Omisirge (omidubicel) / Ayrmid - LARVOL DELTA

Outcomes of omidubicel-expanded umbilical cord blood transplantation in patients with severe aplastic anemia (ASH 2025) - P1/2 | "Theconditioning regimen included horse anti-thymocyte globulin (ATG 40 mg/kg, D-11 to -8),cyclophosphamide (60 mg/kg, D-7 and -6), fludarabine (25 mg/m2, D-5 to -1) and 2 Gy total bodyirradiation (TBI) on D-1. Graft versus host disease (GVHD) prophylaxis consisted oftacrolimus/mycophenolate mofetil (MMF)...ResultsFrom August 2017 to June 2025, 18 SAA patients (3 in Cohort 1 and 15 in Cohort 2) who failedATG/cyclosporine/eltrombopag were transplanted...Despite high-risk features, patients experienced extremely rapidand sustained engraftment, early immune recovery, low rates of GVHD, and encouraging survivaloutcomes. These promising interim results warrant further investigation."

Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Neutropenia • Transplantation • CD34

Evaluating gvhd outcomes after chemotherapy-only conditioning in nicotinamide-expanded umbilical cord blood (omidubicel) transplantation: A retrospective cohort study (ASH 2025) - "All patients received conditioning withthiotepa, busulfan, and fludarabine...All receivedGVHD prophylaxis with mycophenolate mofetil and tacrolimus... Our practice with Omi HCT using exclusively chemo-only conditioning showed comparableoutcomes with the pivotal P3 and with the EAP. However, rates of aGVHD appeared lower, and cGVHDwas comparable to the lower rates in the EAP compared to the P3, in which 31/62 (50%) of patients in theOmni group received TBI. We did find increased cGVHD with CMV mismatch and younger DCB units, butnot with other factors previously reported to affect GVHD rates in HCT."

Retrospective data • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Lymphoma • Transplantation

Monte Carlo simulation of HLA-DQ heterodimers and their interactions to predict relapse and GVHD risk in cord blood transplantation (ASH 2025) - "Applied to the omidubicel cord blood cohort, our model identifieda relapse gradient across DQ heterodimer subtypes, with worse outcomes with higher donor group 2burden. These findings show the utility of Monte Carlo simulation for advancing donor selection scienceand hypothesis generation in cord blood transplantation."

Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Transplantation • HLA-C

A pilot study of nicotinamide riboside supplementation in allogeneic bone marrow transplantation (ASH 2025) - P1 | "In the human umbilical cord blood setting, nicotinamide-based cell expansiontechnology (NiCord) has been used ex-vivo to expand the number of donor cells, while maintaining theirfunction. We conclude that NR supplementation is safe and well tolerated in alloBMT with dose dependentincreases in NAD+/NADPH levels that preliminarily are associated with more rapid PE and shorter LOS. These findings may have implications for improved resource utilization and cost-effectiveness in alloBMT,which will be further assessed as the trial proceeds to cohort 4."

Clinical • Bone Marrow Transplantation • Hematological Malignancies • Infectious Disease • Pneumonia • Respiratory Diseases • Transplantation • TINCR

Innovative Transplant Strategies: Omidubicel Interim Phase II Results in Severe Aplastic Anemia (ASH 2025) - "Supported By Gamida Cell For in-person participants only"

P2 data • Anemia • Aplastic Anemia • Hematological Disorders • Transplantation

Additional Positive Results for Omisirge in Treating Severe Aplastic Anemia Presented at ASH (ACCESS Newswire) - "The ongoing open-label, single-center study (NCT03173937|17-H-0091), led by Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), demonstrated highly encouraging results. Among 19 patients (median age 20 years) whose disease had not responded to standard therapies, 18 (95%) achieved rapid neutrophil recovery with a median time of 8 days. Both disease-free survival and overall survival were 94%. Importantly, only 16% of patients experienced (BMT-CTN Grade II) acute GvHD and no cases of severe (BMT-CTN Grade III-IV) acute GVHD or chronic GVHD were observed."

P1/2 data • Aplastic Anemia

FDA approves Omisirge as First Approved Cell Therapy to Treat Severe Aplastic Anemia (ACCESS Newswire) - "This approval is based on the ongoing open-label, single-center study, led by Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH)..."

FDA approval • Aplastic Anemia

HMCT/CT2401: Abatacept GVHD Prophylaxis Following Omidubicel HCT (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: Duke University | Initiation date: Sep 2025 ➔ Dec 2025

Trial initiation date • Graft versus Host Disease • Hematological Malignancies • Immunology • Oncology • Transplantation

Cellular Therapy and HSCT Mobilizers – Omidubicel-onlv (ASH 2023) - No abstract available

Bone Marrow Transplantation

Omidubicel-Onlv for Allogeneic Transplantation (allo-HCT) in Patients with Hematologic Malignancies: Results of a Multicenter Open Label Expanded Access Program (ASH 2023) - P3 | "ConclusionIn a real-world EAP setting, the outcomes of allo-HCT with omidubicel in patients with hematologic malignancies were consistent with those from the phase 3 registration study. These data support the role of omidubicel as a donor source, particularly for patients from diverse racial Background s."

Clinical • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology • Respiratory Diseases • Transplantation

AM25-MN-22-L - CBC: State of the Science Symposium – Striking a New Cord in the Evolution of Stem Cell Transplant (sponsored by Gamida Cell) (AABB 2025) - "The leading academic and clinical experts experts will also present insights into the evolving future of stem cell transplantation. Learning Objectives: Describe the evolution of cord derived cell therapies across a range of hematologic and non-hematologic diseases Describe long term data comparing outcomes with omidubicel to alternative donor sources in hematologic malignancies Describe break through data of Omidubicel in Severe Aplastic Anemia Describe proposals for use of cord and cord derived cell therapies in non-hematologic diseases, including Bone Marrow Failure Syndromes, Inborn errors of Immunity, Inherited Disorders of Metabolism and other novel uses"

Anemia • Aplastic Anemia • Bone Marrow Transplantation • Genetic Disorders • Transplantation

Opening New Horizons: Advanced Hematopoietic Stem Cell Expansion Strategies Bridging Cord Blood Therapy from Bench To Bedside. (PubMed, Stem Cell Rev Rep) - "Novel expanded CB-derived hematopoietic stem and progenitor cells (HSPCs) therapies, including OMISIRGE (Omidubicel onlv.), Zemcelpro (Dorocubicel), and upcoming products with International Nonproprietary Name designations, introduce innovative concepts and comprehensive considerations for improving CB transplantation. This progress enables novel therapeutic options and represents a breakthrough in traditional CB transplants. In this context, we summarize and explore representative techniques and products to provide insights that inspire future developments in CB-derived HSPC therapies."

Journal • Review • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Transplantation

Gamida Cell Presents Positive Initial Results on Treating Severe Aplastic Anemia (SAA) with Omidubicel (Businesswire) - "Prepares for Prescription Drug User Fee Act (PDUFA) target action date of December 10, 2025...Interim results from the study, led by Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), were highly encouraging, with 13 of the 14 patients (92.9%) reaching rapid neutrophil recovery, with a median recovery time of 7 days. The disease-free survival rate, as well as overall survival, was 92.3%, comparing favorably with outcomes from patients who receive transplants from matched donors. Additionally, no severe (Grade III–IV) graft-versus-host disease (GvHD) or chronic GvHD was observed, and only 14% of patients experienced moderate (Grade II) GvHD."

P1/2 data • PDUFA • Aplastic Anemia

HMCT/CT2401: Abatacept GVHD Prophylaxis Following Omidubicel HCT (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: Duke University | Not yet recruiting ➔ Recruiting

Enrollment open • Graft versus Host Disease • Hematological Malignancies • Immunology • Oncology • Transplantation

Ayrmid Ltd…announced that the U.S. Food and Drug Administration (“FDA”) has accepted the Company’s priority review application for omidubicel for the treatment of Severe Aplastic Anemia (“SAA”) and has assigned a Prescription Drug User Fee Act (“PDUFA”) target action date of December 10, 2025. (Businesswire) - "The supplementary Biologics License Application ('sBLA') submission is based on results of an investigator-sponsored study at the National Heart, Lung, and Blood Institute of the National Institutes of Health."

PDUFA • Priority review • Aplastic Anemia

REMOVED: Interim Results of a Phase II Trial of Omidubicel, Ex-Vivo Expanded Umbilical Cord Blood Transplantation in Patients with Treatment-Refractory Severe Aplastic Anemia. (PubMed, Transplant Cell Ther) - "This article has been removed at the request of the author. This abstract has been removed because it was not presented at the 2025 BMT Tandem Meeting."

Journal • P2 data • Preclinical • Anemia • Aplastic Anemia • Hematological Disorders • Transplantation

Expanded Access of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies (clinicaltrials.gov) - P3 | N=36 | Completed | Sponsor: Gamida Cell ltd | Active, not recruiting ➔ Completed

Trial completion • Hematological Malignancies • Oncology • Transplantation

Omidubicel-Onlv: The First Commercially Available Alternative Allogeneic Hematopoietic Stem Cell Transplantation Donor Source. (PubMed, Clin J Oncol Nurs) - "Omidubicel provides an alternative donor source for patients undergoing alloHSCT for whom there is no matched donor available. This need may be particularly great in ethnic and racial minority populations who may be underrepresented in the NMDP BioTherapies database."

Journal • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Oncology • Transplantation

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome (clinicaltrials.gov) - P1/2 | N=37 | Recruiting | Sponsor: National Heart, Lung, and Blood Institute (NHLBI) | Trial completion date: Mar 2028 ➔ Mar 2032

Trial completion date • Anemia • Aplastic Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Neutropenia • Oncology • Transplantation • CD34 • HLA-B • HLA-C

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome (clinicaltrials.gov) - P1/2 | N=37 | Recruiting | Sponsor: National Heart, Lung, and Blood Institute (NHLBI) | Trial primary completion date: Apr 2026 ➔ Apr 2027

Trial primary completion date • Anemia • Aplastic Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Neutropenia • Oncology • Transplantation • CD34 • HLA-B • HLA-C

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome (clinicaltrials.gov) - P1/2 | N=37 | Recruiting | Sponsor: National Heart, Lung, and Blood Institute (NHLBI) | Trial primary completion date: Apr 2025 ➔ Apr 2026

Trial primary completion date • Anemia • Aplastic Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Neutropenia • Oncology • Transplantation • CD34 • HLA-B • HLA-C

BiTE Therapy: Just in Time Education for Newly Approved Medications (ONS 2025) - "One cellular therapy, omidubicel-only, was approved to reduce time to neutrophil recovery in individuals receiving umbilical cord blood transplant after a myeloablative regimen...BiTE therapy has been available since 2014 (blinatumomab) with additional drug approvals in 2021 through 2024... Information for six agents are currently available on the hospital oncology nursing Microsoft Team site. The site is available to all oncology nursing staff at the facility. A copy of the package insert is also posted on the Team site."

Oncology

Omidubicel-onlv Transplantation for Hematologic Malignancies: Results of a Multicenter Expanded Access Program. (PubMed, Transplant Cell Ther) - "This real-world study of omidubicel transplantation for hematologic malignancies finds that this graft source is commonly used for non-white allogeneic transplant recipients. The rapid engraftment kinetics observed following transplantation with omidubicel appears to have addressed excessive non-relapse mortality that has been previously observed following myeloablative umbilical cord blood transplantation."

Journal • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Oncology • Transplantation

Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS (clinicaltrials.gov) - P3 | N=125 | Completed | Sponsor: Gamida Cell ltd | Active, not recruiting ➔ Completed

Trial completion • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Myeloid Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Lymphoma • Myelodysplastic Syndrome • Oncology • Transplantation

Omidubicel-Onlv: The First Commercially Available Alternative Allogeneic Hematopoietic Stem Cell Transplantation Donor Source. (PubMed, Clin J Oncol Nurs) - "Omidubicel provides an alternative donor source for patients undergoing alloHSCT for whom there is no matched donor available. This need may be particularly great in ethnic and racial minority populations who may be underrepresented in the NMDP BioTherapies database."

Journal • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Oncology • Transplantation