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

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 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

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

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 • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • 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

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

[VIRTUAL] The clinical study of anti-BCMA CAR-T with single-domain antibody as antigen binding domain. (ASCO 2021) - P1 | "The patients had received multiple lines of prior treatment (including bortezomib, lenalidomide, and others) . Following a lymphodepleting regimen of cyclophosphamide (300-600 mg/m2, d-5, -4) and fludarabine (25-30 mg/m2, d-5 to d-3), patients were infused with 2.5-10.0 × 106 CAR+ cells/kg body weight... Our result demonstrates that the CART employing one humanized sdAb targeting BCMA is safe and efficacious for clinical application . The phase I clinical trial has been initiated in China for searching the RP2D using the cryopreserved CAR-T cells."

Clinical • Anemia • Hematological Disorders • Hematological Malignancies • Leukopenia • Multiple Myeloma • Neutropenia • Oncology • Thrombocytopenia

Pregene Enters into Innovative Partnership with CellPoint to Develop anti-BCMA CAR-T Cell Therapy in Europe and the US (PRNewswire) - "Shenzhen Pregene Biopharma...and CellPoint...announce an exclusive license agreement for the development and commercialization of PRG-1801, Pregene's single domain antibody-based anti-BCMA chimeric antigen receptor T cell (CAR-T) program, for the treatment of hematological indications in Europe and the United States....Under the terms of the collaboration, Pregene will receive over €20 million of upfront and near-term consideration, and is eligible to receive additional development, commercial milestone and royalty payments."

Licensing / partnership • Hematological Malignancies • Multiple Myeloma • Oncology

Single VHH-directed BCMA CAR-T cells cause remission of relapsed/refractory multiple myeloma. (PubMed, Leukemia) - No abstract available

CAR T-Cell Therapy • Clinical • Journal • Hematological Malignancies • Multiple Myeloma • Oncology

Pregene hands BCMA CAR-T to Dr. Reddy’s in India for USD 163m (GBI Health) - "China-based Shenzhen Pregene Biopharma Company...struck a licensing deal with India-based Dr. Reddy’s Laboratories Ltd (NYSE:RDY/BSE:500124/NSE:DRREDDY). Pregene will grant Dr. Reddy’s exclusive clinical development and commercialization rights to its nanobody-based chimeric antibody receptor(CAR)-T cell therapy PRG1801 in India, also providing the necessary viral vectors. As per the agreement, Dr. Reddy’s will pay Pregene USD 5 million upfront, and is committed to up to USD 7.5 million in milestone payments and USD 150 million sales commissions."

Licensing / partnership • Oncology

Pregene and Dr. Reddy's Announce License Agreement for Anti-BCMA CAR-T PRG1801 in India (PRNewswire) - "Dr. Reddy's Laboratories...announced an agreement whereby Dr Reddy's will acquire an exclusive license in the Republic of India for PRG1801, Pregene's single domain antibody-based anti-BCMA chimeric antigen receptor T (CAR-T) cell therapy injection...Under the terms of the license agreement, Pregene will receive an upfront payment and future milestone payments of USD 5 million for first indication and up to USD 7.5 million of milestone payments for subsequent indications...PRG1801 is an autologous anti-BCMA CAR-T therapy in development for the treatment of relapsed/refractory multiple myeloma."

Licensing / partnership • Hematological Malignancies • Multiple Myeloma • Oncology