Augmentation of NK Cell Proliferation and Anti-Tumor Immunity by Transgenic Expression of Receptors for Erythropoietin or Thrombopoietin

To reduce or preclude the necessity for IL-2 use, scientists investigated whether genetic engineering of NK cells to express the erythropoietin receptor or thrombopoietin receptor could be used as a method to improve NK cell survival and function.
[Molecular Therapy]
Chanswangphuwana, C., Allan, D. S. J., Chakraborty, M., Reger, R. N., & Childs, R. W. (2020). Augmentation of NK Cell Proliferation and Anti-tumor Immunity by Transgenic Expression of Receptors for Erythropoietin or Thrombopoietin. Molecular Therapy, 0(0). https://doi.org/10.1016/j.ymthe.2020.09.023 Cite
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Papyrus Therapeutics Inc. Signs Research Collaboration with Oxford Biomedica

Papyrus Therapeutics, Inc. and Oxford Biomedica plc announced the signing of a research collaboration agreement, which includes the assessment of the impact and therapeutic benefit of Papyrus’ PYTX-002, a potential first-in-class gene replacement therapy.
[Papyrus Therapeutics, Inc.]
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Tmunity Announces First Patient Dosed in Phase 1 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 chimeric antigen receptor T-cell therapy in patients with TnMUC1-positive advanced cancers.
[Tmunity Therapeutics, Inc.]
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Stem/Progenitor Cell-Based Transplantation for Retinal Degeneration: A Review of Clinical Trials

Scientists provide a comprehensive overview on the progression of clinical trials for retinal degeneration treatment using four types of stem/progenitor cell-based transplantation to replace degenerative retinal cells and/or to supplement trophic factors from the aspects of safety, effectiveness and their respective advantages and disadvantages.
[Cell Death & Disease]
Wang, Y., Tang, Z., & Gu, P. (2020). Stem/progenitor cell-based transplantation for retinal degeneration: a review of clinical trials. Cell Death & Disease, 11(9), 1–14. https://doi.org/10.1038/s41419-020-02955-3 Cite
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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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