ribrecabtagene autoleucel (DRL-1801) / Pregene Biotech, Dr. Reddy’s, Lakefront Biotherapeutics - LARVOL DELTA

Impact of Gamma-Secretase Inhibition on Outcomes Following BCMA CAR-T Therapy in Multiple Myeloma: A Comparison of Two Phase 1 Trials. (PubMed, Transplant Cell Ther) - "Co-administration of a GSI with BCMA CAR-T therapy was associated with improved survival in BCMA-naïve RRMM patients, particularly those with low baseline tumor BCMA levels. These findings suggest GSI modulation of BCMA surface expression may enhance CAR-T efficacy in select patients and support further prospective investigation."

IO biomarker • Journal • P1 data • Hematological Malignancies • Multiple Myeloma • Oncology

A phase 1 trial of fully human BCMA CAR-T therapy for relapsed/refractory multiple myeloma with 5-year follow-up. (PubMed, Blood) - P1 | "FCARH143, an autologous BCMA-targeted CAR-T therapy which incorporates a fully human BCMA-specific scFv and 4-1BB costimulatory domain, was evaluated in a phase 1 trial (NCT03338972) for relapsed/refractory multiple myeloma (RRMM). FCARH143 demonstrated potent anti-myeloma activity with a 100% response rate and manageable toxicity, independent of disease burden or cytogenetic risk. Further evaluation in high-risk RRMM is warranted."

Journal • P1 data • Hematological Malignancies • Multiple Myeloma • Oncology

BCMA-directed CAR-T cell therapy induces durable drug-free remission in refractory primary immune thrombocytopenia. (PubMed, Blood) - "This first clinical evaluation of BCMA CAR-T cell therapy in four patients with refractory primary ITP demonstrated manageable safety, rapid and sustained platelet recovery, and durable drug-free complete remission lasting up to 15 months, supporting further evaluation in larger studies."

Journal • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

BCMA CAR-T CELL THERAPY INDUCES COMPREHENSIVE IMMUNE RESETTING AND DURABLE REMISSION IN REFRACTORY PRIMARY IMMUNE THROMBOCYTOPENIA (EHA 2026) - P1 | "Background Platelet-specific autoantibodies, particularly anti-GPIb/IX antibodies, drive primary immune thrombocytopenia (ITP) and are linked to lower platelet counts, poor responses to corticosteroids, IVIG, rituximab and TPO-receptor agonists, and higher risk of refractory/relapsed disease. (I) Single-cell pathway analysis showing broad immune resetting, including reduced humoral/inflammatory and platelet-related programs and enhanced cytotoxic T-cell signatures. CR, complete response; PR, partial response; sBCMA, soluble BCMA; dPCR, digital PCR."

CAR T-Cell Therapy • Clinical • Late-breaking abstract • Hematological Disorders • Immune Thrombocytopenic Purpura • Infectious Disease • Thrombocytopenia • Thrombocytopenic Purpura • CD8 • GZMB • GZMK • NKG7

In vivo generation of anti-BCMA CAR-T cells in relapsed or refractory multiple myeloma: a phase 1 study. (PubMed, Nat Med) - P1 | "This phase 1, single-arm, open-label trial evaluated the safety and tolerability of ESO-T01, a nanobody-directed, immune-shielded lentiviral vector encoding a humanized anti-B cell maturation antigen (BCMA) CAR, in adults with relapsed or refractory multiple myeloma...Cytokine release syndrome occurred in four patients (three grade 3 and one grade 2) and was managed with corticosteroids, tocilizumab, or supportive care...These findings provide preliminary evidence on the feasibility and safety of in vivo CAR-T generation using an immune-shielded vector. ClinicalTrials.gov registration: NCT06791681 ."

Journal • P1 data • Preclinical • Hematological Disorders • Hematological Malignancies • Infectious Disease • Multiple Myeloma • Oncology

Anti-BCMA CAR T-cell for IgG4-related disease: is this necessary? - Authors' reply. (PubMed, Lancet Rheumatol) - No abstract available

Journal • Inflammation

BCMA-targeted CAR T cells for multi-refractory primary immune thrombocytopenia: A pilot study (ASH 2025) - P1 | "Here, we report the resultsfrom a single-center, single-arm, open-label pilot study (NCT06519565) evaluating autologous BCMA CART cells (PRG-1801) in patients with primary multi-refractory ITP.. Eligible patients were aged 18 to 75 years, had a ≥6-month history of primary ITP, testedpositive for platelet-specific autoantibodies, and had failed multiple prior first- and second-line therapies,with platelet counts persistently below 30×10⁹/L...Twodays later, patients underwent a 3-day lymphodepleting chemotherapy regimen with fludarabine (25mg/m²) and cyclophosphamide (250 mg/m²) on Days -5 to -3... These results provide preliminary evidence that CAR-T cell therapy is effective in refractoryITP patients driven primarily by B-cell–mediated mechanisms, enabling rapid and sustained clearance ofanti-platelet autoantibodies, prompt platelet recovery, and drug-free remission. Given the heterogeneityof ITP, patients with T cell driven disease may be less..."

CAR T-Cell Therapy • Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Infectious Disease • Inflammation • Thrombocytopenia • Thrombocytopenic Purpura • CD4 • CD8

Safety and Efficacy of PRG-1801 in Recurrent/Refractory Primary Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P1 | N=6 | Recruiting | Sponsor: Union Hospital, Tongji Medical College, Huazhong University of Science and Technology | Trial primary completion date: Aug 2025 ➔ Dec 2026

Trial primary completion date • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Impact of Gamma-Secretase Inhibition on the Multiple Myeloma Immune Microenvironment (ASH 2023) - "In a phase I, first-in-human clinical trial (n=18; relapsed/refractory MM) combining the GSI, crenigacestat, with anti-BCMA CAR T-cell therapy (FCARH143), we recently demonstrated that plasma cell BCMA antibody-binding capacity increased a median of 12-fold among 17/18 (94%) of participants after they received a 5-day GSI "run-in" (25 mg orally administered QOD for 3 doses) [Cowan AJ, et al...Accessibility of CD38, the target of daratumumab, was significantly increased in B cells, and SLAMF7, the target of elotuzumab, was significantly increased in plasma cells... BCMA cleavage from myeloma cells' surface is a putative resistance mechanism to BCMA-targeting immunotherapy. This study assessed the single-cell transcriptome and chromatin accessibility in the bone marrow environment of 16 patients given GSI monotherapy to ultimately enhance the efficacy of subsequent anti-BCMA CAR T-cell therapy. We found that prior BCMA-targeted therapy resulted in reduced..."

IO biomarker • Hematological Malignancies • Multiple Myeloma • Oncology • ERBB4 • HES1 • Neurl1 • NOTCH1 • NOTCH2 • SLAMF7 • TNFRSF17

Gamma Secretase Inhibition May Mitigate the Impact of Low BCMA Target Density in Relapsed/Refractory Multiple Myeloma: Results from a Comparative Analysis of Two Phase 1 Clinical Trials (ASH 2024) - "Pts on FH9952 received the γ-secretase inhibitor (GSI), crenigacestat, during a pre-CAR-T run-in (3 doses 48 h apart) followed by post-infusion dosing 3x weekly for up to 9 doses. Limitations of this study include a small sample size and retrospective analysis. A randomized prospective study is needed to confirm a survival benefit of incorporating a GSI into treatment with FCARH143, or other BCMA CAR-T products."

Clinical • P1 data • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology • Plasma Cell Leukemia

Anti-BCMA CAR-T for the treatment of IgG4-related disease: clinical assessment and single-cell transcriptomic analysis. (PubMed, Lancet Rheumatol) - No abstract available

Journal • Inflammation

BCMA-targeted CAR T cell therapy can effectively induce disease remission in refractory lupus nephritis. (PubMed, Ann Rheum Dis) - "Anti-BCMA CAR T therapy can help LN patients inducing and maintaining disease remission status safely and effectively, which indicated the potential feasibility of the BCMA-only-targeting strategy in treating autoimmune diseases with abnormal humoral immune responses through CAR products."

Journal • Glomerulonephritis • Immunology • Infectious Disease • Inflammation • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Systemic Lupus Erythematosus

Engineering T Cell-Specific Lentiviral Vectors for Safe and Effective In Vivo CAR-T Therapy (ASGCT 2025) - "BCMA CAR-T cells generated using VSV-GI331E TCRαβ VHH ENaBL-T (referred to as ESO-T01) exhibited potent antitumor activity in vitro against target tumor cells. ENaBL-T represents a promising step toward the clinical translation of cost-effective, scalable, and safe in vivo CAR-T cell therapies. Disease Focus of Abstract:Cancer Hematologic"

IO biomarker • Preclinical • Viral vector • Hematological Malignancies • Oncology • CD8

ENaBL, a Novel Lentiviral Vector Platform for In Vivo Engineering of Therapeutic Anti-BCMA CAR T Cells, Shows Effective T Cell Transduction and Tumor Control in a Humanized Murine Model (ASGCT 2025) - P1 | "Chimeric antigen receptor (CAR) T-cell therapies have transformed treatment for B-cell malignancies, though their availability is restricted by manufacturing limitations, logistical demands, long waiting time and high cost of treatment. We have successfully manufactured ENaBL-T encoding a clinically validated BCMA CAR construct (referred to as ESO-T01) at clinical scale using adherent fixed bed bioreactor and first in human testing was initiated in 4Q2024 (NCT06691685). Disease Focus of Abstract:Cancer Hematologic"

CAR T-Cell Therapy • IO biomarker • Preclinical • Viral vector • Hematological Malignancies • Multiple Myeloma • Oncology • CD34 • CD8

Development of Low-Cost CAR-T Process Street with Improved Manufacturing Success (ASGCT 2025) - "A Phase 1 clinical trial, SWASTH, (CTRI/2023/11/059795 [Registered on: 14/11/2023]) of an anti-BCMA CAR-T in patients with r/r MM has been completed (n = 8) and the results show that a single dose of DRL-1801 (2.0–10.0 × 106 CAR+ T cells/Kg) administered by IV infusion in r/r MM patients is effective and safe, with an overall response rate of 100%, indicating that DRL-1801 may provide significant benefit to patients with r/r MM (Upload 01)...Our preliminary data indicate that this robust and cost-effective process can also be implemented across multiple CAR-T products in a "plug and play" mechanism. Disease Focus of Abstract:Myeloma"

IO biomarker • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Gene Therapies • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology

A Study of ESO-T01 in Treating Relapsed/ Refractory Multiple Myeloma (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: Chunrui Li | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Malignancies • Multiple Myeloma • Oncology

A Study of ESO-T01 in Treating Relapsed/ Refractory Multiple Myeloma (clinicaltrials.gov) - P1 | N=24 | Not yet recruiting | Sponsor: Chunrui Li

New P1 trial • Hematological Malignancies • Multiple Myeloma • Oncology

Safety and Efficacy of PRG-1801 in Recurrent/Refractory Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P1 | N=9 | Recruiting | Sponsor: Wuhan Union Hospital, China | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Preclinical delayed toxicity studies of BCMA CAR T-cell injection in B-NDG mice with multiple myeloma. (PubMed, Front Immunol) - "The present study demonstrated a low toxicity of BCMA CAR T-cell injection, with manageable side effects and good anticancer activity and without observable adverse effects. This study provides data to support future clinical studies of BCMA CAR T-cell injection for MM."

CAR T-Cell Therapy • Journal • Preclinical • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation • IFNG • IL10 • IL12A • IL2 • IL4 • IL6 • TNFA

Aurigene Oncology Limited Announces Promising Results of Phase 1 Study for India’s First Trial for Novel Autologous CAR-T Cell Therapy for Multiple Myeloma (Businesswire) - P1/2 | N=08 | SWASTH (CTRI/2023/11/059795) | "The study reported initial results from the first 8 patients. All patients were heavily pre-treated with median of 5.5 previous lines of treatment. Most patients had also received transplant in the past and had disease progression post-transplant. All 8 patients (100%) achieved clinical response, with 5/8 (62.5%) having achieved stringent complete response. With respect to safety, there were no high-grade events of Cytokine Release Syndrome (CRS) or neurotoxicity, in any of the patients....After reviewing the Phase 1 data, the Indian Regulatory Agency i.e., Drugs Controller General of India (DCGI), has given the nod to commence Phase 2 part of the trial. These results of Phase 1 were presented at the 21st annual meeting of the International Myeloma Society at Rio De Janeiro, Brazil, held recently."

P1 data • Trial status • Multiple Myeloma

SWASTH: Phase 1/2 Study of Ribrecabtagene Autoleucel, a BCMA–Directed CAR-T Cell Therapy, in Relapsed-Refractory Multiple Myeloma (IMW 2024) - P1 | "Introduction: Introduction: Anti BCMA CAR-T cell therapy has demonstrated benefit in relapsed /refractory Myeloma (RRMM)...Cyclophosphamide and fludarabine were used for lymphodepletion... The preliminary data from SWASTH indicates that a single dose infusion of Ribre-cel has a very manageable safety profile with early responses seen in heavily pretreated pts with RRMM."

CAR T-Cell Therapy • P1/2 data • Dengue Fever • Hematological Disorders • Hematological Malignancies • Infectious Disease • Multiple Myeloma • Neutropenia • Oncology • Thrombocytopenia

Safety and Efficacy of PRG-1801 in Recurrent/Refractory Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P1 | N=9 | Not yet recruiting | Sponsor: Wuhan Union Hospital, China

New P1 trial • Hematological Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Safety and Efficacy of PRG-1801 for Refractory Lupus Nephritis and IgG4-Related Disease (clinicaltrials.gov) - P1 | N=30 | Recruiting | Sponsor: Tongji Hospital

CAR T-Cell Therapy • New P1 trial • Glomerulonephritis • Immunology • Inflammation • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology

Refractory ANCA Associated Vasculitis and Lupus Nephritis Treated With BCMA-targeting CAR-T Cells (clinicaltrials.gov) - P=N/A | N=24 | Recruiting | Sponsor: Lingli Dong

CAR T-Cell Therapy • New trial • ANCA Vasculitis • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Vasculitis

PAPILIO-1: PHASE 1/2, MULTICENTER, OPEN-LABEL STUDY TO EVALUATE FEASIBILITY, SAFETY AND EFFICACY OF POINT-OF-CARE–MANUFACTURED ANTI-BCMA CAR T-CELL THERAPY (GLPG5301) IN RELAPSED/REFRACTORY MULTIPLE MYELOMA (EBMT 2024) - "Exploratory assessments include additional GLPG5301 pharmacokinetics (PK) and pharmacodynamics (PD).After screening, patients will undergo leukapheresis of mononuclear cells for point-of-care manufacturing of GLPG5301, followed by fludarabine/cyclophosphamide lymphodepleting chemotherapy (Figure). In Papilio-1, the feasibility, safety and efficacy of GLPG5301, a point-of-care–manufactured BCMA-directed CAR T-cell therapy, will be evaluated in adult patients with rrMM. GLPG5301 will be administered as a fresh product, with a targeted vein-to-vein time of 7 days. >"

CAR T-Cell Therapy • Clinical • P1/2 data • Hematological Malignancies • Multiple Myeloma • Oncology