[Cancer Cell] To elucidate factors associated with CAR-T therapy resistance, investigators conducted high-dimensional analyses of pre- and post-CAR-T cell specimens.

[Advanced Healthcare Materials] A new family of ionizable cationic lipids called “Self-Immolative Lipids” is reported. This innovative lipid architecture was designed to overcome a critical challenge in non-viral gene therapy: the need for a delivery vector that is both efficacious and stable during biodistribution while remaining biodegradable upon reaching the target cell.

[Nature Communications] Scientists described the development of a genetically-controlled, yet versatile mouse modeling platform allowing delivery of different genetic lesions by muscle electroporation in wildtype mice.

[Molecular Therapy] A novel Pompe rat model was used to test the hypothesis that neonatal gene therapy with adeno-associated virus serotype 9 restores cardiorespiratory neuromuscular function across the lifespan.

[Molecular Therapy] Investigators reported first-in-human experience with a covalently closed-end adeno-associated virus vector. High quality clinical grade cceAAV vector based on AAV9 produced in 200 liters of suspension 293 cells with a total yield of 4.3 × 10¹⁶ vector genomes.

[Cancer Cell] Researchers performed genome-wide CRISPR screens to reveal the complete IL-15R signaling mechanism in natural killer cells and discovered that ubiquitin-dependent IL-15R degradation is the dominant mechanism restraining IL-15R signaling.

[Cell Reports Methods] Scientists developed a light-triggerable circular RNA system for dynamic LbCas12a regulation. By employing circular CRISPR guide RNA (crRNA) or a split circular universal direct repeat region with a replaceable spacer, they resolved the incompatibility between isothermal amplification and CRISPR detection.

[Clinical Cancer Research] The authors write a foundational overview of the classification of cell and gene therapy in treating solid tumors and explore how it targets the cross-talk between cancer cells and the tumor immune microenvironment from cellular and molecular perspectives.

[Mesoblast (GlobeNewswire)] Mesoblast provided an update on continued momentum with US FDA regarding both accelerated approval pathway for Revascor^® in the treatment of patients with ischemic chronic heart failure with reduced ejection fraction and inflammation, and label extension for Ryoncil^® in adults with steroid refractory acute graft versus host disease (SR-aGvHD).

[Teva Pharmaceuticals] Teva Pharmaceuticals and Fosun Pharma announced that they have entered into strategic partnership for the development of investigational TEV-56278, an anti-PD1 therapy in Phase I for the treatment of cancer, including melanoma.

[Galderma (BioSpace)] Galderma announced that nemolizumab has been recommended for routine funding on the National Health Service in England and Wales for moderate-to-severe atopic dermatitis in final draft guidance from the National Institute for Health and Care Excellence.

[Immunity] Deletion of Na_v1.8+ nociceptors, cDC1-specific ablation of the CGRP receptor, or treatment with a CGRP receptor antagonist abrogated CD8⁺ T cell autoreactivity and prevented skin depigmentation in a mouse model of vitiligo.