rimiducid (AP1903) / Bellicum - LARVOL DELTA

Early results from a phase 1, multicenter trial of PSCA-specific GoCAR T cells (BPX-601) in patients with metastatic castration-resistant prostate cancer (mCRPC). (ASCO-GU 2023) - P1/2 | "BPX-601, a PSCA-directed GoCAR-T cell product, has preliminary evidence of biologic activity with toxicity characteristic of previously reported CAR-T studies. Markers of rimiducid-induced GoCAR-T cell activation and proliferation were observed. Exploration of escalating weekly rimiducid doses > 0.4 mg/kg and BPX-601 cell doses is planned."

CAR T-Cell Therapy • Clinical • IO biomarker • Metastases • P1 data • Genito-urinary Cancer • Oncology • Prostate Cancer • Solid Tumor • CCL4 • CD40 • CSF2 • CX3CL1 • IFNG • IL18 • IL1R1 • IL6R • MYD88 • PSCA

Intravenous GD2-CAR T Cell Therapy in Pediatric and Young Adult CNS Tumors: preliminary data (EANO 2026) - P1 | "Steroids were required in 37% of patients and AP1903 in 21%. GD2-CART01 is feasible in pediatric and young adult patients with high-risk CNS tumors, with encouraging preliminary efficacy."

CAR T-Cell Therapy • Clinical • Brain Cancer • CNS Tumor • Diffuse Intrinsic Pontine Glioma • Diffuse Midline Glioma • Embryonal Tumor • Glioma • High Grade Glioma • Medulloblastoma • Oncology

GD2-targeting CAR T cells in high-risk neuroblastoma: a phase 1/phase 2 trial. (PubMed, Nat Med) - P1/2 | "Grade 3 immune effector cell-associated neurotoxicity syndrome was diagnosed in four children and rapidly controlled with the activation of the inducible caspase-9 suicide gene by rimiducid. These results confirm that GD2-CART01 can induce durable remissions in children with high-risk metastatic, relapsed, or refractory neuroblastoma. ClinicalTrials.gov identifier: NCT03373097 ."

Journal • P1 data • P2 data • Neuroblastoma • Oncology • Solid Tumor

An inducible signal 1 booster overcomes limited access to antigen for solid tumor cell therapy. (PubMed, Mol Ther Oncol) - "These signal 1 boosters are variants of the previously identified MyD88-CD40 fusion protein but mitigate the excessive antigen sensitization of this molecule. We identified a booster that, when expressed with CAR or Tmod, produces small-molecule-inducible T cell expansion and activation while maintaining a favorable safety profile in a surrogate normal-tissue mouse model."

Journal • Hematological Malignancies • Oncology • Solid Tumor • CD40 • MYD88

A Study to Evaluate the Safety, Tolerability, Cellular Kinetics, Pharmacodynamics, and Efficacy of P-CD19CD20-ALLO1 in Participants With Severe, Treatment-refractory Systemic Lupus Erythematosus (SLE) (clinicaltrials.gov) - P1 | N=0 | Withdrawn | Sponsor: Genentech, Inc. | Not yet recruiting ➔ Withdrawn | N=162 ➔ 0

Enrollment change • Trial withdrawal • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

Anti-CD19 CAR-T Cells With Inducible Caspase 9 Safety Switch for B-cell Lymphoma (clinicaltrials.gov) - P1 | N=12 | Active, not recruiting | Sponsor: UNC Lineberger Comprehensive Cancer Center | N=19 ➔ 12

Enrollment change • B Cell Lymphoma • Chronic Lymphocytic Leukemia • Classical Hodgkin Lymphoma • Follicular Lymphoma • Hematological Malignancies • Hodgkin Lymphoma • Indolent Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma • ALK • BCL2 • BCL6 • IRF4

T-Cell Therapy for Advanced Breast Cancer (clinicaltrials.gov) - P1 | N=186 | Active, not recruiting | Sponsor: Memorial Sloan Kettering Cancer Center | Trial primary completion date: Jun 2026 ➔ Jun 2027 | Trial completion date: Jun 2026 ➔ Jun 2027

Trial completion date • Trial primary completion date • Breast Cancer • HER2 Breast Cancer • HER2 Negative Breast Cancer • Oncology • Solid Tumor • HER-2 • MSLN • PD-L1

Phase 2 Trial of CD70.CAR NK Cells for Patients With Primary Refractory or Early Relapsed Diffuse Large B-Cell Lymphoma and Hodgkin Lymphoma (clinicaltrials.gov) - P2 | N=100 | Not yet recruiting | Sponsor: M.D. Anderson Cancer Center | Initiation date: Jun 2026 ➔ Dec 2026

Trial initiation date • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Hodgkin Lymphoma • Large B Cell Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Intravenous GD2 CAR T-Cell Therapy in Pediatric and Young Adult Central Nervous System Tumors: Preliminary Data (ISPNO 2026) - P1 | "Steroids were required in 37% of patients and AP1903 in 21%. GD2-CART01 is feasible in pediatric and young adult patients with high-risk CNS tumors, with encouraging preliminary efficacy."

CAR T-Cell Therapy • Clinical • Brain Cancer • CNS Tumor • Diffuse Intrinsic Pontine Glioma • Diffuse Midline Glioma • Embryonal Tumor • Glioma • High Grade Glioma • Medulloblastoma • Oncology • Pediatrics • Solid Tumor

Administration of Autologous CAR-T CD19 Antigen With Inducible Safety Switch in Patients With Relapsed/Refractory ALL (clinicaltrials.gov) - P1 | N=15 | Active, not recruiting | Sponsor: UNC Lineberger Comprehensive Cancer Center | Phase classification: P1/2 ➔ P1

Phase classification • Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • BCR • CD19

From a research staple to clinical approval for the versatile chemical inducer of dimerization rimiducid (ASGCT 2026) - "In addition to use in regulating clinically relevant gene therapy-based protein delivery, we have applied this technology toward targeted, visceral fat elimination that may surpass the selectivity of current GLP-1 agonists and other incretins. Since visceral fat is regarded as the major cause for the ill health effects associated with obesity, this application alone could have major health implications."

Clinical • IO biomarker • Gene Therapies • Genetic Disorders • Graft versus Host Disease • Hematological Malignancies • Immunology • Obesity • Sarcopenia • Solid Tumor • CASP9 • CD40 • FKBP5 • MYD88

SELECT-NK-THYM: Target-Selected CAR-NK Cells (CD30, CD5, or Mesothelin) for Relapsed/Refractory B2 Thymoma or Thymic Carcinoma (clinicaltrials.gov) - P1/2 | N=36 | Recruiting | Sponsor: Beijing Biotech

New P1/2 trial • Oncology • Solid Tumor • Thymic Carcinoma • Thymoma • Thymus Cancer • CD5 • MSLN • TNFRSF8

P-CD19CD20-ALLO1-001: P-CD19CD20-ALLO1 Allogeneic CAR-T Cells in the Treatment of Subjects With B Cell Malignancies (clinicaltrials.gov) - P1 | N=120 | Active, not recruiting | Sponsor: Poseida Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • High-grade B-cell lymphoma • Indolent Lymphoma • Large B Cell Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Mediastinal Large B-Cell Lymphoma

Phase 1b Study Of TRICK-NK In Combination With T-Dxd In Treatment-Refractory Breast Cancers (clinicaltrials.gov) - P1 | N=60 | Not yet recruiting | Sponsor: M.D. Anderson Cancer Center

New P1 trial • Breast Cancer • HER2 Positive Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • HER-2 • TGFBR2

Phase 2 Trial of CD70.CAR NK Cells for Patients With Primary Refractory or Early Relapsed Diffuse Large B-Cell Lymphoma and Hodgkin Lymphoma (clinicaltrials.gov) - P2 | N=100 | Not yet recruiting | Sponsor: M.D. Anderson Cancer Center | Initiation date: Feb 2026 ➔ Jun 2026

Trial initiation date • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Hodgkin Lymphoma • Large B Cell Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

P-BCMA-ALLO1-001: P-BCMA-ALLO1 Allogeneic CAR-T Cells in the Treatment of Subjects With Multiple Myeloma (clinicaltrials.gov) - P1 | N=275 | Active, not recruiting | Sponsor: Poseida Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Hematological Malignancies • Multiple Myeloma • Oncology

A Study to Evaluate the Safety, Tolerability, Cellular Kinetics, Pharmacodynamics, and Efficacy of P-CD19CD20-ALLO1 in Participants With Severe, Treatment-refractory Systemic Lupus Erythematosus (SLE) (clinicaltrials.gov) - P1 | N=162 | Recruiting | Sponsor: Genentech, Inc. | Initiation date: Feb 2026 ➔ Jun 2026

Trial initiation date • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

Phase 1/2 Window Of Opportunity Study Of TROP2 CAR/IL-15 TGFBR2 KO NK Cells Delivered Intraperitoneally For The Management Of Gastric Cancer Metastatic To The Peritoneum (clinicaltrials.gov) - P1/2 | N=10 | Not yet recruiting | Sponsor: M.D. Anderson Cancer Center

New P1/2 trial • Gastric Adenocarcinoma • Gastric Cancer • Oncology • Ovarian Cancer • Solid Tumor

Functional screen to optimize logic gate potency and selectivity (AACR 2025) - "In another set of experiments, a library of almost 30,000 variants of a signal 1 booster that activate signaling in the presence of a small molecule (rimiducid) was screened. The screening system described here identifies active blocker receptors and boosters from large libraries of variants. Its throughput is sufficient to generate large datasets and train machine learning models to better predict and optimize the function and control of logic-gated cell therapeutics."

Oncology

A Study to Evaluate the Safety, Tolerability, Cellular Kinetics, Pharmacodynamics, and Efficacy of P-CD19CD20-ALLO1 in Participants With Severe, Treatment-refractory Systemic Lupus Erythematosus (SLE) (clinicaltrials.gov) - P1 | N=162 | Recruiting | Sponsor: Genentech, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

Safety and Activity Study of PSCA-Targeted CAR-T Cells (BPX-601) in Subjects With Selected Advanced Solid Tumors (clinicaltrials.gov) - P1 | N=52 | Terminated | Sponsor: Regeneron Pharmaceuticals | Phase classification: P1/2 ➔ P1 | N=151 ➔ 52 | Suspended ➔ Terminated; Due to a Dose Limiting Toxicity.

Enrollment change • Phase classification • Trial termination • Castration-Resistant Prostate Cancer • Genito-urinary Cancer • Oncology • Prostate Cancer • Solid Tumor

Optimizing B7-H3.CAR T-cell Therapy for Medulloblastoma: Preclinical Evidence Supporting CD28.OX40ζ Design (EHA-EBMT-CART 2026) - "Moreover, the dimerizing drug AP1903 was able to cross the murine blood brain barrier and to eliminate circulating B7-H3.CAR T-cells. These findings support the translation of B7-H3.CAR T-cell therapy into clinical trials for patients with B7-H3-positive MB. Conclusions : The B7-H3.CAR.CD28.OX40ζ T construct exhibits robust preclinical efficacy and represents the most promising candidate for clinical evaluation in a phase I/II trial for pediatric patients with medulloblastoma."

CAR T-Cell Therapy • IO biomarker • Preclinical • Brain Cancer • Medulloblastoma • Solid Tumor • CD276

Translational Development of CD70-directed NanoCAR T-cells for Clear Cell Renal Cell Carcinoma: Efficacy, Safety and Innovation (EHA-EBMT-CART 2026) - "For translational safety, an inducible caspase-9 (iC9) suicide switch was incorporated, enabling rapid elimination of engineered T cells upon rimiducid exposure...Patient-derived samples have been used to establish a xenograft model, and in vivo testing in this clinically relevant setting will now be initiated. If these studies confirm the promising in vitro findings, this nanoCAR platform could represent a significant step forward in adapting CAR T cell therapy for solid tumors."

CAR T-Cell Therapy • Clinical • Clear Cell Renal Cell Carcinoma • Diffuse Large B Cell Lymphoma • Genito-urinary Cancer • Hematological Malignancies • Oncology • Solid Tumor • CD70

Using the iCasp9 suicide strategy to control the growth and function of genome-edited B cells with redirected antigen specificity. (PubMed, Mol Ther Oncol) - "Although AP1903 treatment strongly curbed edited cell survival, this was constantly followed by the selection of resistant cells with lowered expression of both iCasp9 and the therapeutic antibody cassette. Therefore, in adoptive immunotherapy protocols, the iCasp9/AP1903 safety switch could stand as an efficient neoadjuvant therapy, as well as a rheostat to modulate the infusion of a therapeutic molecule."

IO biomarker • Journal • B Cell Lymphoma • Hematological Malignancies • Lymphoma • Oncology • CASP9

A nucleic acid sensing system for selective therapeutic protein expression in breast cancer (SABCS 2025) - "Transfection of both linear and gap fusion-targeting sensors into MCF7 cells and subsequent treatment with iCaspase9 inducer drug AP1903 resulted in a significant reduction in cell viability (up to 84% 36-hours post sensor transfection vs. non-targeting controls, p < 0.0001)... This study demonstrates the successful design and in vitro proof-of-principle of a biomolecule sensing system that selectively triggers a therapeutic protein effector in the presence of a cancer-specific nucleic acid. These findings have the potential to establish a novel therapeutic paradigm-in which an RNA medicine can repurpose cancer-defining nucleic acid sequences into a targeted, modular, and precise cell-killing mechanism. Our ongoing efforts focus on optimizing sensor design, interrogating mechanisms of sensor-mediated cell killing with diverse protein payloads, and addressing challenges of in vivo delivery."

Breast Cancer • HER2 Breast Cancer • HER2 Positive Breast Cancer • Oncology • Solid Tumor • ADAR • ER • FGFR • HER-2 • NTRK • SOX9 • SULF2