“The spirit of the collaboration since the beginning has been that there was no waiting on formality,” Vallabh says. “As soon as we realized our mutual excitement to do this, everything was off to the races.” Co-corresponding authors Weissman and…
The MHRA's extension drew primarily on data from two Phase 3 pediatric trials: CLIMB-151, which enrolled children aged two to eleven with SCD, and CLIMB-141, which enrolled children aged two to eleven with TDT. "These results — the first clinical data ever…
... UK Healthcare Regulator OKs Vertex Pharmaceuticals' Casgevy Gene Therapy for Children From Age Five Published on 10/30/2026 at 05:30 am EDT
The FDA on Oct. 27 released draft guidance that could speed approval of some gene-editing treatments built for a single patient's genetic mutation, a step rare disease families have pushed for since a Philadelphia infant received the first personalized CRISPR-based therapy…
The Academy of Medical Sciences of Nigeria has called on the Federal Government to increase investment in biomedical research to develop affordable treatments for diseases and prepare the country for future pandemics. It said sustained funding for genomic research would…
An experimental treatment has kept one patient with heavily pretreated hairy cell leukemia free of detectable disease for more than two years, raising questions about whether CAR T-cell therapy could offer a path toward a cure. After almost four decades…
A new review maps how mutations in more than twenty genes weaken bone in osteogenesis imperfecta and surveys therapies from bisphosphonates to CRISPR gene editing. Subject of Research: Genetic mechanisms and emerging therapies for osteogenesis imperfecta, a rare inherited bone…
Hands are made by killing. The embryonic limb bud arrives as a paddle and fingers appear only once the tissue between them has received the ‘time to die’ memo. Pruning is not damage control. It is construction. The architecture is…
An international study reports 60% progression-free survival at 10 years after autologous bone marrow transplantation, adding important evidence on sustained positive clinical outcomes and long-term safety in patients treated for early severe systemic sclerosis. The NISSC1-LTE study conducted by the Autoimmune Diseases…
Rare mutations that shut down a single gene are linked to lower cholesterol levels and a 50 percent reduction in the risk of heart attack, according to new research from Washington University School of Medicine in St. Louis, the Broad Institute…
CAR‑T, CRISPR, in vivo delivery, manufacturing, and long‑term follow‑up.
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