Cell & Gene Therapies

7+ hour, 14+ min ago   (858+ words) “We are not always beholden to the misspellings in our DNA,” Liu said. KJ’s story “really made people realize that it is possible — although not easy — to diagnose a child with an otherwise fatal genetic disorder, generate a customized gene-editing…...

11+ hour, 14+ min ago   (88+ words) Crispr genome editing could in theory be programmed to address any gene mutation, providing cures on demand for life-threatening inherited rare diseases in newborns. The challenge is to develop a platform that can be applied at scale and with buy-in…...

23+ hour, 14+ min ago   (446+ words) EMJ This site is intended for healthcare professionals Fatal Inflammatory Risks Associated with CAR T-Cell Therapy - IEC-HS occurred in 4.7% of patients receiving CAR T-cell therapy, typically emerging ten days post-infusion. - High baseline ferritin, elevated LDH, cytopenias, and high tumor burden…...

11+ hour, 58+ min ago   (141+ words) Bulgarian scientists have helped reveal how mutations in the H1.4 gene cause Rahman syndrome, a rare disorder associated with severe neurodevelopmental problems and distinctive facial features. An international study found that the mutated H1.4 histone protein loses its electrical charge and can…...

11+ hour, 45+ min ago   (478+ words) Meditope-enabled CAR T cells can be tracked, selectively expanded, and directed toward additional tumor antigens using externally administered protein adaptors A modular CAR T-cell platform could allow clinicians to modify the behavior and targeting of engineered immune cells after they…...

15+ hour, 20+ min ago   (744+ words) Researchers say study results support advancing treatment to human trials Written by Michela Luciano, PhD | Sept. 23, 2026 The treatment involves engineering immune T-cells to selectively kill B-cells producing self-reactive antibodies (autoantibodies) against the acetylcholine receptor (AChR). This protein is found on…...

16+ hour, 25+ min ago   (633+ words) Yahoo Finance UK UK markets close in 2h 9m Autologous Hematopoietic Stem Cell Gene Therapy Market Global Report 2026: A $13.44 Billion Market by 2030 as Novartis, Vertex, CRISPR Therapeutics and Orchard Therapeutics Accelerate the Gene-Editing Race Boost ROI via rare-disease R&D, CRISPR therapies,…...

17+ hour, 47+ min ago   (609+ words) A new preclinical study describes the meCAR T platform, a meditope-based docking system that allows molecular add-ons to be delivered to CAR T cells already in the body. Researchers at City of Hope have developed a new ‘plug-and-play’ technology that…...

1+ day, 2+ hour ago   (79+ words) In June, Chinese regulators approved the world’s first CAR-T cell therapy for solid tumour cancers, developed by a drug company based in Shanghai. One month later, at a hospital in the city, a New Zealander with advanced cancer of the…...

1+ day, 13+ min ago   (721+ words) A rare and devastating inherited immune disorder may have a new path toward treatment, thanks to a proof-of-concept study demonstrating that CRISPR base editors can correct disease-causing mutations in the Artemis gene outside the body....