Ocugen Receives Fourth FDA Orphan Drug Designation for the Same Product, OCU400 (AAV-NR2E3) Gene Therapy, for the Treatment of Another Key Inherited Retinal Disease (IRD), PDE6B Gene Mutation-Associated Retinal Diseases

Ocugen, Inc. announced the FDA granted the fourth Orphan Drug Designation for OCU400 in the treatment of PDE6B gene mutation-associated retinal diseases.
[Ocugen, Inc.]
Press Release

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Immediate Intracoronary Delivery of Human Umbilical Cord Mesenchymal Stem Cells Reduces Myocardial Injury by Regulating the Inflammatory Process via Cell-Cell Contact with T Lymphocytes

To investigate the effects of human umbilical cord mesenchymal stem cell (HUCMSC) delivery on the acute inflammatory stage of ischemia reperfusion injury, the authors transplanted HUCMSCs or HUCMSCs with cyclosporin A via the coronary artery simultaneously during ischemia reperfusion in pigs.
[Stem Cells and Development]
Liu, C., Kang, L.-N., Chen, F., Mu, D., Shen, S., Wang, K., Hu, J.-X., Xie, J., & Xu, B. (2020). Immediate intracoronary delivery of human umbilical cord mesenchymal stem cells reduces myocardial injury by regulating the inflammatory process via cell-cell contact with T lymphocytes. Stem Cells and Development. https://doi.org/10.1089/scd.2019.0264 Cite
Abstract

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Mesenchymal Stem/Stromal Cells Therapy for Sepsis and Acute Respiratory Distress Syndrome

The authors focus on the therapeutic potential of mesenchymal stem/stromal cells (MSCs). MSCs are available from several tissues, including bone marrow, umbilical cord, and adipose tissue.
[Seminars in Respiratory and Critical Care Medicine]
Byrnes, D., Masterson, C. H., Artigas, A., & Laffey, J. G. (2020). Mesenchymal Stem/Stromal Cells Therapy for Sepsis and Acute Respiratory Distress Syndrome. Seminars in Respiratory and Critical Care Medicine. https://doi.org/10.1055/s-0040-1713422 Cite
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Sangamo Announces Global Collaboration with Novartis to Develop Genomic Medicines for Autism and other Neurodevelopmental Disorders

Sangamo Therapeutics, Inc. announced that it has executed a global licensing collaboration agreement with Novartis to develop and commercialize gene regulation therapies to address three neurodevelopmental targets, including autism spectrum disorder and other neurodevelopmental disorders.
[Sangamo Therapeutics]
Press Release

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Celularity Announces the Activation of First California Clinical Trial Site following CIRM Grant Award to Advance Treatments for COVID-19

Celularity announced that it has been awarded a $750,000 COVID-19 Project grant by the California Institute for Regenerative Medicine (CIRM), one of the three clinical awards targeting the coronavirus. This grant will support California Institutions participating in the Phase I/II clinical trial of human placental hematopoietic stem cell derived natural killer cells for the treatment of adults with COVID-19.
[Celularity Inc.]
Press Release

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Gene Therapy for Alpha 1-Antitrypsin Deficiency with an Oxidant-Resistant Human Alpha 1-Antitrypsin

Scientists hypothesized that a onetime administration of a serotype 8 adeno-associated virus gene transfer vector coding for the oxidation-resistant variant alpha 1-antitrypsin (AAT) would maintain antiprotease activity under oxidant stress compared with normal AAT.
[JCI Insight]
Sosulski, M. L., Stiles, K. M., Frenk, E. Z., Hart, F. M., Matsumura, Y., De, B. P., Kaminsky, S. M., & Crystal, R. G. (2020). Gene therapy for alpha 1-antitrypsin deficiency with an oxidant-resistant human alpha 1-antitrypsin. JCI Insight, 5(15). https://doi.org/10.1172/jci.insight.135951 Cite
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Mesenchymal Stem Cell Therapy for Ischemic Stroke: A Look into Treatment Mechanism and Therapeutic Potential

Researchers show the transplantation protocol for mesenchymal stem cells (MSCs), discuss the potential therapeutic mechanisms, and summarize clinical trials on MSCs for treating ischemic stroke.
[Journal of Neurology]
Li, J., Zhang, Q., Wang, W., Lin, F., Wang, S., & Zhao, J. (2020). Mesenchymal stem cell therapy for ischemic stroke: A look into treatment mechanism and therapeutic potential. Journal of Neurology. https://doi.org/10.1007/s00415-020-10138-5 Cite
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Cardiomyocyte Transplantation after Myocardial Infarction Alters the Immune Response in the Heart

The authors showed for the first time that the immune response is altered as a result of syngeneic neonatal cardiomyocyte transplantation after myocardial infarction leading to improved cardiac pump function as observed by magnetic resonance imaging in C57BL/6J mice.
[Cells]
Vasudevan, P., Wolfien, M., Lemcke, H., Lang, C. I., Skorska, A., Gaebel, R., Koczan, D., Lindner, T., Engelmann, R., Vollmar, B., Krause, B. J., Wolkenhauer, O., Lang, H., Steinhoff, G., & David, R. (2020). Cardiomyocyte Transplantation after Myocardial Infarction Alters the Immune Response in the Heart. Cells, 9(8), 1825. https://doi.org/10.3390/cells9081825 Cite
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Hematopoietic Stem Cell Transplantation and Chimeric Antigen Receptor T Cell for Relapsed or Refractory Diffuse Large B-Cell Lymphoma

Scientists treated 14 relapsed or refractory diffuse large B-cell lymphoma patients by combining autologous hematopoietic stem cell transplantation and anti-CD19 chimeric antigen receptor T-cell therapy.
[Immunotherapy]
Wang, T., Gao, L., Wang, Y., Zhu, W., Xu, L., Wang, Y., Yue, W., Tang, G., Chen, L., Chen, J., Zhang, W., Yu, X., Feng, D., & Yang, J. (2020). Hematopoietic stem cell transplantation and chimeric antigen receptor T cell for relapsed or refractory diffuse large B-cell lymphoma. Immunotherapy. https://doi.org/10.2217/imt-2020-0075 Cite
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A Lung Tropic AAV Vector Improves Survival in a Mouse Model of Surfactant B Deficiency

Scientists report a rationally designed adeno-associated virus 6 capsid that demonstrates efficiency in lung epithelial cell transduction based on imaging and flow cytometry analysis.
[Nature Communications]
Kang, M. H., van Lieshout, L. P., Xu, L., Domm, J. M., Vadivel, A., Renesme, L., Mühlfeld, C., Hurskainen, M., Mižíková, I., Pei, Y., van Vloten, J. P., Thomas, S. P., Milazzo, C., Cyr-Depauw, C., Whitsett, J. A., Nogee, L. M., Wootton, S. K., & Thébaud, B. (2020). A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency. Nature Communications, 11(1), 3929. https://doi.org/10.1038/s41467-020-17577-8 Cite
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In Vivo Gene Therapy for Canine SCID-X1 Using Cocal-Pseudotyped Lentiviral Vector

Scientists developed an in vivo gene therapy approach to treat canine X-linked severe combined immunodeficiency after hematopoietic stem and progenitor cell mobilization and systemic delivery of the therapeutic vector.
[Human Gene Therapy]
Rajawat, Y. S., Humbert, O., Cook, S. M., Radtke, S., Pande, D., Enstrom, M., Wohlfahrt, M. E., & Kiem, H.-P. (2020). In vivo gene therapy for canine SCID-X1 using cocal-pseudotyped lentiviral vector. Human Gene Therapy. https://doi.org/10.1089/hum.2020.127 Cite
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