Category Archives: Cell Therapy

Cell Therapy News is an online publication and email newsletter dedicated to sharing the latest cell therapy, gene therapy, and regenerative medicine research.
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Published weekly 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.

Arcellx Announces FDA Clearance of IND Application for ACLX-001, a Controllable Cell Therapy Utilizing the Company’s ARC-SparX Platform, for the Treatment of Multiple Myeloma

Arcellx announced that the FDA has cleared the company’s Investigational New Drug (IND) application for ACLX-001, an engineered cell therapy for the treatment of multiple myeloma. ACLX-001 is the first clinical application of the company’s ARC-SparX platform of controllable and adaptable cell therapies.
[Arcellx]
josh@comocreative.com. (2021, April 6). Arcellx Announces FDA Clearance of IND Application for ACLX-001, a Controllable Cell Therapy Utilizing the Company’s ARC-SparX Platform, for the Treatment of Multiple Myeloma. Arcellx. https://arcellx.com/arcellx-announces-fda-clearance-of-ind-application-for-aclx-001-a-controllable-cell-therapy-utilizing-the-companys-arc-sparx-platform-for-the-treatment-of-multiple-myeloma/ Cite
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Celularity Receives Orphan Drug Designation for Natural Killer Cell Therapy CYNK-001 in the Treatment of Malignant Gliomas

Celularity, Inc. announced FDA has granted Orphan Drug Designation to the company’s non-genetically modified cryopreserved human placental hematopoietic stem cell-derived natural killer cell therapy, CYNK-001, for the treatment of patients with malignant gliomas.
[Celularity, Inc.]
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Long-Term Effects of Human Induced Pluripotent Stem Cell-Derived Retinal Cell Transplantation in Pde6b Knockout Rats

Scientists evaluated the long-term effects of human induced pluripotent stem cell-derived retinal pigment epithelial and photoreceptor cell transplantation in Pde6b knockout rats to study retinitis pigmentosa.
[Experimental & Molecular Medicine]
Yang, J. M., Chung, S., Yun, K., Kim, B., So, S., Kang, S., Kang, E., & Lee, J. Y. (2021). Long-term effects of human induced pluripotent stem cell-derived retinal cell transplantation in Pde6b knockout rats. Experimental & Molecular Medicine, 1–12. https://doi.org/10.1038/s12276-021-00588-w Cite
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Hypoxia-Sensing CAR T Cells Provide Safety and Efficacy in Treating Solid Tumors

Using murine xenograft models, scientists demonstrated that, despite widespread expression of ErbB receptors in healthy organs, the approach provided anti-tumor efficacy without off-tumor toxicity.
[Cell Reports Medicine]
Kosti, P., Opzoomer, J. W., Larios-Martinez, K. I., Henley-Smith, R., Scudamore, C. L., Okesola, M., Taher, M. Y. M., Davies, D. M., Muliaditan, T., Larcombe-Young, D., Woodman, N., Gillett, C. E., Thavaraj, S., Maher, J., & Arnold, J. N. (2021). Hypoxia-sensing CAR T cells provide safety and efficacy in treating solid tumors. Cell Reports Medicine, 0(0). https://doi.org/10.1016/j.xcrm.2021.100227 Cite
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Genetic In Vivo Engineering of Human T Lymphocytes in Mouse Models

Scientists describe how to humanize immunodeficient mice with hematopoietic stem cells and precondition and administer the vector stocks.
[Nature Protocols]
Weidner, T., Agarwal, S., Perian, S., Fusil, F., Braun, G., Hartmann, J., Verhoeyen, E., & Buchholz, C. J. (2021). Genetic in vivo engineering of human T lymphocytes in mouse models. Nature Protocols, 1–31. https://doi.org/10.1038/s41596-021-00510-8 Cite
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Risk Factors and Outcomes of Diffuse Alveolar Haemorrhage after Allogeneic Hematopoietic Stem Cell Transplantation

Investigators explored the occurrence and prognosis of diffuse alveolar haemorrhage after allogeneic hematopoietic stem cell transplantation.
[Bone Marrow Transplantation]
Wu, J., Fu, H.-X., He, Y., Mo, X.-D., Liu, X., Cai, X., Gui, R.-Y., Liu, H.-X., Yan, C.-H., Chen, Y.-H., Chang, Y.-J., Xu, L.-P., Liu, K.-Y., Huang, X.-J., & Zhang, X.-H. (2021). Risk factors and outcomes of diffuse alveolar haemorrhage after allogeneic haematopoietic stem cell transplantation. Bone Marrow Transplantation, 1–11. https://doi.org/10.1038/s41409-021-01293-y Cite
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Frequent Loss-of-Heterozygosity in CRISPR-Cas9–Edited Early Human Embryos

Scientists developed computational pipelines to assess single-cell genomics and transcriptomics datasets from OCT4 CRISPR-Cas9–targeted and control human preimplantation embryos
[Proceedings of the National Academy of Sciences of the United States of America]
Alanis-Lobato, G., Zohren, J., McCarthy, A., Fogarty, N. M. E., Kubikova, N., Hardman, E., Greco, M., Wells, D., Turner, J. M. A., & Niakan, K. K. (2021). Frequent loss-of-heterozygosity in CRISPR-Cas9–edited early human embryos. Proceedings of the National Academy of Sciences. https://doi.org/10.1073/pnas.2004832117 Cite
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A Novel Full-Human CD22-CAR T Cell Therapy with Potent Activity against CD22low B-ALL

Researchers aimed to develop a new CD22-CAR construct with low immunogenicity and potent activity for treating B acute lymphoblastic leukemia (B-ALL) patients who failed from prior CD22-CAR T cell therapies.
[Blood Cancer Journal]
Tan, Y., Cai, H., Li, C., Deng, B., Song, W., Ling, Z., Hu, G., Yang, Y., Niu, P., Meng, G., Cheng, W., Xu, J., Duan, J., Wang, Z., Yu, X., Feng, X., Zhou, J., & Pan, J. (2021). A novel full-human CD22-CAR T cell therapy with potent activity against CD22 low B-ALL. Blood Cancer Journal, 11(4), 1–6. https://doi.org/10.1038/s41408-021-00465-9 Cite
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Integrative Bulk and Single-Cell Profiling of Pre-manufacture T-cell Populations Reveals Factors Mediating Long-Term Persistence of CAR T-cell Therapy

The authors extensively characterized the pre manufacture T-cells of 71 patients with B-cell malignancies on trial to receive anti-CD19 CAR T-cell therapy.
[Cancer Discovery]
Chen, G. M., Chen, C., Das, R. K., Gao, P., Chen, C.-H., Bandyopadhyay, S., Ding, Y.-Y., Uzun, Y., Yu, W., Zhu, Q., Myers, R. M., Grupp, S. A., Barrett, D. M., & Tan, K. (2021). Integrative bulk and single-cell profiling of pre-manufacture T-cell populations reveals factors mediating long-term persistence of CAR T-cell therapy. Cancer Discovery. https://doi.org/10.1158/2159-8290.CD-20-1677 Cite
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The Role of B Cells in PE Pathophysiology: A Potential Target for Perinatal Cell-Based Therapy?

The authors disscuss evidence on the ability of perinatal cells to inhibit B cell proliferation, impair B cell differentiation, and promote regulatory B cell formation.
[International Journal of Molecular Sciences]
Magatti, M., Masserdotti, A., Cargnoni, A., Papait, A., Stefani, F. R., Silini, A. R., & Parolini, O. (2021). The Role of B Cells in PE Pathophysiology: A Potential Target for Perinatal Cell-Based Therapy? International Journal of Molecular Sciences, 22(7), 3405. https://doi.org/10.3390/ijms22073405 Cite
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CRISPR Technologies for the Treatment of Duchenne Muscular Dystrophy

The authors review various therapeutic strategies that use CRISPR/Cas to correct or bypass Duchenne muscular dystrophy mutations.
[Molecular Therapy-Methods & Clinical Development]
Choi, E., & Koo, T. (2021). CRISPR Technologies for the Treatment of Duchenne Muscular Dystrophy. Molecular Therapy, 0(0). https://doi.org/10.1016/j.ymthe.2021.04.002 Cite
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