Ipilimumab: An Investigational Immunotherapy for Glioblastoma

Ipilimumab inhibits CTLA-4 and is being investigated for the treatment of glioblastoma, alone or in combination with other treatment modalities, in various preclinical and clinical studies, the results of the most relevant of which are discussed in this review.
[Expert Opinion On Investigational Drugs]
Youssef, G., & Dietrich, J. (2020). Ipilimumab: an investigational immunotherapy for glioblastoma. Expert Opinion on Investigational Drugs, 0(ja), null. https://doi.org/10.1080/13543784.2020.1826436 Cite
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Distinct Macrophage Populations and Phenotypes Associated with IL-4 Mediated Immunomodulation at the Host Implant Interface

Researchers use an in vivo implantation model to analyze how immunomodulation via an IL-4 eluting implant affects distinct macrophage populations at the tissue-implant interface and how this may affect downstream regenerative processes.
[Biomaterials Science]
Hachim, D., LoPresti, S. T., Rege, R. D., Umeda, Y., Iftikhar, A., Nolfi, A. L., Skillen, C. D., & Brown, B. N. (2020). Distinct macrophage populations and phenotypes associated with IL-4 mediated immunomodulation at the host implant interface. Biomaterials Science. https://doi.org/10.1039/D0BM00568A Cite
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Ex-Vivo Gene Therapy Treats Bone Complications of Mucopolysaccharidosis Type II Mouse Models through Bone Remodeling Reactivation

Researchers investigated the pathophysiology of the bone complications associated with mucopolysaccharidosis II and the effect of lentivirus-mediated gene therapy of hematopoietic stem cells on bone lesions of mucopolysaccharidosis type II mouse models in comparison with enzyme replacement therapy.
[Molecular Therapy-Methods & Clinical Development]
Wada, M., Shimada, Y., Iizuka, S., Ishii, N., Hiraki, H., Tachibana, T., Kazuhiro, M., Saito, M., Arakawa, S., Ishimoto, T., Nakano, T., Ida, H., Ohashi, T., & Kobayashi, H. (2020). Ex-vivo gene therapy treats bone complications of mucopolysaccharidosis type II mouse models through bone remodeling reactivation. Molecular Therapy - Methods & Clinical Development, 0(0). https://doi.org/10.1016/j.omtm.2020.09.012 Cite
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Inducible Secretion of IL-21 Augments Anti-Tumor Activity of piggyBac-Manufactured Chimeric Antigen Receptor T Cells

Scientists engineered CAR T cell to co-express IL-21 and studied the effects of IL-21 on CAR T cells specific to CD19 and prostate-specific membrane antigens using an in vitro co-culture model and NSG mice transplanted with B-cell tumors.
[Cytotherapy]
Štach, M., Ptáčková, P., Mucha, M., Musil, J., Klener, P., & Otáhal, P. (2020). Inducible secretion of IL-21 augments anti-tumor activity of piggyBac-manufactured chimeric antigen receptor T cells. Cytotherapy, 0(0). https://doi.org/10.1016/j.jcyt.2020.08.005 Cite
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Adeno-Associated Virus-Mediated Gene Delivery Promotes S-Phase Entry-Independent Precise Targeted Integration in Cardiomyocytes

Scientists demonstrated that direct delivery of large amounts of transgene encoding guide RNA and repair template DNA via intra-ventricular injection of adeno-associated virus promotes precise targeted genome replacement in adult murine cardiomyocytes expressing Cas9.
[Scientific Reports]
Kohama, Y., Higo, S., Masumura, Y., Shiba, M., Kondo, T., Ishizu, T., Higo, T., Nakamura, S., Kameda, S., Tabata, T., Inoue, H., Motooka, D., Okuzaki, D., Takashima, S., Miyagawa, S., Sawa, Y., Hikoso, S., & Sakata, Y. (2020). Adeno-associated virus-mediated gene delivery promotes S-phase entry-independent precise targeted integration in cardiomyocytes. Scientific Reports, 10(1), 15348. https://doi.org/10.1038/s41598-020-72216-y Cite
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Tmunity Announces First Patient Dosed in Phase I Clinical Trial with CART-TnMUC1

Tmunity Therapeutics, Inc. announced that it has dosed the first patient in its Phase I CART-TnMUC1-01 clinical trial with the Tn/STn glycoform of mucin 1 (TnMUC1) CAR-T therapy in patients with TnMUC1-positive advanced cancers.
[Tmunity Therapeutics, Inc.]
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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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