FDA grants CTX001 fast-track eligibility for blood transfusion-dependent beta thalassemia

recently, gene editing(http://CRISPR Therapeutics and partner VertexPharmaceutical(http://announced that the U.SFood andMedicines(http://Administration (
FDA(http://has granted CTX001 fast-track eligibility for the treatment of blood transfusion-dependent beta thalassemia (TDT)in January, the FDA also granted CTX001 fast-track eligibility to treat sickle cell disease (SCD)CTX001 is an experimental, self-contained, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy that has the potential to become a one-time, potential cure for patients with severe hemoglobinCTX001 is an experimental in vitro (ex vivo), CRISPR gene-editing therapy that is currently evaluating the potential of treating Patients with TDT and severe SCD; HbF is an oxygen-carrying protein that is naturally present at birth and later replaced by adult hemoglobinThe rise of CTX001 to HBF has the potential to alleviate blood transfusion needs in Patients with TDT, as well as the painful and decaying sickle of Patients with SCD 　　CRISPR Therapeutics and Vertex partnered in 2015 to develop a strategic research collaboration focusing on the use of CRISPR/Cas9 to identify and develop potential new therapies for the underlying genetic causes of human diseases CTX001 is the first treatment to be born in a joint research project The two companies will jointly develop and commercialize CTX001 and share all research and development costs and profits equally worldwide 　　In February, the two sides announced that the first patient had received CTX001 treatment in a Phase I/II study evaluating CTX001 treatment for TDT The patient treatment also marks the first company-sponsored use of CRISPR/Cas9 therapy in clinical trial (http:// The open label Phase I/II is designed to assess the safety and efficacy of single-dose CTX001 treatment in patients aged 18-35 with TDT non-beta 0/beta 0 subtype in addition, both sides are also investigating the CTX001 treatment SCD In February, a severe SCD I/II study in the United States was the first in a group of patients, and CTX001 is expected to be infusion in mid-2019 Currently, patient recruitment in these two studies is still ongoing