PIPELINE
We’re expanding what’s possible with 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.
- CANDIDATE
- PRECLINICAL
- PHASE 1
- PHASE 2
- PHASE 3
Omidubicel
High-risk Hematologic Malignancies
Read MoreHigh-risk Hematologic Malignancies
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
In May 2020, we reported topline results from an international, multi-center, randomized Phase 3 clinical study in 125 patients demonstrating that omidubicel was generally well tolerated and the median time to neutrophil engraftment was significantly shorter for patients who were randomized to omidubicel than those in the comparator group. In October 2020, we reported that the study met all three of its secondary endpoints related to platelet engraftment, infections, and hospitalizations, which are key measures in bone marrow transplant. These secondary endpoints were the proportion of patients who achieved platelet engraftment by day 42, the proportion of patients with grade 2 or grade 3 bacterial or invasive fungal infections in the first 100 days following transplant, and the number of days alive and out of the hospital in the first 100 days following transplant. All three secondary endpoints demonstrated statistical significance in an intent-to-treat analysis.
We anticipate reporting the full data set in a peer-reviewed setting in the first half of 2021 and expect to initiate the submission of a biologics license application (BLA) filing, on a rolling basis, in the fourth quarter of 2020.
For more information about the details of the secondary endpoints, please see here.
Severe Aplastic Anemia
Read MoreSevere Aplastic Anemia
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.
GDA-201
Non-Hodgkin Lymphoma, Multiple Myeloma
Read MoreNon-Hodgkin Lymphoma, Multiple Myeloma
About GDA-201
GDA-201 is an innate natural killer (NK) cell immunotherapy 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.
Clinical Trial Information
GDA-201 is being evaluated in a Phase 1 clinical study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.
In an oral presentation at the American Society of Hematology (ASH) 62nd Annual Meeting in December 2020, it was shown that GDA-201 was well tolerated and no dose-limiting toxicities were observed in the Phase 1 clinical study. GDA-201 demonstrated significant clinical activity in patients with non-Hodgkin lymphoma, with 13 complete responses and one partial response observed in 19 patients, for a response rate of 74 percent. Details of the presentation can be found in the press release.
For more information about the study, please visit www.clinicaltrials.gov.
PLATFORM
Cell-based therapies are recognized for their tremendous potential to provide curative treatment options for patients with blood cancers and rare, serious blood disorders. However, efforts to create effective treatments to date have been hampered by the inability to expand cells in the laboratory while maintaining therapeutic functionality.
At Gamida Cell, we’re pioneering a new approach with the potential to improve treatment outcomes beyond what is possible with donor-derived therapies. Our proprietary platform allows us to expand multiple cell types — including stem cells and natural killer (NK) cells — while maintaining their original phenotype and potency. This approach enables us to administer a therapeutic dose of cells to patients.
Selected Publications and Presentations
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
2020
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.
2019
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.
2018
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.
2017
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.
