Latest Research Highlights
The Journey from Lab to Clinic: Zolgensma, the First Gene Therapy Treatment for Spinal Muscular Atrophy (SMA)
Translational research bridges the gap between scientific discoveries and real-world applications, transforming scientific breakthroughs into life-changing therapies. Success in this endeavor hinges on several factors, including collaboration between scientists and clinicians. A notable example of this is the approval of Zolgensma, a gene therapy for spinal muscular atrophy (SMA), which marked a significant milestone in the field of translational research.
CAR T-Cell Therapy and the Cytokine Storm: The Balancing Act at the Center of Next-Generation Cancer Therapies
Recent advances in chimeric antigen receptor (CAR) T-cell therapy have opened exciting new avenues for the treatment of a variety of malignancies. Cytokine release syndrome (CRS), a powerful immune response to activated CAR T cells, is the primary side effect of CAR T-cell therapy, but existing CRS treatments can make CAR T-cell treatment less effective. In two recent studies, researchers used multiplexed cytokine immunoassays to better understand how CAR T-cell therapy triggers CRS and to identify strategies for early intervention in CRS development.
