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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