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Cell Therapy News is an online resource dedicated to sharing the latest cell therapy, gene therapy, and regenerative medicine research.
AAV Gene Therapy for Autosomal Recessive Deafness 9: A Single-Arm Trial
[Nature Medicine] Investigators conducted a single-arm trial of adeno-associated virus (AAV)-OTOF gene therapy using the Anc80L65 capsid in ten participants with autosomal recessive deafness 9 aged 1.5 to 23.9 years at five sites in China.
Rewiring Endogenous Genes in CAR T Cells for Tumor-Restricted Payload Delivery
[Nature] Researchers have developed a CRISPR knock-in strategy that leverages the regulatory mechanisms of endogenous genes to drive transgene expression in a tumor-localized manner.
Bioengineered Immunocompetent Preclinical Trial-on-Chip Tool Enables Screening of CAR T Cell Therapy for Leukemia
[Nature Biomedical Engineering] Scientists engineered an organotypic immunocompetent chip that recapitulates microarchitectural and pathophysiological characteristics of human leukemia bone marrow stromal and immune niches for CAR T cell therapy modeling.
Humoral Vaccine Responses following Chimeric Antigen Receptor T-Cell Therapy for Hematological Malignancies
[Blood Cancer Journal] This single-center, retrospective study analyzed vaccine responses in patients who received post-CAR T-cell therapy vaccination between 2018 and 2024.
A Cross-Talk Established by Tumor-Targeted Cytokines Rescues CAR T Cell Activity and Engages Host T Cells against Glioblastoma in Mice
[Science Translational Medicine] The authors used an engineered hematopoietic stem cell–based platform that delivers cytokines to the tumor microenvironment to improve the efficacy of B7-homolog 3–targeting CAR T cells in an orthotopic syngeneic mouse model of glioblastoma.
Engineered CD4 TCR T Cells with Conserved High-Affinity TCRs Targeting NY-ESO-1 for Advanced Cellular Therapies in Cancer
[Science Advances] Researchers screened HLA-DRB3*02:02 patients with melanoma for tumor-specific CD4 T cells and identified robust New York esophageal squamous cell carcinoma 1/HLA-DRB3*02:02 CD4 T cell activity in both peripheral blood and tumor tissue.
Targeted In Vivo Delivery of Genetic Medicines Utilizing an Engineered Lentiviral Vector Platform Results in CAR T and NK Cell Generation
[Molecular Therapy] Investigators described the development of a lentiviral platform based on a novel, detargeted viral fusogen and a membrane-bound targeting moiety to enable in vivo targeted delivery of stably integrating genetic medicines without the need for lymphodepletion.
Sustained High Expression of Human FVII following AAV8-Mediated Gene Delivery in Mice
[Molecular Therapy-Methods & Clinical Development] The authors explored whether AAV mediated gene therapy can achieve durable and functional expression of human FVII in vivo.
U of T Engineering Researchers and Startup boutIQ Solutions Partner to Advance Heart Repair Therapies
[U of T Engineering News] Professors Craig Simmons and Julie Audet, in collaboration with boutIQ solutions Inc., a University of Toronto (U of T) spinout company specializing in AI-driven media optimization, has been awarded a $421,000 research grant through the Stem Cell Network’s 2025 Research Funding Competition.
STEMCELL Technologies Introduces STEMprepâ„¢ Tissue Dissociator System to Accelerate Research Discoveries
[STEMCELL Technologies] To help scientists accelerate their workflows, STEMCELL Technologies has commercially launched the STEMprep™ Tissue Dissociator System—a new benchtop instrument that automates, standardizes, and streamlines tissue dissociation.
AAV Mini-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy: A Phase Ib Trial
[Nature Medicine] Pediatric ambulatory male participants with a genetic Duchenne muscular dystrophy diagnosis and receiving stable glucocorticoids received a single intravenous low-dose or high-dose fordadistrogene movaparvovec.
Modified hTERT Promoters-Driven Purine Nucleoside Phosphorylase-Gene Therapy in Association with chemo- and Targeted Therapy in the Context of Ovarian Cancer
[Cancer Gene Therapy] Researchers proposed an original ovarian cancer treatment through the use of two recently developed modified hTERT promoters: the mutated hTERT and the chimeric hTERT-CMV.
Published since 2002, Cell Therapy News is our longest running online publication. Our editorial team curates the top publications, reviews, and news on in vivo gene therapy, cell therapy, and immunotherapy research. With over 15,000 subscribers and followers across our website, email newsletter, and Twitter feed, we are proud to facilitate the communication of cutting-edge cell therapy innovations.
