CAR T-cell therapy works by engineering a patient’s T cells to express synthetic receptors that recognize and kill cancer cells without relying on HLA presentation. This approach has shown remarkable success in relapsed or refractory B-cell cancers and multiple myeloma, resulting in several approved treatments. However, no CAR T therapy is currently approved for acute myeloid leukemia (AML) or T-cell acute lymphoblastic leukemia (T-ALL).
Despite the success of traditional viral-based CAR T-cell therapies against several blood malignancies, their efficacy remains limited against solid tumors. Non-viral engineering of CAR T cells using electroporation or lipid nanoparticle delivery of CAR-encoding mRNA achieves high but transient CAR expression, highlighting the limitations of current preclinical models for evaluating mRNA-based CAR T cells.
Hangzhou Qihan Biotech Co. Ltd. has gained IND approval by the FDA for QT-019B, a universal, dual-target CAR T-cell therapy for refractory systemic lupus erythematosus (SLE). A phase I/II trial will be conducted in the U.S.
Cptx GmbH’s QUIET-CAR collaborative project with Nanocell Therapeutics Inc. has been awarded a Eurostars Grant from the EU through the Horizon Europe program and Eureka Network. The QUIET-CAR project aims to develop targeted lipid nanoparticles carrying novel immune-silent single-stranded DNA (ssDNA) for in vivo CAR T therapy.
Orbital Therapeutics Inc. has presented preclinical results supporting the development of OTX-201, a potential best-in-class in vivo CAR T therapy that comprises an optimized circular RNA encoding a CD19-targeted CAR delivered via targeted lipid nanoparticles.
Despite the increasing sophistication of anticancer therapies, many malignancies continue to evade treatment. T cells expressing chimeric antigen receptor (CAR) can effectively attack some tumors by recognizing antigens expressed on the tumor surface, but the therapy may prove ineffective if the target antigen is not abundant enough throughout the tumor.
Aussie researchers have used CRISPR gene editing tools to “armor” chimeric antigen receptor (CAR) T cells to activate additional cancer-fighting proteins at the tumor site, enabling them to target cancer cells in solid tumors.
Aussie researchers have used CRISPR gene editing tools to “armor” chimeric antigen receptor (CAR) T cells to activate additional cancer-fighting proteins at the tumor site, enabling them to target cancer cells in solid tumors.
Glioblastoma is the most frequent and aggressive primary brain cancer in adults, and patients can expect to live shorter than 2 years, regardless of therapy. The cancer can be treated with CAR T cells, but many patients develop resistance because tumors mutate or delete the antigens recognized by the T cells, while the tumor microenvironment suppresses T-cell activity.
Myrio Therapeutics Pty Ltd. is set to advance lead product PHOX2B PC-CAR T (PHOX2B peptide-centric chimeric antigen receptor autologous T cells) into clinical trials for relapsed neuroblastoma following IND clearance by the FDA. The first patient will be enrolled around mid-year.
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