Combination Hematopoietic Stem Cell Transplantation and Intravenous AAVrh10-Mediated Gene Therapy in a Canine Model Proves Pivotal for Translation of Krabbe Disease Therapy.
Investigators evaluated hematopoietic stem cell transplantation (HSCT), intravenous (IV) AAVrh10 gene therapy, and combination HSCT + IV AAVrh10 in the canine model of globoid cell leukodystrophy.
[FDA] The FDA has received reports of T cell malignancies, including chimeric antigen receptor CAR-positive lymphoma, in patients who received treatment with BCMA- or CD19-directed autologous CAR T cell immunotherapies.
[Nature Biomedical Engineering] Investigators showed that replacement of the extracellular domains of heterodimeric cytokine receptors in T cells with two leucine zipper motifs provided optimal Janus kinase (JAK)/signal transducer and activator of transcription (STAT) signaling.
[blood] The authors presented the clinicogenomic characterization of a CARTITUDE-4 patient who developed a CAR+ T cell lymphoma post cilta-cel. Diagnostic and staging workup included biopsy analyses and FDG-PET scan.