Gene therapy and gene editing: CRISPR, clinical trials and the first approved treatments.
Reducing the 10–21 day expansion phase in CAR T-cell therapy production lowers the dose required and speeds up delivery, addressing the fact that 30% of annual therapies go unused due to patient deterioration.
Blocking an age-related protein restored cartilage in aged mice and prevented osteoarthritis after injuries, while human cartilage began producing new functional tissue.
Five researchers from Harvard and NIH received the 2026 Warren Alpert Foundation prize for identifying BCL11A as a key regulator of the fetal-to-adult hemoglobin switch, enabling the development of Casgevy, the first CRISPR medicine for sickle cell disease and thalassemia.
A study of 24 dogs showed reduced biological age and muscle improvements after receiving GF-1004 plasmid DNA therapy.