CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies. The FDA's Fast Track program is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical ...
Good news at ASH 2020 for CRISPR Therapeutics and Vertex Pharma as their gene-editing therapy CTX001 improves outcomes in thalassaemia and sickle cell patients
“The study of CTX001 in β-thalassemia will be the first company-sponsored clinical trial of a CRISPR-based therapy and is a major step forward for both the treatment of certain inherited blood diseases and for our colla...
These data further suggest an avenue for effective combination therapy in treating solid tumors.doi:10.1371/journal.pone.0053900Moon CHLee SJLee HYLee JCCha HCho WJPark JWPark HJSeo JLee YHPlos One