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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
47
In vivo CAR-T drug targeting CD20 based on mRNA-LNP
Dose limiting toxicity (DLT)
Time frame: Within 28 days after the initial treatment
The incidence of adverse effects
Time frame: Through study completion, an average of 2 years
Maximum tolerated dose (MTD) or optimal biological dose (OBD)
Time frame: Through study completion, an average of 2 years
0bjective response rate (ORR)
Time frame: Through study completion, an average of 2 years
Disease control rate (DCR)
Time frame: Through study completion, an average of 2 years
Duration of response (DoR)
Time frame: 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)
Time frame: 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
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.