Vertex, CRISPR Therapeutics Share Promising Exa-Cel Data Readout in SCD, Beta-Thalassemia at ASGCT
The gene therapy improved hemoglobin levels in transfusion-dependent beta-thalassemia patients and hindered severe vaso-occlusive crises in those with sickle cell disease.
Suzhou GenAssist Therapeutics Gets Pre-IND Consultation With FDA for DMD Gene-Editing Therapy
GEN6050 is designed to restore expression of dystrophin by skipping exon 50 in the DMD gene through in vivo base editing.
Survey Suggests Polygenic Embryo Screening Has Higher Public Acceptance Than Anticipated
A national survey of more than 6,800 US participants asked about attitudes towards polygenic embryo screening, germline genome editing, and SAT prep courses.
Cardiac-Related Consequences of Alcohol Flushing Genetic Variant Reduced by Drug
With endothelial cells derived from induced pluripotent stem cells, researchers tracked functional changes and potential treatment targets in alcohol flushing variant carriers.
Cancer Patients in Early Trial Receive Personalized, CRISPR-Edited T-Cell Therapy
The PACT Pharma-funded study found that the edited T cells trafficked to patients' solid tumors and indicated that the treatment was well tolerated.