with severe aplastic anemia.
In a poster presentation at the American Society of Hematology (ASH) 62nd Annual Meeting in December 2020, it was shown that patients with severe aplastic anemia treated with omidubicel achieved sustained early engraftment. These data are the first evidence that omidubicel can result in rapid engraftment and can achieve sustained hematopoiesis in patients who are at high risk for graft failure with conventional umbilical cord blood transplant.
For more information about the study, please visit www.clinicaltrials.gov.
About GDA-201
GDA-201 is an innate natural killer (NK) cell immunotherapy candidate for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. When combined with targeted antibodies, GDA-201 has shown enhanced antibody-dependent cellular toxicity, or ADCC. In April 2022, the U.S. Food and Drug Administration (FDA) cleared our investigational new drug (IND) application and removed the clinical hold for a cryopreserved formulation of GDA-201.
Clinical Trial Information
The study of the cryopreserved formulation of GDA-201 is currently open to enrollment at Henry Ford Cancer Institute (Detroit, MI) and the Masonic Cancer Center at the University of Minnesota, and additional sites will be added in the coming months. The Phase 1 portion of the study is designed to evaluate the safety of GDA-201 in patients with follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL)/high grade B-cell lymphoma (HGBCL), marginal zone lymphoma or mantle cell lymphoma. The Phase 2 expansion phase is designed to evaluate the safety and efficacy of GDA-201 in two patient cohorts, FL and DLBCL/HGBCL. The study will include patients who have relapsed or refractory lymphoma after at least two prior treatments. For more information about the study, please visit www.clinicaltrials.gov (NCT03019666).
GDA-201 has been evaluated in a Phase 1 investigator-led study in patients with refractory non-Hodgkin lymphoma (NHL) and multiple myeloma, demonstrating that GDA-201 generally was well tolerated in 35 patients. Of the 19 patients with NHL, 13 complete responses and one partial response were observed (CRR = 68%, ORR = 74%). No dose-limiting toxicities were observed.
Two-year follow up data in the same Phase 1 study demonstrated an overall survival rate of 78% at two years, with a median duration response of 16 months (Bachanova, et al. ASH 2021. Abstract #3854).
Gamida Cell is pioneering a diverse approach to cellular therapy that utilizes nicotinamide (NAM) to expand multiple cell types — including stem cells and natural killer (NK) cells — while maintaining their original phenotype and potency. When applied to umbilical cord blood–derived cells in the production of our investigational omidubicel product for allogeneic stem cell transplant, NAM-enabled cells have improved clinical measures such as engraftment time and infection rate, potentially leading to better outcomes for patients.
NK cells are cytotoxic lymphocytes of the innate immune system capable of killing virally infected and/or cancerous cells, and can be engineered using CAR and gene editing to increase targeting and activation. After expansion in culture however, NK cells typically lose some of their ability to traffic, localize and proliferate in vivo. This has been suggested as a major cause for the poor survival of adoptively transferred NK cells and their inability to overcome immune resistance in the tumor microenvironment. To address this obstacle, Gamida Cell has developed a reliable, scalable and GMP-compliant culture method for NAM expansion that yields highly functional NK cells. These cells have demonstrated ability to kill cancerous cells in both animal models and clinical trials, and are being developed in both unmodified and genetically engineered forms.
PEGS 2021
Gamida Cell 2021 Virtual R&D Day
Comparative effectiveness and safety of omidubicel versus other current allogeneic hematopoietic stem cell donor sources using network meta-analysis
AMCP Nexus 2021
Improved Clinical Outcomes with Omidubicel versus Standard Myeloablative Umbilical Cord Blood Transplantation: Results of a Phase III Randomized, Multicenter Study
Presented at the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2021)
Improved Clinical Outcomes with Omidubicel versus Standard Myeloablative Umbilical Cord Blood Transplantation: Results of a Phase III Randomized, Multicenter Study
Presented at the 2021 TCT Meetings of ASTCT and CIBMTR
Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer (NK) Cells in Patients with Refractory Non-Hodgkin Lymphoma and Multiple Myeloma
Rapid Engraftment, Immune Recovery, and Resolution of Transfusion Dependence in Treatment-Refractory Severe Aplastic Anemia Following Transplantation with Ex Vivo Expanded Umbilical Cord Blood (Omidubicel)
Impact of Donor Age on HSCT Outcomes
Nicotinamide (NAM) Modulates Transcriptional Signature of Ex Vivo Cultured UCB CD34+ Cells (Omidubicel) and Preserves Their Stemness and Engraftment Potential
Presented at the 2020 TCT Annual Meeting.
Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer Cells (NAM-NK) in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma (MM)
Presented at the 2019 ASH Annual Meeting.
Rapid and Robust CD4+ and CD8+ T-, NK-, B-Cell, Dendritic Cell, and Monocyte Reconstitution after Nicotinamide-Expanded Cord Blood Transplantation.
Presented at the 2019 Transplantation & Cellular Therapy (TCT) Meetings of American Society for Blood and Marrow Transplantation (ASBMT) and Center for International Blood and Marrow Transplant Research (CIBMTR).
First-in-Human Phase I Study of Nicotinamide-Expanded Related Donor Natural Killer Cells for the Treatment of Relapsed/Refractory Non-Hodgkin Lymphoma and Multiple Myeloma
Presented at the 2019 TCT Meetings of ASBMT and CIBMTR.
Ex Vivo Nicotinamide-Expanded (NAM-Expanded) Unrelated Cord Blood (UCB) Transplantation for Refractory Severe Aplastic Anemia Results in Rapid Engraftment and Expedites Immune Recovery
Presented at the 2019 TCT Meetings of ASBMT and CIBMTR.
Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo with Nicotinamide
Journal of Clinical Oncology 2019 Feb 10;37(5):367-374.
Phase I Study of Nicotinamide-Expanded Related Donor Natural Killer (NK) Cells for the Treatment of Relapsed/Refractory CD20+ non-Hodgkin Lymphoma
Presented at the 2018 International Meeting on Advances in Malignant Lymphoma.
Presented at the 2017 ASH Annual Meeting.
NiCord Single Unit Expanded Umbilical Cord Blood Transplantation (UCBT): Final Results of a Multicenter Phase I/ II Trial
Presented at the 2017 ASH Annual Meeting.
Transplantation of Ex Vivo Expanded Umbilical Cord Blood (NiCord) Decreases Early Infection and Hospitalization
Biol Blood Marrow Transplant. 2017 Jul;23(7):1151-1157.