Many CRISPR treatments are in trials, but in 2022, Vertex Pharmaceuticals, based in Boston, was first to bring one to regulators for approval. That treatment was for sickle-cell., Mar. 13, 2025 — Researchers have discovered a handful of new CRISPR-Cas systems that could add to the capabilities of the already transformational gene editing and DNA manipulation toolbox., In a decade, scientists have transformed CRISPR from a natural system used by bacteria to block viral attacks into a molecular scalpel for genetic engineering. CRISPR permits researchers to, CRISPR Therapeutics and Vertex have shared data from 17 patients with SCD and 27 patients with TDT: the results are dramatic and durable. 25 of 27 individuals with TDT were no longer transfusion dependent following the treatment, some for longer than three years., Leveraging gene editing tools such as CRISPR, researchers are now actively engineering CAR-T cells, aiming to develop allogeneic CAR-T therapies and/or enhance the persistence of autologous CAR-T., With the first CRISPR–Cas9 gene therapy now approved, scientists are turning to newer editing technologies to produce safer, faster and better treatments for genetic diseases..