Vor Biopharma and Arbor Biotechnologies to Collaborate on Engineered Hematopoietic Stem-Cell Therapies

Vor Biopharma and Arbor Biotechnologies announced an agreement to use Arbor’s gene editing technologies to engineer hematopoietic stem cells, towards the goal of developing therapies for the treatment of blood cancers, such as acute myeloid leukemia.
[Vor Biopharma]
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AVROBIO Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease

AVROBIO, Inc. announced that the European Commission has granted orphan drug designation for AVR-RD-02, the company’s investigational gene therapy for the treatment of Gaucher disease.
[AVROBIO, Inc.]
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Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months After Administration

Sarepta Therapeutics, Inc. announced positive results from the ongoing study of SRP-9003 the company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E.
[Sarepta Therapeutics, Inc.]
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Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months after Administration

Sarepta Therapeutics, Inc. announced positive results from the ongoing study of SRP-9003, the company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E.
[Sarepta Therapeutics, Inc. (GlobeNewswire, Inc.)]
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A Modified Human Myogenin Promoter That Is Highly Active in Alveolar Rhabdomyosarcoma

Investigators designed a suicide gene therapy by generating an ARMS-targeted promoter to drive the herpes simplex virus thymidine kinase suicide gene.
[Cancer Gene Therapy]
A modified human Myogenin promoter that is highly active in alveolar rhabdomyosarcoma | Cancer Gene Therapy. (n.d.). Retrieved September 25, 2020, from https://www.nature.com/articles/s41417-020-00225-0 Cite
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Reduced Dose of Posttransplant Cyclophosphamide in HLA-Haploidentical Peripheral Blood Stem Cell Transplantation

Scientists conducted two consecutive prospective multicenter phase II studies to evaluate the safety and efficacy of 80 mg/kg of PTCy in 137 patients who underwent HLA-haploidentical peripheral blood stem cell transplantation following reduced-intensity conditioning
[Bone Marrow Transplantation]
Sugita, J., Kamimura, T., Ishikawa, T., Ota, S., Eto, T., Kuroha, T., Miyazaki, Y., Kumagai, H., Matsuo, K., Akashi, K., Taniguchi, S., Harada, M., & Teshima, T. (2020). Reduced dose of posttransplant cyclophosphamide in HLA-haploidentical peripheral blood stem cell transplantation. Bone Marrow Transplantation, 1–9. https://doi.org/10.1038/s41409-020-01065-0 Cite
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BATF3 Programs CD8+ T Cell Memory

Investigators showed that BATF3 was expressed transiently within the first days after T cell priming and had long-lasting T cell–intrinsic effects.
[Nature Immunology]
Ataide, M. A., Komander, K., Knöpper, K., Peters, A. E., Wu, H., Eickhoff, S., Gogishvili, T., Weber, J., Grafen, A., Kallies, A., Garbi, N., Einsele, H., Hudecek, M., Gasteiger, G., Hölzel, M., Vaeth, M., & Kastenmüller, W. (2020). BATF3 programs CD8 + T cell memory. Nature Immunology, 1–11. https://doi.org/10.1038/s41590-020-0786-2 Cite
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Calreticulin del52 and ins5 Knock-In Mice Recapitulate Different Myeloproliferative Phenotypes Observed in Patients with MPN

Researchers generated heterozygous and homozygous conditional inducible knock-in mice expressing a chimeric murine CALR del52 or ins5 with the human mutated C-terminal tail to investigate their pathogenic effects on hematopoiesis.
[Nature Communications]
Benlabiod, C., Cacemiro, M. da C., Nédélec, A., Edmond, V., Muller, D., Rameau, P., Touchard, L., Gonin, P., Constantinescu, S. N., Raslova, H., Villeval, J.-L., Vainchenker, W., Plo, I., & Marty, C. (2020). Calreticulin del52 and ins5 knock-in mice recapitulate different myeloproliferative phenotypes observed in patients with MPN. Nature Communications, 11(1), 4886. https://doi.org/10.1038/s41467-020-18691-3 Cite
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Morphology of Tumor and Nontumor Tissue in Liver Resection Specimens for Hepatocellular Carcinoma following Nivolumab Therapy

Twelve patients were treated with nivolumab in the neoadjuvant setting, whereas eight were treated with nivolumab, usually along with other therapies, before undergoing liver transplantation.
[Modern Pathology]
Simoes, C. C., Thung, S. N., Fiel, M. I., Sung, M. W., Schwartz, M. E., & Ward, S. C. (2020). Morphology of tumor and nontumor tissue in liver resection specimens for hepatocellular carcinoma following nivolumab therapy. Modern Pathology, 1–11. https://doi.org/10.1038/s41379-020-00679-5 Cite
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Translation Elongation Factor 2 Depletion by siRNA in Mouse Liver Leads to mTOR-Independent Translational Upregulation of Ribosomal Protein Genes

Researchers demonstrated the feasibility of perturbing protein synthesis in a mouse liver by targeting translation elongation factor 2 with RNAi. They were able to achieve over 90% knockdown efficacy and maintain it for two weeks effectively slowing down the rate of translation elongation.
[Scientific Reports]
Gerashchenko, M. V., Nesterchuk, M. V., Smekalova, E. M., Paulo, J. A., Kowalski, P. S., Akulich, K. A., Bogorad, R., Dmitriev, S. E., Gygi, S., Zatsepin, T., Anderson, D. G., Gladyshev, V. N., & Koteliansky, V. E. (2020). Translation elongation factor 2 depletion by siRNA in mouse liver leads to mTOR-independent translational upregulation of ribosomal protein genes. Scientific Reports, 10(1), 15473. https://doi.org/10.1038/s41598-020-72399-4 Cite
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CAR T Cell Therapies for Patients with Multiple Myeloma

Scientists highlight potential future innovations that could enhance the efficacy and/or reduce the toxicities associated with CAR T cell therapies.
[Nature Reviews Clinical Oncology]
CAR T cell therapies for patients with multiple myeloma | Nature Reviews Clinical Oncology. (n.d.). Retrieved September 25, 2020, from https://www.nature.com/articles/s41571-020-0427-6 Cite
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