Gene Editing for the Treatment of Primary Immunodeficiency Diseases

The authors summarize the progresses made towards the development of gene editing technologies to treat Primary immunodeficiency diseases and the optimizations that still need to be implemented to turn genome editing into a next-generation lifesaving treatment for rare monogenic life-threatening disorders.
[Human Gene Therapy]
Rai, R., Thrasher, A. J., & Cavazza, A. (2020). Gene editing for the treatment of Primary Immunodeficiency Diseases. Human Gene Therapy. https://doi.org/10.1089/hum.2020.185 Cite
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Allogeneic Vγ9Vδ2 T-Cell Immunotherapy Exhibits Promising Clinical Safety and Prolongs the Survival of Patients with Late-Stage Lung or Liver Cancer

Researchers developed a novel formula to improve the expansion of peripheral γδ T cells from healthy donors. Then, they used a humanized mouse model to validate the therapeutic efficacy of expanded γδ T cells in vivo; furthermore, the expanded γδ T cells were adoptively transferred into late-stage liver and lung cancer patients.
[Cellular & Molecular Immunology]
Xu, Y., Xiang, Z., Alnaggar, M., Kouakanou, L., Li, J., He, J., Yang, J., Hu, Y., Chen, Y., Lin, L., Hao, J., Li, J., Chen, J., Li, M., Wu, Q., Peters, C., Zhou, Q., Li, J., Liang, Y., … Yin, Z. (2020). Allogeneic Vγ9Vδ2 T-cell immunotherapy exhibits promising clinical safety and prolongs the survival of patients with late-stage lung or liver cancer. Cellular & Molecular Immunology, 1–13. https://doi.org/10.1038/s41423-020-0515-7 Cite
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Kiadis Receives $9.5 Million from the Advanced Regenerative Manufacturing Institute’s BioFabUSA Program to Fund Kiadis K-NK Cell COVID-19 Therapy

Kiadis Pharma N.V. announced that Kiadis has received $9.5 million in funding from the Advanced Regenerative Manufacturing Institute’s BioFabUSA program, in partnership with the United States Department of Defense, to fund Kiadis’ K-NK-ID101 program.
[Kiadis Pharma N.V.]
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Perforin-Deficient CAR T Cells Recapitulate Late-Onset Inflammatory Toxicities Observed in Patients

Scientists investigated the role of perforin in anti-CD19 chimeric antigen receptor T cell efficacy and hemophagocytic lymphohistiocytosis-like toxicities in a syngeneic murine model.
[Journal of Clinical Investigation]
Ishii, K., Pouzolles, M., Chien, C. D., Erwin-Cohen, R. A., Kohler, M. E., Qin, H., Lei, H., Kuhn, S., Ombrello, A. K., Dulau-Florea, A., Eckhaus, M. A., Shalabi, H., Yates, B., Lichtenstein, D. A., Zimmermann, V. S., Kondo, T., Shern, J. F., Young, H. A., Taylor, N., … Fry, T. J. (2020). Perforin-deficient CAR T cells recapitulate late-onset inflammatory toxicities observed in patients. The Journal of Clinical Investigation, 130(10). https://doi.org/10.1172/JCI130059 Cite
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REGENXBIO Announces Dosing of First Patient in Phase II AAVIATE Trial of RGX-314 for the Treatment of Wet AMD Using Suprachoroidal Delivery

REGENXBIO Inc. announced that the first patient has been dosed in the AAVIATE trial, a Phase II trial to evaluate the suprachoroidal delivery of RGX-314 using the SCS Microinjector for the treatment of wet age-related macular degeneration.
[REGENXBIO Inc.]
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The Sustained Expression of Cas9 Targeting Toxic RNAs Reverses Disease Phenotypes in Mouse Models of Myotonic Dystrophy Type 1

Using adult and neonatal mouse models of DM1, scientists showed that intramuscular or systemic injections of adeno-associated virus vectors encoding nuclease-dead Cas9.
[Nature Biomedical Engineering]
Batra, R., Nelles, D. A., Roth, D. M., Krach, F., Nutter, C. A., Tadokoro, T., Thomas, J. D., Sznajder, Ł. J., Blue, S. M., Gutierrez, H. L., Liu, P., Aigner, S., Platoshyn, O., Miyanohara, A., Marsala, M., Swanson, M. S., & Yeo, G. W. (2020). The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1. Nature Biomedical Engineering, 1–12. https://doi.org/10.1038/s41551-020-00607-7 Cite
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ImCheck Achieves Initial Positive Independent Safety Review and Initiates Next Stage of Phase I/IIa EVICTION Trial for ICT01

ImCheck Therapeutics announced that the independent Safety Review Committee for the EVICTION Phase I/II clinical trial for its lead antibody ICT01 unanimously approved dose escalation in the solid tumor indications and the start of enrollment in the two other arms of the study: treatment with ICT01 in patients with hematologic malignancies as monotherapy and in solid tumor patients in combination with pembrolizumab.
[ImCheck Therapeutics]
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Potential of Mesenchymal Stem Cells as Topical Immunomodulatory Cell Therapies for Ocular Surface Inflammatory Disorders

Researchers highlight considerations, including whether the source of mesenchymal stem cell (MSC) isolation impacts the efficacy and safety of the therapy, in addition to assessing the feasibility of MSC topical application to the cornea and ocular surface through analysis of potential scaffolds and cell carriers for application to the eye.
[Stem Cells Translational Medicine]
Beeken, L. J., Ting, D. S. J., & Sidney, L. E. (n.d.). Potential of mesenchymal stem cells as topical immunomodulatory cell therapies for ocular surface inflammatory disorders. STEM CELLS Translational Medicine, n/a(n/a). https://doi.org/10.1002/sctm.20-0118 Cite
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Developing Bio-Inspired 3D Models of Brain Cancer to Evaluate Tumor-Homing Neural Stem Cell Therapy

When neural stem cells (NSCs) were implanted 2 mm lateral from the tumor foci, NSCs co-localized with the GBM within seven days. In models of multi-focal disease, NSCs were found to co-localize with multiple tumors, preferentially migrating to tumor foci closest to the site of NSC implantation.
[Tissue Engineering]
Carey-Ewend, A. G., Hagler, S. B., Bomba, H. N., Goetz, M. J., Bago, J. R., & Hingtgen, S. D. (2020). Developing Bio-Inspired 3D Models of Brain Cancer to Evaluate Tumor-Homing Neural Stem Cell Therapy. Tissue Engineering Part A. https://doi.org/10.1089/ten.TEA.2020.0113 Cite
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Mannitol Augments the Effects of Systemical Stem Cell Transplantation without Increasing Cell Migration in a Stroke Animal Model

Researchers elucidated the neuroprotective effects of a combination of mannitol pretreatment and stem cell transplantation on stroke-induced neural injury.
[Journal of Tissue Engineering and Regenerative Medicine]
Lee, S.-H., Kang, H.-Y., Kim, J.-H., & Park, D.-H. (2020). Mannitol Augments the Effects of Systemical Stem Cell Transplantation without Increasing Cell Migration in a Stroke Animal Model. Tissue Engineering and Regenerative Medicine. https://doi.org/10.1007/s13770-020-00293-1 Cite
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In Vivo HSC Gene Therapy Using a Bi-Modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model

Investigators generated a HDAd5/35++ adenovirus vector containing both modules and tested it in vitro and after in vivo hematopoietic stem cell/progenitor transduction in “healthy” CD46/β-YAC mice and in a Sickle Cell Disease mouse model.
[Molecular Therapy]
Li, C., Wang, H., Georgakopoulou, A., Gil, S., Yannaki, E., & Lieber, A. (2020). In vivo HSC gene therapy using a bi-modular HDAd5/35++ vector cures Sickle Cell Disease in a mouse model. Molecular Therapy, 0(0). https://doi.org/10.1016/j.ymthe.2020.09.001 Cite
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