Bone Therapeutics and Rigenerand Sign Partnership For Cell Therapy Process Development

Bone Therapeutics and Rigenerand SRL, the biotech company that both develops and manufactures medicinal products for cell therapy applications, primarily for regenerative medicine and oncology, announced the signing of a first agreement for a process development partnership.
[Bone Therapeutics]
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Sarepta Therapeutics and Genevant Sciences Announce Research Collaboration for Lipid Nanoparticle-Based Gene Editing Therapeutics

Sarepta Therapeutics, Inc. and Genevant Sciences announced a research collaboration and option agreement for the delivery of LNP-gene editing therapeutics in Sarepta’s pipeline for neuromuscular diseases.
[Sarepta Therapeutics, Inc.]
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Sarepta Therapeutics and Genevant Sciences Announce Research Collaboration for Lipid Nanoparticle-Based Gene Editing Therapeutics

Sarepta Therapeutics, Inc. and Genevant Sciences announced a research collaboration and option agreement for the delivery of LNP-gene editing therapeutics in Sarepta’s pipeline for neuromuscular diseases.
[Sarepta Therapeutics, Inc.]
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Surgical Transplantation of Human RPE Stem Cell-Derived RPE Monolayers into Non-Human Primates with Immunosuppression

Scientists placed human retinal pigment epithelium (RPE) stem cell-derived RPE monolayers under the macula of a non-human primate model for three months.
[Stem Cell Reports]
Liu, Z., Parikh, B. H., Tan, Q. S. W., Wong, D. S. L., Ong, K. H., Yu, W., Seah, I., Holder, G. E., Hunziker, W., Tan, G. S. W., Barathi, V. A., Lingam, G., Stanzel, B. V., Blenkinsop, T. A., & Su, X. (2021). Surgical Transplantation of Human RPE Stem Cell-Derived RPE Monolayers into Non-Human Primates with Immunosuppression. Stem Cell Reports, 0(0). https://doi.org/10.1016/j.stemcr.2020.12.007 Cite
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Low-Dose Decitabine Priming Endows CAR T Cells with Enhanced and Persistent Antitumor Potential via Epigenetic Reprogramming

Anti-tumor activities, cytokine production, and proliferation were enhanced in decitabine-treated chimeric antigen receptor T cells both in vitro and in vivo.
[Nature Communications]
Low-dose decitabine priming endows CAR T cells with enhanced and persistent antitumour potential via epigenetic reprogramming | Nature Communications. (n.d.). Retrieved January 18, 2021, from https://www.nature.com/articles/s41467-020-20696-x Cite
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Adoptive Transfer of JC Virus-Specific T Lymphocytes for the Treatment of Progressive Multifocal Leukoencephalopathy

Scientists analyzed the safety and evaluate preliminary efficacy of polyomavirus JC-specific T cell therapy in a cohort of hematological patients with Progressive Multifocal Leukoencephalopathy.
[Annals of Neurology]
Berzero, G., Basso, S., Stoppini, L., Palermo, A., Pichiecchio, A., Paoletti, M., Lucev, F., Gerevini, S., Rossi, A., Vegezzi, E., Diamanti, L., Bini, P., Gastaldi, M., Delbue, S., Perotti, C., Seminari, E., Faraci, M., Luppi, M., Baldanti, F., … Comoli, P. (n.d.). Adoptive transfer of JC virus-specific T lymphocytes for the treatment of Progressive Multifocal Leukoencephalopathy. Annals of Neurology, n/a(n/a). https://doi.org/https://doi.org/10.1002/ana.26020 Cite
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Development of a Dual Hybrid AAV Vector for Endothelial-Targeted Expression of Von Willebrand Factor

Researchers aimed at developing a gene therapy strategy for gene delivery into endothelial cells, the natural site of biosynthesis of Von Willebrand disease.
[Gene Therapy]
Barbon, E., Kawecki, C., Marmier, S., Sakkal, A., Collaud, F., Charles, S., Ronzitti, G., Casari, C., Christophe, O. D., Denis, C. V., Lenting, P. J., & Mingozzi, F. (2021). Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor. Gene Therapy, 1–10. https://doi.org/10.1038/s41434-020-00218-6 Cite
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Chaperone-Mediated Autophagy Sustains Hematopoietic Stem-Cell Function

Scientists showed that chaperone-mediated autophagy, a selective form of lysosomal protein degradation, is involved in sustaining hematopoietic stem cell function in adult mice.
[Nature]
Dong, S., Wang, Q., Kao, Y.-R., Diaz, A., Tasset, I., Kaushik, S., Thiruthuvanathan, V., Zintiridou, A., Nieves, E., Dzieciatkowska, M., Reisz, J. A., Gavathiotis, E., D’Alessandro, A., Will, B., & Cuervo, A. M. (2021). Chaperone-mediated autophagy sustains haematopoietic stem-cell function. Nature, 1–7. https://doi.org/10.1038/s41586-020-03129-z Cite
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Successful Gene Therapy of Diamond-Blackfan Anemia in a Mouse Model and Human CD34+ Cord Blood Hematopoietic Stem Cells Using a Clinically Applicable Lentiviral Vector

Researchers observed that transduced ribosomal protein S19 (Rps19)-deficient bone marrow cells could reconstitute mice longterm and rescue the bone marrow failure and severe anemia observed in Rps19-deficient mice, with a low risk of mutagenesis and a highly polyclonal insertion site pattern.
[Haematologica]
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Beyond Conventional Immune-Checkpoint Inhibition — Novel Immunotherapies for Renal Cell Carcinoma

Using the structure provided by the well-described cancer–immunity cycle, scientists outline the key steps required for a successful antitumour immune response in the context of renal cell carcinoma, and describe the development of promising new immunotherapies within the context of this framework.
[Nature Reviews Clinical Oncology]
Braun, D. A., Bakouny, Z., Hirsch, L., Flippot, R., Van Allen, E. M., Wu, C. J., & Choueiri, T. K. (2021). Beyond conventional immune-checkpoint inhibition — novel immunotherapies for renal cell carcinoma. Nature Reviews Clinical Oncology, 1–16. https://doi.org/10.1038/s41571-020-00455-z Cite
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Nanomaterials for T-Cell Cancer Immunotherapy

Scientists present an overview of nanomaterials that have been used to overcome clinical barriers to T-cell-based immunotherapies and provide their outlook of this emerging field at the interface of cancer immunotherapy and nanomaterial design.
[Nature Nanotechnology]
Gong, N., Sheppard, N. C., Billingsley, M. M., June, C. H., & Mitchell, M. J. (2021). Nanomaterials for T-cell cancer immunotherapy. Nature Nanotechnology, 16(1), 25–36. https://doi.org/10.1038/s41565-020-00822-y Cite
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