plerixafor / Generic mfg. - LARVOL DELTA

Diluting high yield hematopoietic stem cell apheresis for autologous transplantation results in high viability of fresh grafts beyond 72h (ASH 2025) - "At the Instituto de Prevision Social all lymphoma patients are transplanted with fresh grafts using a shortened BEAM protocol with time to collect to infusion of 96 hours (BCNU d-4; Ara-C d-4, d-3, d-2; Etoposide d-4, d-3, d-2; Melphalan d-2) and minimum CD34+ cells of 2,5 x 10*6...All patients were mobilized with GCSF +/- Plerixafor... While this is a very limited study due to number of participants, its retrospective nature and single center experience the data suggests diluting high yield apheresis products result in high viability of HPSC beyond 72 hours. Any HCT Program should aim to have cryopreservation facilities available as there are some patients that cannot be transplanted safely without said capacities. However, this may not be the case for Lymphoma patients."

Hematological Malignancies • Immunology • Lymphoma • Multiple Myeloma • Solid Tumor • Transplantation • CD34

Single-cell RNA-seq uncovers that plerixafor can mobilize more monocyte progenitor cells and enhance the activating graft immune status in multiple myeloma autologous transplantation. (ASH 2025) - "This finding suggests that Plerixafor mobilization not only significantly increases the proportion of CD34+ cells and monocytic progenitor cells but also alters the overall immune status of the graft, shifting it towards an immune-activated state, providing a new perspective for optimizing mobilization strategies in MM autologous transplantation and may have significant implications for immune reconstitution and transplantation outcomes."

Bone Marrow Transplantation • Hematological Malignancies • Multiple Myeloma • Transplantation • C1QA • C1QB • CD34 • CD68

Enhancing the prediction of CD34+ cells yield in autologous stem cell collection using complete blood count and clinical information: A quality improvement initiative spanning 4.5 years of data from a tertiary academic center (ASH 2025) - "It involves mobilizing CD34-positive (CD34+) peripheral blood stem cells (PBSCs) using granulocyte colony-stimulating factor (G-CSF) and plerixafor, known as the G+P regimen... The predictive model using information available pre-collection (clinical and CBC) provided a degree of accuracy for predicting a successful collection; however, this model was less accurate than models that incorporate pre-CD34. Future analyses are needed to improve prediction with only pre-initiation data. 1."

Clinical • Immunology • Lymphoma • Solid Tumor • CD34

Dara-RVD in newly diagnosed multiple myeloma: Real-world clinical practice (ASH 2025) - "Depending on post-ASCT MRD status, patients received lenalidomide maintenance therapy, if MRD negativity was achieved...Antitumor treatment was administered alongside supportive therapy (sulfamethoxazole-trimethoprim, acyclovir), immunoglobulin infusions, osteomodifying agents...Seven patients underwent stem cell harvesting (etoposide 750 mg/m 2 )combined with short-acting filgrastim without routine plerixafor administration...Conclusion : In the context of limited access to CAR T-cell and bispecific therapies in Russia, early use of Dara-RVd with MRD-adaptive strategies shows high efficacy, including high-risk NDMM patients. This quadruplet-based approach may shift the treatment paradigm, though careful monitoring for complications like hypogammaglobulinemia is essential."

Clinical • Real-world • Real-world evidence • Hematological Malignancies • Infectious Disease • Multiple Myeloma • Musculoskeletal Pain • Pneumococcal Infections • Renal Disease • Respiratory Diseases • CD34 • CTCs

autologous hematopoietic cell transplantation (AHCT) in the era of novel therapies for multiple myeloma– real-world data from a single cente (ASH 2025) - "This study evaluates the long-term efficacy and outcomes of AHCT in a real-world cohort of MM patients receiving either daratumumab-based quadruplets or bortezomib-based triplets as induction therapy...Disease status at the time of transplantation showed complete response (CR) in 22 patients (49%) in Group A and 10 patients (28%) in Group B. Stem cell mobilization yielded a median of 3.42×10⁶ CD34⁺ cells/kg (range: 2.00–9.5) vs 4.11×10⁶ cells/kg (range: 2.00–9.3) and plerixafor was required in 59% vs 46% in Groups A and B and respectively .Median time to neutrophil and platelet engraftment was similar to both treatment groups [11 days in both groups for neutrophil (range: 9–13 and 7–13), and 13 days (range: 10–26) vs 11 days (range: 8–23) for platelet in Groups A and B...Conclusion Autologous stem cell transplantation remains an effective treatment option for multiple myeloma, even in the era of novel induction regimens. Daratumumab-based quadruplets do not..."

Clinical • Real-world • Real-world evidence • Hematological Malignancies • Multiple Myeloma • Plasmacytoma • Transplantation • CD34

Peripheral blood stem cell collection for patients with multiple myeloma undergoing autologous transplant after quadruplet induction (ASH 2025) - "Background: In recent years, quadruplet induction regimens, including daratumumab, bortezomib, lenalidomide and dexamethasone (D-VRD) and daratumumab, carfilzomib, lenalidomide and dexamethasone (D-KRD) are increasingly used for patients with newly diagnosed multiple myeloma (MM)...The majority of patients in both CD34 groups (n=123, 88%) received GCSF+plerixafor for stem cell mobilization. Six patients received chemo-mobilization with cyclophosphamide, all in the high CD34 group... In our cohort, approximately one in six patients who received quadruplet induction had suboptimal PBSC collection prior to autoHCT, which was more commonly seen in older patients. The number of induction cycles and lenalidomide dose during induction did not significantly impact the yield of stem cell collection. A lower CD34 collection did not adversely impact the engraftment, response rates or survival outcomes."

Clinical • Hematological Malignancies • Multiple Myeloma • Transplantation • CD34

Impact of pomalidomide-containing induction chemotherapy on hematopoietic stem cell mobilization in multiple myeloma: A retrospective cohort study (ASH 2025) - "Preclinical data suggest POM upregulates CXCR4 on hematopoietic stem cells (HSCs), potentially impairing mobilization, similar to lenalidomide (LEN)...Mobilization used G-CSF ± plerixafor ± high-dose cyclophosphamide (HD-CTX)...Daratumumab exposure significantly impairs mobilization efficiency. Older age, prolonged induction, and multi-agent regimens negatively impact stem cell harvest. These findings support routine use of HD-CTX or plerixafor for POM-exposed patients and highlight the need for risk-adapted mobilization strategies in TE-NDMM."

Retrospective data • Hematological Malignancies • Multiple Myeloma • CD34 • CXCR4

Real-world stratified treatment strategies in transplant-eligible newly diagnosed multiple myeloma: A multicohort study (ASH 2025) - "Methods Real-world cohorts from First Affiliated Hospital of Sun Yat-sen University (2024–2025): - UHiR cohort (n=26): Dara-KAD (daratumumab, carfilzomib, liposomal doxorubicin, dexamethasone) induction → tandem ASCT → carfilzomib/pomalidomide maintenance. Mobilization remained feasible across all cohorts with proactive plerixafor use. Real-world evidence supports risk-adapted induction, transplantation, and mobilization strategies."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Hematological Malignancies • Infectious Disease • Multiple Myeloma • Neutropenia • Transplantation • CD34

High transplant conversion rates with gemcitabine, dexamethasone and carboplatin (GDP)-based therapy in Relapsed/Refractory lymphoma: A real-world experience (ASH 2025) - "G-CSF and Plerixafor-mobilized peripheral blood stem cell were used in all patients...Eligible patients received agents like Rituximab, Brentuximab, and Nivolumab with additional cost per cycle...The regimen's daycare feasibility and reduced hospitalization needs offer significant quality-of-life and economic advantages. Substituting Carboplatin for Cisplatin further enhances administration convenience."

Clinical • Real-world • Real-world evidence • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Lymphoma • Neutropenia • Non-Hodgkin’s Lymphoma • Thrombocytopenia • Transplantation

Integrative single-cell multi-omic and functional profiling of human bone marrow regulatory T cells to enhance GVHD-preventive cell therapies (ASH 2025) - "Using CyTOF, we analyzed Tregs from donorperipheral blood collected under two mobilization regimens: granulocyte colony-stimulating factor (G-CSF) alone, and G-CSF combined with plerixafor (n = 8)...Suppression assaysusing conventional T cells and CRISPR-edited Tregs showed that marker deletion also affected Tregsuppressive function.Together, our work identifies distinct transcriptional, epigenetic, and phenotypic features of human BMTregs and demonstrates that specific markers contribute to their suppressive function. These findingsprovide mechanistic insight into Treg biology in the context of allo-HSCT and offer strategies to enhanceTreg-based cell therapies for GVHD prevention."

Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • CXCR4 • FOXP3

Romiplostim N01 enhances platelet engraftment in ASCT using non-cryopreserved pbscs for plasma cell neoplasms: A single-center phase II study in China (ASH 2025) - "Other baseline characteristics were also comparable, such as Durie-Salmon stage, R-ISS stage, HCT-CI scpre, disease response prior to HSCT, Plerixafor use, single dayapheresis and Melphalan dose.All patients achieved hematologic engraftment. At a median follow-up of 10.8 months, 2-year overall survival did not differsignificantly (85.7% ± 13.2% vs 84.4% ± 8.3%, P=0.717).In patients with plasma cell neoplasms undergoing ASCT with non-cryopreserved PBSCs, RomiplostimN01 significantly accelerated platelet engraftment and reduced hospitalization costs withoutcompromising safety. These findings support its use as an effective alternative to traditionalcryopreserved PBSCs with rhTPO support."

Clinical • P2 data • Amyloidosis • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Mucositis • Multiple Myeloma • Neutropenia • Plasmacytoma

Targeting the SDF-1/CXCR4 axis to attenuate pain in sickle cell disease: A preclinical evaluation of AMD3100 as a non-opioid therapeutic strategy (ASH 2025) - "These findings back the potential of CXCR4 antagonism as a non-opioid option for managing SCDpain. They also emphasize the need to focus on vascular-neuro-immune interactions in precise medicinemethods."

Preclinical • Genetic Disorders • Hematological Disorders • Immunology • Pain • Sickle Cell Disease • CXCL12 • IL6 • TNFA

Apheresis blood processing with heparin improves stem cell collection outcomes for sickle cell gene therapy (ASH 2025) - "Patients were mobilizedwith single-agent plerixafor, and the target procedure duration was ≥4 total blood volumes (TBV), up to amaximum of 8 hours...Further investigation into the underlying mechanisms, includingreal-time sampling of the buffy coat and cellular activation markers, may clarify the frequently observedinterface instability and guide future protocol optimization. Our strategy has the potential to reduce thenumber of HPC collections needed and improve the likelihood of achieving target cell doses for GTmanufacture."

Gene therapy • Endocrine Disorders • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CD34

Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a (ASH 2025) - P1, P2 | "Achieving an adequate dose of genetically modified hematopoietic stem cells (HSCs) for gene therapy inpatients with sickle cell disease (SCD) remains a challenge due to limitations related to stem cellmobilization using plerixafor alone, reduced apheresis collection efficiency, and losses during ex vivo cellmanipulation...The time interval from first collection cycle tocompletion of product testing was a median of 39 days (mean 56) for all patients, and a median of 37days (mean 38) for those collected in 1 cycle.Preparative transfusions were given before collections to bridge patients after stopping hydroxyurea orto mitigate stress erythropoiesis...The success of this study was driven byoptimizing apheresis strategies, including suppression of stress erythropoiesis through transfusion, real-time instrument adjustments, and efficient manufacturing that allowed a collection target of nearly halfthe CD34+ cells recommended in the FDA-approved gene product...."

Clinical • Gene therapy • Viral vector • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CD34

Eltrombopag plus G-CSF for peripheral blood stem cell mobilization: Final analysis of a single arm, Phase 2 trial (ASH 2025) - "However, 9% of patients in the plerixafor era still could not harvestadequate peripheral blood stem cells (PBSC) for auto-HSCT1. Interestingly, ahigher peak eltrombopag serum concentration on ESHAP D8 correlated with a higher quantity ofharvested CD34+ cells (Spearman's rank correlation, rho = 0.41, p = 0.04119), which is compatible with theproposed mechanism of action of this intervention.ConclusionsAdding eltrombopag to stem cell mobilization for autologous PBSC harvest is safe and promising inimproving PBSC harvest efficiency. The efficacy and long-term safety warrant further exploration withconfirmatory studies."

P2 data • Bone Marrow Transplantation • Cardiovascular • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Lymphoma • Venous Thromboembolism • CD34

Real-world experience with apheresis for gene therapy in transfusion-dependent β-thalassemia: The largest single-center report (ASH 2025) - "The edited CD34⁺hematopoietic stem cells are then reinfused into the patient following a four-day regimen ofmyeloablative busulfan conditioning chemotherapy...Seven patients underwent hematopoieticstem cell mobilization with plerixafor and granulocyte colony stimulating factor (G-CSF) followed byleukapheresis over a three-day period...Adverse events observed were consistent with expected toxicities from myeloablativeconditioning. To our knowledge, this is the largest single-centre series of its kind reported to date."

Clinical • Gene therapy • Real-world • Real-world evidence • Beta-Thalassemia • Gene Therapies • Genetic Disorders • Hematological Disorders • BCL11A

Etoposide + cytarabine + pegfilgrastim vs cyclophosphamide + granulocyte colony-stimulating factor for stem-cell mobilization in patients with multiple myeloma: A phase III trial (ASH 2025) - P3 | "Theirprevious exposure to lenalidomide (either4 cycles or ≤ 4 cycles) determined this allocation.Intravenousetoposide at 75 mg/m² daily, intravenous cytarabine at 200 mg/m² every 12 hours, and subcutaneouspegfilgrastim at 6 mg on day 6 were the therapies administered to patients in the EAP group on days 1and 2. In comparison to CG, EAP showed better mobilization efficiency, with a greater percentage ofpatients reaching optimal/target HSC collection thresholds in fewer apheresis sessions. There were nounanticipated side effects, and the regimen demonstrated good safety and acceptability. EAP's lowplerixafor requirement emphasizes self-sufficiency and establishes it as a good first-line treatment forMM patients, especially in situations with limited resources where access to plerixafor is restricted orprohibitively expensive."

P3 data • Hematological Disorders • Hematological Malignancies • Infectious Disease • Multiple Myeloma • Neutropenia • Thrombocytopenia • CD34

Motixafortide (CXCR4 inhibition) alone and in combination with natalizumab (VLA-4 inhibition) to mobilize hematopoietic stem cells for gene therapy in sickle cell disease: A first-in-human, safety and feasibility study (ASH 2025) - P1 | "G-CSF is unsafe inSCD and the CXCR4 inhibitor (CXCR4i) plerixafor (P) does not reliably yield optimal HSC numbers. Remarkably, HSC transplant of non-SCD marrow into SCD mice (myeloablative cKit-ADCconditioning) reverted the enhanced mobilization phenotype in SCD mice to that of non-SCD mice,indicating enhanced CXCR4i-based mobilization in SCD may track with the hematopoietic system.In conclusion, our first-in-human trial demonstrates the potential of M and N+M as novel G-CSF-freeregimens to safely optimize HSC mobilization in SCD (median CD34+ cells/μl: P=73, M=189, N+M=312).Correlative FC and scRNA seq highlight regimen-specific mobilization of unique HSC subsets, includingincreased CLPs, ERPs and MEPs with N+M. Further mechanistic study of HSC mobilization biology maybuild upon our finding that Townes SCD mice and SCD humans share an enhanced mobilizationphenotype with CXCR4i +/- VLA4i."

Clinical • Combination therapy • First-in-human • Gene therapy • P1 data • Dermatology • Gene Therapies • Genetic Disorders • Hematological Disorders • Immunology • Pruritus • Sickle Cell Disease • Urticaria • CD34 • CXCR4

Spatial communication modules identify prognostic cell-cell signaling hubs in chronic graft-versus-host disease (ASH 2025) - "In multivariate analysis, MCIMs-0 and -6 were independently associated withincreased mortality (HR=2.08 and 2.83), while MCIM-3 showed a protective trend (HR=0.51).SpatialDrug2Cell, applied to MCIM-localized cell populations and spatially restricted therapeutic targets,highlighting candidate agents such as rituximab, atezolizumab, and plerixafor. Our analysis establishes spatially resolved MCIMs as prognostic biomarkers in chronicGVHD. Our analysis establishes spatially resolved MCIMs as prognostic biomarkers in chronicGVHD. The emergence of high-risk communication modules in MSGs, particularly in patients with fataloutcomes, reveals tissue-localized immune architectures associated with disease progression. Byintegrating STARComm with adapted drug inference tools like SpatialDrug2Cell, we identify clinicallyrelevant, spatially anchored therapeutic targets in routinely sampled tissues."

Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • CXCL12 • CXCR4 • SDC4

Peptide-based antagonists for targeting CXCR4 in ibrutinib resistant chronic lymphocytic leukemia (ASH 2025) - "Out of 6 tested peptides, the fatty acid-coupled JM#143, JM#194 and JM#198 showed bindingto CXCR4 with IC50s of 14, 16 and 27nM compared to 575nM for plerixafor in JEKO1 cells. Targeting CXCR4 using JM#198 stronglysuppressed transmigration of tumor cells towards CXCL12 without adversely affecting T cellimmunomodulation by ibrutinib. Combined targeting of CXCR4 and BTK could effectively block tissuehoming of CLL cells, improving BTKi efficacy and potentially overcoming resistance."

Chronic Lymphocytic Leukemia • Hematological Malignancies • Immunology • Leukemia • Lymphoma • CD5 • CD8 • CXCL12 • FOXP3 • IL2RA • IL7R

An open-label, multi-center Phase 2 study to assess the safety and efficacy of burixafor (GPC-100) and propranolol with G-CSF for the mobilization of hematopoietic progenitor cells in patients with multiple myeloma (ASH 2025) - P2 | "Mediantimes to neutrophil and platelet engraftment were 11 days (range 11-17 days) and 15 days (range 11-27days), respectively.ConclusionsBurixafor, in combination with propranolol and G-CSF, demonstrated an excellent safety profile andeffectively mobilized sufficient HPCs for AHCT, including patients previously treated with lenalidomideand daratumumab. Notably, burixafor enabled same-day administration with leukapheresis, offering akey advantage over other CXCR4 inhibitors, such as plerixafor and motixafortide through its rapidmobilization kinetics. Its favorable safety profile, characterized by minimal adverse events, supporting itspotential clinical utility."

Clinical • IO biomarker • P2 data • Cardiovascular • Constipation • Diabetes • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Hypotension • Multiple Myeloma • Musculoskeletal Diseases • Musculoskeletal Pain • CD34 • CXCR4

Enhanced CD34+ cell mobilizations, collections, and comparable safety profile with fixed-dose versus weight-based plerixafor dosing in patients with sickle cell disease receiving autologous CD34+ base-edited hematopoietic stem cells (BEAM-101) in the ongoing BEACON study (ASH 2025) - P1/2 | "A highernumber of sickle cell crises was observed during the mobilization period in WB (3 AEs in 2 pts) vs FD (1 AEin 1 pt).ConclusionIn the BEACON study, pts receiving plerixafor using an FD regimen required fewer days and cycles ofapheresis than pts on the traditional WB dose regimen. More CD34+ cells were apheresed in pts receivingFD due to the greater number of CD34+ cells mobilized in the peripheral blood, particularly on Day 1.Pre-dose CD34+ levels were similar, with a generally comparable AE profile between WB and FD groups."

Clinical • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CD34 • CXCR4

Efficient prime editing of human hematopoietic stem cells corrects the recurrent SBDS C.258+2T>C mutation and recovers engraftment potential in shwachman-diamond syndrome (ASH 2025) - "After mobilization with G-CSF andplerixafor, 28 CD34+ HSPCs per mcL were mobilized and 3 blood volumes were processed by apheresisyielding 2.3x106 CD34+ HSPCs per kg...In contrast, after PE treatment, theTP53 mutation frequency decreased from 19% in input HSPCs to 5% in engrafting cells. Colony-formingunit (CFU) analysis from engrafting HSPCs showed that 3 of 15 colonies lacking SBDS editing had a TP53mutation (20%), while only 1 of 87 colonies with SBDS mutation correction showed a TP53 mutation(1.1%), suggesting that SBDS mutation correction counter-selects for engraftment of TP53 mutant HSPCs.Together, these results demonstrate a near-universal and efficient PE approach with high product puritythat corrects the recurrent SBDS c.258+2T>C mutation and restores hematopoietic engraftment functionin SDS patient HSPCs."

Acute Myelogenous Leukemia • Aplastic Anemia • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • CD34 • TP53

First in human prime edited autologous hematopoietic stem cell therapy for the treatment of p47phox CGD: Initial results of PRIME-0101 (ASH 2025) - "Two study participants, Participant 1 (18yo male) and Participant 2 (57yo male) underwent HSCmobilization with G-CSF and plerixafor, and the apheresis product was transferred to a centralmanufacturing facility to generate PM359. Importantly, frequency of both DHR+ neutrophils and PrimeEdited CD34+ cells exceed the 20% threshold expected to be sufficient for restoration of NADPH oxidaseanti-pathogen activity and amelioration of disease pathology. Safety was consistent with busulfanconditioning; neither participant required platelet or red blood cell transfusion support.The initial results from the Prime-0101 study provide the first-in-human demonstration of the safety andefficacy of Prime Editing and offer an autologous cell therapy for individuals with p47phox CGD."

First-in-human • P1 data • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Inflammation • Primary Immunodeficiency • CD34

CRISPR-Cas12a gene editing of the HBG1/2 promoters leads to sustained normalization of total hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease: Updated Results from the RUBY trial (ASH 2025) - P1/2 | "After plerixafor mobilization, autologous CD34+hematopoietic stem and progenitor cells were collected from each patient by apheresis...The safety profile ofreni-cel was consistent with busulfan. Data from the RUBY trial continue to demonstrate early and sustained normalization oftotal Hb and durable increases in HbF following reni-cel infusion, with 31 of 32 patients VOE-free at thetime of this data cut. These updated findings, derived from a larger patient cohort and extended follow-up period, validate this novel gene-editing approach as a potential one-time treatment for patients withsevere SCD."

Clinical • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CD34 • HBG1 • HP