Vor Biopharma Announces Collaboration with Janssen to Develop Engineered Hematopoietic Stem Cell Transplants Combined with a Bi-Specific Antibody Therapy for Acute Myeloid Leukemia (AML)

Vor Biopharma, a cell therapy company pioneering engineered hematopoietic stem cell therapies combined with targeted therapies for the treatment of cancer, announced the formation of a collaboration with Janssen Biotech, Inc.
[Vor Biopharma]
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The Role of the PZP Domain of AF10 in Acute Leukemia Driven by AF10 Translocations

Incorporation of functional PZP domain of AF10 into the leukemogenic CALM-AF10 fusion prevented the transforming activity of the fusion in bone marrow-derived hematopoietic stem and progenitor cells in vitro and in vivo and abrogated CALM-AF10-mediated leukemogenesis in vivo.
[Nature Communications]
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De Novo Generation of Macrophage from Placenta-Derived Hemogenic Endothelium

Researchers uncovered and experimentally validated that a CD44+ subpopulation of placental endothelial cells exhibited hemogenic potential.
[Developmental Cell]
Liang, G., Zhou, C., Jiang, X., Zhang, Y., Huang, B., Gao, S., Kang, Z., Ma, D., Wang, F., Gottgens, B., Wang, H., Han, J.-D. J., & Liu, F. (2021). De novo generation of macrophage from placenta-derived hemogenic endothelium. Developmental Cell. https://doi.org/10.1016/j.devcel.2021.06.005 Cite
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Clonal Hematopoiesis and Its Emerging Effects on Cellular Therapies

Scientists review the relevance and complications of clonal hematopoiesis for the treatment of hematologic malignancies with cell therapeutic approaches.
[Leukemia]
von Bonin, M., Jambor, H. K., Teipel, R., Stölzel, F., Thiede, C., Damm, F., Kroschinsky, F., Schetelig, J., Chavakis, T., & Bornhäuser, M. (2021). Clonal hematopoiesis and its emerging effects on cellular therapies. Leukemia, 1–7. https://doi.org/10.1038/s41375-021-01337-8 Cite
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Luspatercept as a Therapy for Myelodysplastic Syndromes with Ring Sideroblasts

Luspatercept is a promising alternative to ESAs for a subset of transfusion-dependent patients with lower risk myelodysplastic syndromes, namely those with a sideroblastic phenotype who are either not suitable for or have already failed erythropoietin-based treatment.
[Expert Review of Hematology]
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Diet-Regulated Production of PDGFcc by Macrophages Controls Energy Storage

Scientists found that resident macrophage deficiency prevents storage of lipids in adipocytes from wild-type and Ccr2–/– mice fed a high-fat diet, as well as hyperphagic leptin receptor–deficient mice.
[Science]
Cox, N., Crozet, L., Holtman, I. R., Loyher, P.-L., Lazarov, T., White, J. B., Mass, E., Stanley, E. R., Elemento, O., Glass, C. K., & Geissmann, F. (2021). Diet-regulated production of PDGFcc by macrophages controls energy storage. Science, 373(6550). https://doi.org/10.1126/science.abe9383 Cite
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Predicting Genotoxicity of Viral Vectors for Stem Cell Gene Therapy Using Gene Expression-Based Machine Learning

Researchers demonstrated that genotoxic vectors induced a unique gene expression signature linked to stemness and oncogenesis in transduced murine hematopoietic stem- and progenitor cells, and developed the Surrogate Assay for Genotoxicity Assessment.
[Molecular Therapy]
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Spatial Transcriptome Profiling by MERFISH Reveals Fetal Liver Hematopoietic Stem Cell Niche Architecture

Scientists investigated the spatial organization and transcriptional profile of individual HSCs in both wild type and Tet2−/− fetal livers, by multiplexed error robust fluorescence in situ hybridization.
[Cell Discovery]
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Increasing Recognition and Emerging Therapies Argue for Dedicated Clinical Trials in Chronic Myelomonocytic Leukemia

Investigators discuss recent therapeutic advances and innovative treatment strategies in chronic myelomonocytic leukemia (CMML), including global and molecularly targeted approaches, and discuss what may help to make progress in the design of rationally derived and disease-modifying therapies for CMML.
[Leukemia]
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Optimizing the Treatment of Acute Lymphoblastic Leukemia in Younger and Older Adults: New Drugs and Evolving Paradigms

With the development of the anti-CD22 antibody-drug conjugate inotuzumab ozogamicin, the CD3-CD19 bispecific T-cell engager antibody blinatumomab, CD19 chimeric antigen receptor T-cell therapy, and the potent BCR-ABL1 tyrosine kinase inhibitor ponatinib, the outlook of acute lymphoblastic leukemia in both younger and older adults has substantially improved.
[Leukemia]
Short, N. J., Kantarjian, H., & Jabbour, E. (2021). Optimizing the treatment of acute lymphoblastic leukemia in younger and older adults: new drugs and evolving paradigms. Leukemia, 1–15. https://doi.org/10.1038/s41375-021-01277-3 Cite
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Reduced Dose of PTCy Followed by Adjuvant α-Galactosylceramide Enhances GVL Effect without Sacrificing GVHD Suppression

Researchers investigated the effects of reducing the dose of posttransplantation cyclophosphamide (PTCy) followed by α-galactosylceramide, a ligand of iNKT cells, on the reciprocal balance between graft-versus-host disease (GVHD) and the graft-versus-leukemia (GVL) effect.
[Scientific Reports]
Nakamura, M., Meguri, Y., Ikegawa, S., Kondo, T., Sumii, Y., Fukumi, T., Iwamoto, M., Sando, Y., Sugiura, H., Asada, N., Ennishi, D., Tomida, S., Fukuda-Kawaguchi, E., Ishii, Y., Maeda, Y., & Matsuoka, K. (2021). Reduced dose of PTCy followed by adjuvant α-galactosylceramide enhances GVL effect without sacrificing GVHD suppression. Scientific Reports, 11(1), 13125. https://doi.org/10.1038/s41598-021-92526-z Cite
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