A single systemic dose of an immune-shielded lentiviral vector delivering the MMUT gene produced lasting metabolic correction in a mouse model of methylmalonic acidemia, according to newly published preclinical data.
News|Articles|June 19, 2025
Top News and Expert Insights for World Sickle Cell Day 2025
Author(s)Noah Stansfield
In honor of World Sickle Cell Day, observed annually on June 19, we took a look back at news and expert insights from the past year in cell and gene therapy for SCD.
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The landscape of care for sickle cell disease (SCD) is rapidly changing. On a single day in 2023—December 8—the field of hematology was shaken by the FDA’s simultaneous approval of 2 gene therapy products for the treatment of SCD: Vertex Pharmaceuticals' and CRISPR Therapeutics’
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The FDA approved Tregzi as the first allogeneic regulatory T cell-based immunotherapy based on Precision-T phase 3 data showing a significant 1-year cGVHD-free survival rate of 78% vs 38% with conventional alloHSC.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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