Genetic Diseases in Active Gene Editing Clinical Trials and Progress (As of May 2025, based on global clinical trial data) I. Hematologic Disorders 1. Sickle Cell Disease (SCD) & β-Thalassemia Candidate Therapies: exa-cel (CTX001): Co-developed by CRISPR Therapeutics and Vertex, this therapy act … Genetic Diseases in Active Gene Editing Clinical Trials and ProgressRead more
Genetic Disorder
Genetic Diseases Treatable with Gene Editing and Key Case Studies
Genetic Diseases Treatable with Gene Editing and Key Case Studies (As of May 2025) Gene editing technologies, particularly CRISPR-Cas9, have enabled precise interventions for monogenic disorders while making breakthroughs in polygenic diseases, mitochondrial disorders, and rare conditions. Below is … Genetic Diseases Treatable with Gene Editing and Key Case StudiesRead more