Qartemi (varnimcabtagene autoleucel) / August Pi i Sunyer Biomedical Research Institute, Immuneel Therap - LARVOL DELTA

Collaborating Across Borders to Advance Patient Care (ASGCT 2025) - P1/2 | "It evaluates VTX-801 in adult patients with Wilson's Disease (NCT04537377)...The second case focuses on the development of varnimcabtagene autoleucel (Var-Cel) , a CAR-T cell therapy initially created within an academic hospital. Var-Cel is designed to treat certain types of relapsed or refractory B-cell malignancies, such as acute lymphoblastic leukemia and non-Hodgkin lymphoma. This groundbreaking, hospital-originated CAR-T therapy is now reaching patients at a fraction of the traditional cost through a national network, with international expansion already underway."

Clinical • Acute Lymphocytic Leukemia • Genetic Disorders • Hematological Malignancies • Hepatology • Leukemia • Lymphoma • Metabolic Disorders • Movement Disorders • Non-Hodgkin’s Lymphoma • Oncology • ATP7B

VARNIMCABTAGENE AUTOLEUCEL DEMONSTRATES SIGNIFICANT, SUSTAINED IMPROVEMENTS AS A FIRST-IN-INDIA PATIENT-REPORTED QUALITY OF LIFE OUTCOMES IN RELAPSED / REFRACTORY B-CELL MALIGNANCIES (IMAGINE STUDY) (EHA 2025) - "As a first QoL outcomes report from India for CAR-T cell therapy, these findings reveal QoL improvements complementary to the previously published positive survival and clinical response outcomes observed with varnim-cel infusion in the IMAGINE study. Early and sustained meaningful improvements in all dimensions including overall health score were commensurate with improved QoL over time indicating superior clinical and humanistic outcomes."

Clinical • HEOR • B Cell Non-Hodgkin Lymphoma • CNS Disorders • Depression • Hematological Malignancies • Lymphoma • Mood Disorders • Non-Hodgkin’s Lymphoma • Oncology • Pain • Pediatrics • Psychiatry

HARNESSING CMV-VSTS FOR ALLOGENEIC CART84 THERAPY TO TARGET AML (EHA 2025) - "To better preserve CMV specificity in thawed CMV-VSTs, we tested OKT-3 as an activation method prior to transduction, which successfully maintained CMV specificity but resulted in low CAR84 expression.Additionally, we evaluated ARI-0001, an academic CD19-targeting CAR, as a control. Given the limited CAR84 expression in CMV-VSTs, efforts continue to optimize CAR84 expression while maintaining CMV specificity. This study highlights CMV-VSTs as a potential allogeneic CART platform and underscores the need for further refinement of transduction protocols to enhance therapeutic efficacy."

IO biomarker • Acute Myelogenous Leukemia • Cytomegalovirus Infection • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Oncology • CD28 • CD4 • CD8

HIGH CD70 EXPRESSION IN NEWLY DIAGNOSED FOLLICULAR LYMPHOMA IS LINKED TO RELAPSE AND PRESENTS OPPORTUNITIES FOR DUAL CD19-CD70 CAR-T THERAPY (EHA 2025) - "Thus using a co-transduction approach we combined this truncated CD 7 construct with the approved anti-CD19 academic product ARI-0001 (Martinez-Cibrian N Br J Haematol 0 4)... Overall this study uncovers the prognosis potential of CD70 its biological role in FL patients and the generation of a novel dual CD19-CD70 CAR-T with therapeutic potential in FL as well as other B-NHL. Ongoing studies aim to further validate these results and to optimize its clinical application."

IO biomarker • B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Indolent Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • CD40LG • CD70 • CD8

CD19 CAR-T cell therapy in a pediatric patient with MDA5+ dermatomyositis and rapidly progressive interstitial lung disease. (PubMed, Med) - "A pediatric patient with aggressive MDA5+DM-RPILD achieved progressive long-term improvement and immunosuppressant-free remission over 11 months after compassionate use of a CD19 CAR-T cell therapy (ARI-0001)."

Journal • Dermatomyositis • Immunology • Inflammatory Arthritis • Interstitial Lung Disease • Lupus • Myositis • Oncology • Pediatrics • Pulmonary Disease • Respiratory Diseases • Rheumatoid Arthritis • Rheumatology • Solid Tumor • Systemic Lupus Erythematosus • IFIH1

QUALITY OF LIFE, ANXIETY, AND DEPRESSION REPORTED BY PATIENTS DURING THE FIRST 24 MONTHS FOLLOWING CAR-T CELL THERAPY (EBMT 2025) - "Before CAR-T, 69% had received ≥4 lines of treatment, 89% had an ECOG performance status of ≥1, and 24% presented ≥4 comorbidities.Eighty-seven patients were approved for CAR-T therapy (37% within clinical trials); 46% received varnimcabtagene autoleucel and 20% axicabtagene ciloleucel. Long-term follow-up of CAR-T patients shows significant improvements in QoL and emotional well-being, with a marked reduction in anxiety and depression symptoms. Persistent issues such as fatigue and financial impact highlight the need for ongoing psychological and social support. Advanced nurse practitioner play a key role in implementing systematic assessments of QoL and psycho-emotional status using validated tools, enabling early intervention and comprehensive patient care."

CAR T-Cell Therapy • Clinical • HEOR • Acute Lymphocytic Leukemia • Bone Marrow Transplantation • CNS Disorders • Depression • Fatigue • Hematological Disorders • Hematological Malignancies • Insomnia • Leukemia • Mood Disorders • Oncology • Pain • Psychiatry • Sleep Disorder

OUTCOMES OF CAR T-CELL THERAPY (CART19) FOR B-ALL: A GOCART COALITION REPORT ON BEHALF OF THE PDWP, ALWP, AND CTIWP OF THE EBMT (EBMT 2025) - " A total of 345 B-ALL patients (173 adults and 172 paediatrics) received therapy with Kymriah (65%), ARI-0001 (26%), Tecartus (5.5%), and others (3.5%), with a reverse Kaplan-Meier median follow-up of 2.2 years...Notably, this association was lost in alloHCT-exposed patients, with no impact for prior blinatumomab exposure and response. Prior inotuzumab refractoriness was strongly associated with worse 2-year OS and LFS for both alloHCT-naïve and alloHCT-exposed patients (p≤0.003), although inotuzumab-responsive and inotuzumab-naïve patients showed comparable outcomes... Commercial and academic CART19 therapy remains an effective treatment for adult and paediatric B-ALL patients. This "real-world" study suggests that efficacy is more impacted by prior refractoriness and disease burden than by age, preceding therapy, or cytogenetics. Also, the relevance of preceding factors and the biology affecting CART19 therapy might be different for..."

CAR T-Cell Therapy • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Pediatrics

HEMATOPOIETIC STEM CELL TRANSPLANTATION AFTER CAR19 T-CELL THERAPY IN B-ALL: A GOCART COALITION STUDY ON BEHALF OF THE PDWP, ALWP, AND CTIWP OF THE EBMT (EBMT 2025) - " Of 345 patients (173 adults and 172 children) infused with CART19 (Tisagenlecleucel in 65.2%, Varnimcabtagene autoleucel 25.8%, brexucabtagene autoleucel 5.5%, and others 3.5%), 113 subsequently received an HSCT (38 adults and 75 children; median age 14.1 years, IQR 8.3-22.4). To the best of our knowledge this is the largest cohort of transplanted patients after CART19. We found that patients transplanted without disease recurrence had a better LFS, suggesting that strategies to identify patients at higher risk of relapse are needful. HSCT provided a higher benefit in children, with no differences in NRM, although differences in conditioning and previous HSCT might have contributed to these discrepancies."

Bone Marrow Transplantation • Transplantation

HEMATOPOIETIC STEM CELL TRANSPLANTATION AFTER CAR19 T-CELL THERAPY IN B-ALL: A GOCART COALITION STUDY ON BEHALF OF THE PDWP, ALWP, AND CTIWP OF THE EBMT (EBMT 2025) - " Of 345 patients (173 adults and 172 children) infused with CART19 (Tisagenlecleucel in 65.2%, Varnimcabtagene autoleucel 25.8%, brexucabtagene autoleucel 5.5%, and others 3.5%), 113 subsequently received an HSCT (38 adults and 75 children; median age 14.1 years, IQR 8.3-22.4). To the best of our knowledge this is the largest cohort of transplanted patients after CART19. We found that patients transplanted without disease recurrence had a better LFS, suggesting that strategies to identify patients at higher risk of relapse are needful. HSCT provided a higher benefit in children, with no differences in NRM, although differences in conditioning and previous HSCT might have contributed to these discrepancies."

Bone Marrow Transplantation • Transplantation

EXPANDING CAR T-CELL THERAPY BOUNDARIES: DEVELOPING A MIXED OUTPATIENT-AT HOME MODEL (EBMT 2025) - "Eligible patients and their caregivers underwent pre-enrolment education, providing symptom detection tools, including the caregiver-administered ICE score and an adverse event action plan.The protocol involved AH lymphodepletion with fludarabine (30 mg/m²/d i.v.) and cyclophosphamide (300 mg/m²/d i.v.) for three days, followed by fractionated infusions (10%, 30%, and 60%) of academic CD19-directed (varnimcabtagene autoleucel, ARI0001, 1x10⁶ CAR T cells/kg) or BCMA-directed (cesnicabtagene autoleucel, ARI0002h, 3x10⁶ CAR T cells/kg) based on the haematologic malignancy...The median age was 38 years (range 33–71), with 60% female, receiving a median of three prior therapies (range 2-4), including inotuzumab (20%), autologous stem cell transplantation (20%) and allogeneic stem cell transplantation (allo-SCT) (40%).The median follow-up in the home care unit was 21 days (range 12–30)...CRS was managed with a single dose of tocilizumab and dexamethasone in one..."

CAR T-Cell Therapy • Clinical • Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Multiple Myeloma • Oncology

EXPANDING CAR T-CELL THERAPY BOUNDARIES: DEVELOPING A MIXED OUTPATIENT-AT HOME MODEL (EBMT 2025) - "Eligible patients and their caregivers underwent pre-enrolment education, providing symptom detection tools, including the caregiver-administered ICE score and an adverse event action plan.The protocol involved AH lymphodepletion with fludarabine (30 mg/m²/d i.v.) and cyclophosphamide (300 mg/m²/d i.v.) for three days, followed by fractionated infusions (10%, 30%, and 60%) of academic CD19-directed (varnimcabtagene autoleucel, ARI0001, 1x10⁶ CAR T cells/kg) or BCMA-directed (cesnicabtagene autoleucel, ARI0002h, 3x10⁶ CAR T cells/kg) based on the haematologic malignancy...The median age was 38 years (range 33–71), with 60% female, receiving a median of three prior therapies (range 2-4), including inotuzumab (20%), autologous stem cell transplantation (20%) and allogeneic stem cell transplantation (allo-SCT) (40%).The median follow-up in the home care unit was 21 days (range 12–30)...CRS was managed with a single dose of tocilizumab and dexamethasone in one..."

CAR T-Cell Therapy • Clinical • Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Multiple Myeloma • Oncology

Endothelial Activation as a Predictor of Mortality in CD19-Directed CAR-T Therapy (AAN 2025) - "Independent predictors of ICANS included female sex [HR 1.85, 95%CI 1.1-2.9], treatment with Axicabtagene Cyloleucel (vs ARI-0001) [HR 16.47, 95% CI 6.1-44.4], and prior CRS [HR 44.6, 95%CI 5.8-340.7]. EASIX-preLD was a more reliable predictor of mortality than CRS or ICANS. Its incorporation into clinical practice may improve risk stratification and patient management."

CNS Disorders • Follicular Lymphoma • Hematological Disorders • Hematological Malignancies • Lymphoma • Movement Disorders • Oncology

CART19-BE-02: A MULTICENTRE TRIAL OF VARNIMCABTAGENE AUTOLEUCEL FOR THE TREATMENT OF ADULT PATIENTS WITH RELAPSED/REFRACTORY B-CELL ACUTE LYMPHOBLASTIC LEUKAEMIA (EBMT 2025) - "This abstract is under embargo until 17 March 2025."

Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology

CART19-BE-02: A MULTICENTRE TRIAL OF VARNIMCABTAGENE AUTOLEUCEL FOR THE TREATMENT OF ADULT PATIENTS WITH RELAPSED/REFRACTORY B-CELL ACUTE LYMPHOBLASTIC LEUKAEMIA (EBMT 2025) - "This abstract is under embargo until 17 March 2025."

Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology

A MULTICENTER TRIAL OF VARNIMCABTAGENE AUTOLEUCEL (VAR-CEL) FOR ADULT PATIENTS WITH RELAPSED/REFRACTORY B-CELL ACUTE LYMPHOBLASTIC LEUKEMIA (CART19-BE-02 TRIAL) (EHA-EBMT-CART 2025) - No abstract available

Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology

India’s first global CAR T-cell therapy for Non-Hodgkin’s Lymphoma launched (New Indian Express) - "India’s first global CAR T-cell therapy for adult B-cell Non-Hodgkin Lymphoma (B-NHL), a kind of blood cancer that affects the lymphatic system, has been launched in the country, offering new hope to patients facing aggressive blood cancers...'The therapy is priced between ₹35 lakhs to ₹50 lakhs. While this price reflects the complexity of manufacturing personalized CAR T-cell therapy, it is still significantly more affordable compared to similar global options, ensuring access for Indian patients without compromising on quality....Immuneel has partnered with leading hospitals, including Narayana Health, Apollo Hospitals, CMC Vellore & Ludhiana, Manipal Hospitals, RGCIRC Delhi, SGPGI Lucknow, Amrita Hospital Faridabad, HOC Vedanta Ahmedabad, Cytecare Bangalore, Sparsh Bangalore, Marengo Asia Hospitals among others, to provide Qartemi to their patients."

Launch non-US • Non-Hodgkin’s Lymphoma

Long-Term Safety and Efficacy Outcomes of Varnimcabtagene Autoleucel in Relapsed Refractory B Cell Malignancies: India Phase 2 Imagine Study (TCT-ASTCT-CIBMTR 2025) - "Tocilizumab, steroids and anakinra usage was in 37.5% (n=9/24, majority for persistent G1 CRS), 8% and 4% respectively. Varnimcabtagene autoleucel offers a favourable safety profile achieving long-term efficacy without severe neurotoxicity enhancing patient outcomes."

Clinical • P2 data • Anemia • Hematological Disorders • Hematological Malignancies • Neutropenia • Oncology • Thrombocytopenia

Role of procalcitonin, C reactive protein and ferritin in cytokine release syndrome after CAR T-cell therapy in children and young adults. (PubMed, Biomarkers) - "Our goal is to elucidate the role of procalcitonin (PCT), C-reactive protein (CRP) and ferritin in the context of CRS following CAR T-cell infusion to predict its severity and PICU admission.MethodsProspective observational study (2016-2022) in children and young adult who received CAR T-cell therapy (Tisagenlecleucel/ARI-0001). Biomarkers were higher in those who needed inotropic or respiratory support.ConclusionsPCT levels increase after CAR-T cell therapy in the setting of systemic inflammation and could be a predictor of PICU admission and evolution to death. Further research studying its role in the context of CRS and the differential diagnosis between infection and CRS is needed to better understand the biology of this biomarker and to define its value in clinical practice."

IO biomarker • Journal • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Critical care • Hematological Malignancies • Infectious Disease • Inflammation • Leukemia • Oncology • Pediatrics • CRP

Real-World Outcomes of CD19-Targeted CAR T-Cell Therapy in Adult and Pediatric Patients with B-Cell Acute Lymphoblastic Leukemia (B-ALL): Insights from the GoCART Coalition on Behalf of the PDWP, ALWP, and CTIWP of the EBMT (ASH 2024) - "Results : A total of 345 patients (173 adults and 172 pediatrics) received therapy from 2016 to 2023 with tisagenlecleucel (65.2%), varnimcabtagene autoleucel (25.8%), brexucabtagene autoleucel (5.5%), and others (3.5%)...Bridging therapy was administered in 86.4% (298) of patients based on high or low intensity chemotherapy (32.1% or 26.5%, respectively), inotuzumab-ozogamicin (13.4%), TKI (8.2%), involved-site therapy (intrathecal chemotherapy, radiotherapy, orchiectomy) (3.5%), and blinatumomab (2.6%), alone or in combination...Lymphodepletion was performed in all but one patient, based on fludarabine and cyclophosphamide regimens...These findings support the continued use and further investigation of CAR T-cell therapy in diverse clinical settings, emphasizing the importance of post-infusion strategies to sustain long-term remission. This will provide deeper insights into the factors influencing outcomes and help refine treatment protocols to improve patient care."

CAR T-Cell Therapy • Clinical • Real-world • Real-world evidence • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Pediatrics

CD70 Deregulation in Follicular Lymphoma at Diagnosis Is Associated with Relapse and Opens New Avenues for Dual CD19-CD70 CAR-T Therapy (ASH 2024) - "Here we assessed the immune profile (Nanostring® nCounter) in formalin-fixed paraffin-embedded (FFPE)-derived biopsies in a series of FL patients at diagnosis, homogenously treated with immunochemotherapy (mostly R-CHOP), and followed at Hospital Clínic de Barcelona...Finally, to advance towards personalized therapies for these high-risk patients and taking advantage of the CAR-T program at our institution, we generated a dual CD19-CD70 CAR-T combining an approved academic product, ARI-0001 (Martinez-Cibrian N, Br J Haematol 2024), and a truncated CD27 protein...Ongoing work includes the optimization of the CRISPR protocol during the CAR-T generation and in vivo studies. In summary, this multipronged study has allowed us to uncover the prognosis potential of CD70, its biological role in FL patients and the generation of a novel dual CD19-CD70 CAR-T with therapeutic potential in FL as well as other B-NHL."

IO biomarker • B Cell Lymphoma • B Cell Non-Hodgkin Lymphoma • Follicular Lymphoma • Hematological Malignancies • Indolent Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • CARD11 • CD27 • CD40LG • CD70 • CD8 • IL21 • IL4 • TNFRSF14

Second Infusion of CAR T-Cells in Patients with Relapsed /Refractory B-Cell Acute Lymphoblastic Leukemia: Results from a Gocart Coalition Analysis on Behalf of the PDWP, ALWP, and Ctiwp of the EBMT (ASH 2024) - "CART1 products were varnimcabtagene autoleucel (ARI-0001) (23), tisagenlecleucel (13), brexucabtagene autoleucel (1), and other experimental (2). These results show that CART2 is associated with low NRM and might be a bridge to SCT or other additional therapies. Updated results with data about subsequent therapies after CART2 will be presented at ASH meeting."

CAR T-Cell Therapy • Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • Pediatrics • CD22

"Real World" Outcome of Hematopoietic Stem Cell Transplantation after CAR19 T Cell Therapy in Children and Adults with B-ALL: A Gocart Coalition Study on Behalf of the PDWP, ALWP, and Ctiwp of the EBMT (ASH 2024) - "Results : Of 345 patients (173 adults and 172 children) infused with CART19 (Tisagenlecleucel in 65.2%, Varnimcabtagene autoleucel 25.8%, brexucabtagene autoleucel 5.5%, and others 3.5%), 113 subsequently received an HSCT (37 adults and 76 children; median age 13.5 years, IQR 8.2-22.2) at a median of 6.2 months (3.7-11.8) after CART19 infusion. The low NRM, with no differences between pre-transplanted and transplant naive patients, suggest that HSCT after CART19 is well tolerated, and represent a valid strategy to reduce the risk of relapse. As it remains unclear for which patients autologous CART19 cells remains a stand-alone treatment, future studies should focus on strategies for early identification of patients at higher risk of treatment failure."

Clinical • Real-world • Real-world evidence • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Transplantation

Preclinical development of three novel CARs targeting CD79b for the treatment of non-Hodgkin's lymphoma and characterization of the loss of the target antigen. (PubMed, J Immunother Cancer) - "Based on specificity, efficacy, and loss of the target antigen, CARLY3 represents a potential novel CAR treatment for NHL."

IO biomarker • Journal • Preclinical • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma • CD20 • CD22 • CD79B

Patient-Derived Follicular Lymphoma Tumoroids: A Tool to Recapitulate Biological Features of Low and High-Grade Lymphoma (ASH 2024) - "The anti-CD19 CAR-T product ARI-0001 was able to adhere to the tumoroid's surface but shows moderate infiltration and e`icacy, compared to CART activity in Patient- Derived Lymphoma Spheroids lacking ECM. This indicates that ECM may be a crucial player in cell therapy evasion in lymphoma by preventing CAR-T cells from reaching their target cells, which is a common hindrance in solid tumors. In summary, we have developed a patient-derived model that can recapitulate both indolent and aggressive FL features, providing an in vitro model suitable to study disease biology and personalized therapy testing in the context of structural and cellular elements of the TME, while reducing animal usage."

Clinical • IO biomarker • Follicular Lymphoma • Hematological Malignancies • Indolent Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor • CD40LG • IL21 • VIM

The CAR-Hematotox Identifies Patients at High Risk of Infectious/Inflammatory Complications in a Cohort of Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia (BCP-ALL) Treated with ARI-0001 (Varnimcabtagene Autoleucel) (ASH 2024) - "Finally, we did not find differences between high and low HT either in the expansion peak (median 6.66 cells/µl vs 3.53 cells/µl, p=0.3) or AUC at 30d (median 81.82 μl/l vs 61.59 μl/l, p=0.2). Conclusion : The CAR-HEMATOTOX score at lymphodepletion predicts inflammatory complications and transfusion dependence by identification of a high-risk group in our R/R BCP-ALL cohort, that can help us design individual prophylaxis and support strategies, as well as select patients for outpatient infusion and follow-up."

Clinical • Acute Lymphocytic Leukemia • B Cell Lymphoma • B Cell Non-Hodgkin Lymphoma • Bone Marrow Transplantation • Follicular Lymphoma • Hematological Disorders • Hematological Malignancies • Infectious Disease • Large B Cell Lymphoma • Leukemia • Lymphoma • Mantle Cell Lymphoma • Non-Hodgkin’s Lymphoma • Oncology