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PIPELINE
We’re enabling the next generation of cancer cell therapy.
We are developing a proprietary pipeline of NAM-enabled 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.
- PRODUCT
- DISCOVERY
- PHASE 1
- PHASE 2
- PHASE 3
Omidubicel
Hematologic Malignancies
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High-risk Hematologic Malignancies
About Omidubicel
Omidubicel is a NAM-enabled stem cell therapy candidate 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 study achieved its primary endpoint (p<0.001). In the intent-to-treat analysis, median time to neutrophil engraftment was 12 days for patients receiving omidubicel (95% CI: 10-15 days) compared to 22 days for the comparator group (95% CI: 19-25 days).
- Omidubicel was generally well tolerated. Among patients who were transplanted per protocol, rates of acute and chronic graft-versus host disease were similar and cumulative incidence of infections was significantly smaller in omidubicel compared to controls for both viral infections and bacterial or invasive fungal infections.
- The study also met all three of its secondary endpoints, improving platelet engraftment, and reductions in infections and hospitalizations, which are key measures for success for bone marrow 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.
On August 1, the U.S. Food and Drug Administration accepted for filing the Company’s Biologics License Application for omidubicel for the treatment of patients with blood cancers in need of an allogenic hematopoietic stem cell transplant. The FDA granted Priority Review for the BLA and has set a target action date of May 1, 2023.
Severe Aplastic Anemia
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Severe 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
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Non-Hodgkin Lymphoma, Multiple Myeloma
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 is a multi-center, open label trial evaluating the cryopreserved formulation of GDA-201. It is currently open to enrollment. 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).
Non-Hodgkin Lymphoma
Trial 2
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PLATFORM
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.
Selected Publications and Presentations
2023
Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials
Transplantation and Cellular Therapy 2023 Feb 10; In Press, Uncorrected Proof
Tumor Microenvironment Spatial Analysis after Adoptive NK Cell Therapy for Lymphoma Revealed Cross-Talk with Adaptive T-Cell Immunity
2023 Tandem Meetings of the ASTCT and CIBMTR
Longitudinal Immune Reconstitution Profiling Suggests Anti-Viral Protection After Transplantation With Omidubicel: A Phase 3 Substudy
2023 Tandem Meetings of ASTCT and CIBMTR
GDA-201: A Cryopreserved, Readily Available Formulation of Nicotinamide-Enabled Natural Killer Cells, Shows High Potency and Cytotoxicity In Vitro
2023 Tandem Meetings of ASTCT and CIBMTR
2022
Clinical outcomes following allogeneic hematopoietic cell transplantation (HCT) with omidubicel or other donor sources in patients with hematologic malignancies
Presented at the 64th Annual Meeting of the American Society of Hematology
Gamida Cell Presents New Data Demonstrating Promising Antitumor Activity Against HER2+ Cancers at SITC 37th Annual Meeting
Engineered NAM-NK cells with HER2-CAR expression demonstrate increased cytotoxicity against HER2-expressing solid tumors
Presented at the Society for Immunotherapy of Cancer's 37th Annual Meeting
Gamida Cell Presents New Data Demonstrating Overall Survival and Sustained Long-Term Outcomes for Patients Treated with Omidubicel at the Society of Hematologic Oncology Meeting
Multicenter Long-Term Follow Up of Allogeneic Hematopoietic Stem Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials
Presented at the Tenth Annual Meeting of the Society of Hematologic Oncology
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
Gamida Cell Presents Data Demonstrating the Impact of Transplantation with Omidubicel for Patients with Hematologic Malignancies at 2022 Cord Blood Connect Meeting
Health-Related Quality of Life Following Transplantation with Omidubicel Versus Umbilical Cord Blood in Patients with Hematological Malignancies: Results from a Phase III Randomized, Multicenter Study
Presented at the 2022 Cord Blood Connect Meeting
Gamida Cell Presents Data Demonstrating Projected Impact of Omidubicel on Racial and Ethnic Disparities at 2022 Cord Blood Connect Meeting
Projected Impact of Omidubicel on Racial and Ethnic Disparities in Allogeneic Hematopoietic Cell Transplant Access and Outcomes for Patients with Hematologic Malignancies in the US
Presented at the 2022 Cord Blood Connect Meeting
Gamida Cell Data Reports Omidubicel Leads to Robust Recovery and Diversity of T cells at 2022 Cord Blood Connect Meeting
Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel Leads to Robust Recovery and Diversity of T cells
Presented at the 2022 Cord Blood Connect Meeting
Gamida Cell Presents Updated Omidubicel Data During Best Abstract Award Session at TCT 2022
Allogeneic Hematopoietic Stem Cell Transplant with Omidubicel Demonstrates Sustained Clinical Improvement Versus Standard Myeloablative Umbilical Cord Blood Transplantation
Presented at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings
Gamida Cell Presents Updated One-Year Follow Up Data from Phase 3 Study of Omidubicel at TCT 2022
Hematopoietic Stem Cell Transplantation with Omidubicel is Associated with Enhanced Circulatory Plasmacytoid Dendritic Cells, NK Cells and CD4+ T Cells with Lower Rates of Severe Infections Compared to Standard Umbilical Cord Blood Transplantation
Presented at the 2022 Transplantation & Cellular Therapy Meetings of ASTCTand CIBMTR Tandem Meetings
Gamida Cell Presents New Data on NAM-Enabled Genetically Modified Natural Killer Cell GDA-301 at ISCT 2022
Engineered NAM-NK Cell GDA-301 via CISH Knockout Membrane-Bound IL-15 Expression Increase Cytotoxicity Against Malignancies
Presented at the 2022 International Society for Cell & Gene Therapy
Gamida Cell Presents New Data on NAM-Enabled Genetically Modified Natural Killer Cell GDA-601 at ISCT 2022
NAM-NK Cell GDA-601 With CD 38 Knockout Expresses Enhanced CD38 Chimeric Antigen receptor and targets Multiple Myeloma Cells With Increased Cytotoxicity
Presented at the 2022 International Society for Cell & Gene Therapy
AACR Advances in Malignant Lymphoma
Development of Nicotinamide Enhanced NK cell Therapeutics for Lymphoma
Presented at the 2022 Third International Meeting for AACR Advances in Malignant Lymphoma
2021
Shifting From T to NK Immunotherapy
PEGS 2021
Pioneering Next-Generation NK Cell Therapies
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
Comparative effectiveness and safety of omidubicel versus other current allogeneic hematopoietic stem cell donor sources using network meta-analysis
AMCP Nexus 2021
Results of Omidubicel Phase 3 Clinical Study in Patients with Hematologic Malignancies Presented at EBMT
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)
Results of Omidubicel Phase 3 Clinical Study in Patients with Hematologic Malignancies Presented at TCT
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 GDA-201 Phase 1 Clinical Study Presented at ASH
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
Results of Omidubicel Phase 2 Clinical Study in Patients with Severe Aplastic Anemia Presented at ASH
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)
Observational Data Presented at Cord Blood Connect
Impact of Donor Age on HSCT Outcomes
NAM Mechanism of Action Data Presented at TCT
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
Phase 1 Data on GDA-201 Presented at ASH
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.
Phase 1/2 Translational Data on Omidubicel Presented at TCT
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).
Phase 1 GDA-201 Data Presented at TCT
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.
Initial Phase 1/2 Data on Omidubicel in Aplastic Anemia Presented at TCT
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 1/2 Data on Omidubicel Published in JCO
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 1 Data on GDA-201 Presented at AACR
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
ASH Presentation on Cell-Expansion Platform for NK Cells
Presented at the 2017 ASH Annual Meeting.
Phase 1/2 Omidubicel Data Presented at ASH
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.
Omidubicel Clinical Outcomes Data Published in BBMT
Transplantation of Ex Vivo Expanded Umbilical Cord Blood (NiCord) Decreases Early Infection and Hospitalization
Biol Blood Marrow Transplant. 2017 Jul;23(7):1151-1157.