Gene Therapy
Gene-edited CAR-T cells show promise against aggressive T-cell leukemia
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Scientists at University College London and Great Ormond Street Hospital have developed a base-edited therapy called BE-CAR7 that uses universal CAR T-cells to treat relapsed or refractory T-cell acute lymphoblastic leukemia. Early trial results published in the New England Journal of Medicine and presented at the American Society of Hematology Annual Meeting indicate deep remissions in most patients, including those who did not respond to standard treatments, by tackling long-standing challenges in T-cell–based therapies.
Researchers at the University of Waterloo have developed engineered bacteria designed to invade and eat solid tumors from the inside out. The approach uses microbes that thrive in oxygen-free environments, targeting the low-oxygen cores of tumors. A genetic modification allows the bacteria to survive near oxygenated edges, controlled by a quorum-sensing mechanism.
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The US Food and Drug Administration has granted fast-track status to an innovative inhalable gene therapy for advanced lung cancer, following promising early trial results. Developed by Krystal Biotech, the treatment uses a modified virus to deliver immune-boosting genes directly to lung cells via inhalation. In initial tests, it shrank tumors in some patients and halted growth in others.
The U.S. Food and Drug Administration has granted accelerated approval to Forzinity, the first treatment for Barth syndrome, a rare X-linked genetic disorder primarily affecting males. Developed by Stealth Biotherapeutics, the therapy targets mitochondrial dysfunction underlying the condition, offering new hope for patients with this life-threatening disease. This milestone follows years of advocacy and highlights progress in addressing ultra-rare disorders.
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Targeted nanomachines for clearing senescent cells in reproductive tissues, along with gene therapies for telomerase reactivation, were reported on September 11, 2025.