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Malignant hematological tumors mainly derived from adult B cells are mainly acute lymphoblastic leukemia (ALL) and non Hodgkin lymphoma (NHL). Overall, although existing therapies have significantly improved the survival rates of most patients, the treatment of relapsed/refractory patients still faces significant challenges. CD20 is a transmembrane protein highly expressed on the surface of B cells, almost penetrating the precursor, mature, and activated stages of B cells, but lacking in plasma cells, making it an ideal target for B cell malignancies.
In recent years, the breakthrough development of in vivo CAR-T therapy has overturned the traditional paradigm of in vitro CAR-T technology. The core principle is to directly deliver the gene encoding chimeric antigen receptor (CAR) to T cells in the patient's body through gene delivery vectors, without the need for in vitro isolation, modification, and amplification processes, and to complete the gene reprogramming of T cells in vivo. At present, the mainstream carrier technologies for CAR-T therapy in vivo are divided into two categories: lentiviral carriers and lipid nanoparticle (LNP) carriers. LNP carriers have significantly broken through the clinical bottlenecks of traditional CAR-T in terms of cost and accessibility, safety, and timeliness.
This experimental drug is a CD20 based messenger ribonucleic acid (mRNA) therapeutic drug, which is an injection formed by loading mRNA onto lipid nanoparticles (LNP). It has shown efficient B-cell clearance activity and good safety in non clinical settings, supporting further clinical exploration in B-cell hematological malignancies. It is expected to provide an innovative, safe, and accessible immunotherapy for B-cell hematological malignancies and bring better clinical benefits to more patients with B-cell hematological malignancies.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| in vivo CAR-T drug, Escalation doses | Experimental | In vivo CAR-T drug targeting CD20 based on mRNA-LNP |
|
| in vivo CAR-T drug, Extended doses | Experimental | In vivo CAR-T drug targeting CD20 based on mRNA-LNP |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| In vivo CAR-T drug targeting CD20 based on mRNA-LNP | Drug | In vivo CAR-T drug targeting CD20 based on mRNA-LNP |
|
| Measure | Description | Time Frame |
|---|---|---|
| Dose limiting toxicity (DLT) | Within 28 days after the initial treatment | |
| The incidence of adverse effects | Through study completion, an average of 2 years | |
| Maximum tolerated dose (MTD) or optimal biological dose (OBD) | Through study completion, an average of 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| 0bjective response rate (ORR) | Through study completion, an average of 2 years | |
| Disease control rate (DCR) | Through study completion, an average of 2 years | |
| Duration of response (DoR) |
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Inclusion Criteria:
Exclusion Criteria:
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| From the date of the first PR/CR to the date of first comfired progression or date of death from any cause, whichever came first, assessed up to 24 months |
| Progression free survival (PFS) | From date of initial treatment until the date of first comfired progression or date of death from any cause, whichever came first, assessed up to 24 months |
| ID | Term |
|---|---|
| D019337 | Hematologic Neoplasms |
| ID | Term |
|---|---|
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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