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[SOTIO Biotech] SOTIO Biotech announced that the US FDA has granted SOT106 Orphan Drug Designation for the treatment of soft tissue sarcoma and Fast Track Designation for the treatment of osteosarcoma, supporting its planned clinical development as an investigational antibody-drug conjugate (ADC) across multiple sarcoma subtypes.

[Amphista Therapeutics Limited ] Amphista Therapeutics Limited announced dosing of the first patient in a Phase I trial for its lead candidate, AMX-883. Initiated following authorization from the US FDA, the BRAMLIE trial will investigate the safety, pharmacokinetics, and preliminary efficacy of AMX-883 monotherapy in participants with AML and high-risk MDS1.

[Leukemia] Investigators elucidated the role of PTEN-induced putative kinase 1 (PINK1), a key sensor of mitochondrial stress, in regulating mitophagy in leukemic cells.

[Cell Stem Cell] Scientists developed a massively parallel single-cell CRISPR screening platform in primary human hematopoietic stem and progenitor cells (HSPCs), profiling 322 regulators across 172,382 single-perturbed cells.

[Blood Cancer Journal] In this Phase I/II study, the authors evaluated the safety and efficacy of an all-oral regimen of decitabine/cedazuridine, venetoclax, and gilteritinib in adults with FLT3-mutated AML and other myeloid malignancies.

[Scientific Reports] Researchers evaluated the effects of localized periodontal ligament-derived MSC (PDL-MSC) sheet transplantation on mandibular wound healing in zoledronate-treated beagle dogs.

[Cell Reports] The authors identified MSC-derived lactate as a signaling metabolite that reprograms monocyte metabolism and differentiation. In experimental autoimmune uveoretinitis, intravenous administration of MSCs ameliorated the disease, but oxamate-pretreated or HIF1A-knockdown MSCs exhibited reduced efficacy.

[Trends In Molecular Medicine] Scientists introduce ‘organellar governance’ as a framework for understanding MSC-mediated bone regeneration. They discuss how adaptive organelle remodeling supports osteogenesis and how organelle regulation is disrupted in disease.

[Chinese Medical Journal] The authors discuss the key MSC-regulated signaling pathways and their therapeutic potential to provide a theoretical foundation for diabetic kidney disease-related drug development and its clinical translation.

[PloS One] Scientists investigated SARS-CoV-2 variant-associated effects in adult MSCs and umbilical cord blood CD34 ⁺ hematopoietic stem and progenitor cells using in vitro exposure models.

[Science Advances] Using mice in which ALIX, a key regulator of small extracellular vesicle (sEV) biogenesis, was selectively deleted in PDGFRα+ mesenchymal progenitors, scientists demonstrated a critical role for mesenchymal sEV production in metabolic regulation.

[Reproductive Sciences] Scientists established a mouse model of premature ovarian failure induced by cyclophosphamide (CTX) treatment. Mice were treated with bone mesenchymal stem cells for four weeks.