Omisirge (omidubicel) / Ayrmid, ACA Pharma - LARVOL DELTA

Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel Is Associated with Enhanced Circulatory Plasmacytoid Dendritic Cells (pDC), NK Cells and CD4+ T Cells with Lower Rates of Severe Infections Compared to Standard Umbilical Cord Blood Transplantation (TCT-ASTCT-CIBMTR 2022) - P3 | "Significantly lower rates of severe bacterial infections in the same period likely reflect the effect of shortened duration neutropenia. We hypothesize that the lower rates of bacteremia and sepsis permitted superior survival of these immune cells despite the lower infused cell dose, potentially contributing to the lower viral infection rate observed in the omidubicel cohort."

Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Neutropenia • Oncology • Septic Shock • Transplantation • CD133 • CD4

Health-Related Quality of Life (HRQL) Following Transplantation with Omidubicel Versus Umbilical Cord Blood (UCB) in Patients with Hematologic Malignancies: Results from a Phase III Randomized, Multicenter Study (TCT-ASTCT-CIBMTR 2022) - P3 | "Averaging across the first year post-transplant, patients receiving omidubicel experienced statistically significantly better HRQL for physical and functional well-being domains (P<0.05 for comparison of AUCs). Conclusion : Alongside statistically significantly faster time to engraftment, shorter hospitalizations and lower infection risk, omidubicel was associated with meaningfully greater preservation or improvement of important HRQL domains compared to UCB."

Clinical • HEOR • P3 data • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Oncology • Transplantation

Omidubicel Versus Standard Myeloablative Umbilical Cord Blood Transplantation: Results of a Phase III Randomized Study. (PubMed, Blood) - "Patients received myeloablative conditioning and graft versus host disease (GvHD) prophylaxis with a calcineurin inhibitor and mycophenolate mofetil. Transplantation with omidubicel results in faster hematopoietic recovery and reduced early transplant-related complications as compared to standard UCBT. The results suggest that omidubicel may be considered as a new standard of care for adult patients eligible for UCBT."

Clinical • Journal • P3 data • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Oncology • Transplantation

Navigating The Dynamic Landscape of Stem Cell Transplantation (Cancer Network) - "Mitchell E. Horwitz, MD, discussed how omidubicel and other advances in allogeneic transplantation have impacted different hematologic oncology populations."

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Immune Reconstitution Following Omidubicel Expanded Cord Blood Results In Accelerated NK Cell and CD4+ T-cell Recovery Compared with Alternative Graft Sources In Severe Aplastic Anemia (SAA) (TCT-ASTCT-CIBMTR 2026) - P1/2, P2 | "Conditioning included horse ATG, cyclophosphamide, fludarabine, and 200 cGy total body irradiation, with tacrolimus and mycophenolate mofetil for GVHD prophylaxis. Conclusions Omidubicel transplantation, characterized by a high CD34⁺ cell dose, is associated with exceptionally rapid NK-cell recovery and accelerated CD4⁺ T-cell reconstitution in SAA patients. This enhanced immune recovery may contribute to sustained engraftment and could reduce the risk of opportunistic infectious complications."

Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • CD4 • NCAM1

Evaluating GVHD Outcomes after Chemotherapy-Only Conditioning in Nicotinamide Expanded Umbilical Cord Blood (omidubicel) Transplantation: A Retrospective cohort study (TCT-ASTCT-CIBMTR 2026) - "All patients received conditioning with thiotepa, busulfan, and fludarabine and prophylaxis with tacrolimus and mycophenolate. Omi HCT with chemo-only conditioning showed comparable outcomes with the pivotal P3 and with the EAP. However, rates of aGVHD appeared lower, and cGVHD was comparable to the lower rates seen in the EAP, compared to the P3, in which 31/62 (50%) of patients in the Omi group received TBI. Our findings support use of a chemo-only MAC prior to HCT with Omi."

Retrospective data • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Transplantation

Real-World Case Series of Omidubicel-Onlv for Allogeneic Transplantation in Patients with Hematologic Malignancies Using a Reduced Toxicity Myeloablative Conditioning Regimen (TCT-ASTCT-CIBMTR 2026) - P3 | "A reduced toxicity myeloablative regimen of total body irradiation (TBI), thiotepa (Thio), fludarabine (Flu), and cyclophosphamide (Cy) aims to support engraftment and antitumor activity while minimizing regimen-related toxicity. Allo-HCT using omidubicel where a reduced toxicity myeloablative conditioning regimen of TBI, Thio, Flu and Cy was used resulted in neutrophil engraftment with no new safety signals. This modified regimen may be considered in patients unable to tolerate a standard myeloablative conditioning when receiving omidubicel. Further evaluation is warranted to explore the long-term outcomes with this conditioning regimen."

Clinical • Real-world • Real-world evidence • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Oncology • Transplantation

Outcomes of myeloablative allogeneic hematopoietic cell transplantation with omidubicel vs alternative donor sources. (PubMed, Blood Neoplasia) - "Omidubicel had a higher incidence of grade 2 to 4 acute graft-versus-host disease (GVHD), but similar rates of grade 3 to 4 acute GVHD and chronic GVHD compared to the CIBMTR cohorts. These results support the role of omidubicel as a viable donor source in the current transplant landscape."

Clinical • Journal • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Oncology • Transplantation

RoslinCT and Ayrmid Ltd. Announce Expansion of Strategic Partnership to Manufacture Omisirge (omidubicel-onlv) for Second FDA-Approved Indication in Severe Aplastic Anemia (SAA) (Businesswire) - "Under the commercial supply agreement, RoslinCT will complete technology transfer and support commercial manufacturing of Omisirge for this additional indication at its state-of-the-art cGMP cell therapy manufacturing facility in Hopkinton, MA."

Commercial • Aplastic Anemia

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

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

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

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

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

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

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