ALdawg
Member
CRSP
Interesting news on CRSP clinical trial at ASH meeting coming up with oral presentation on 12/6. This abstract was selected for the plenary session and the abstract is now public. The researchers modified patient stem cells to turn on fetal hemoglobin which is resistant to effects of thalassemia and Sickle cell disease. Normally fetal hemoglobin is turned off after birth. Fetal hemoglobin was reactivated in all seven patients. So far it looks like they may have cured Sickle cell disease in two patient with no transfusions or admissions for vast-occlusive crisis. Time and more patients will tell but this is very exciting. Similar results in thalassemia patients.
Paper: Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9–Modified CD34+ Hematopoietic Stem and Progenitor Cells
Interesting news on CRSP clinical trial at ASH meeting coming up with oral presentation on 12/6. This abstract was selected for the plenary session and the abstract is now public. The researchers modified patient stem cells to turn on fetal hemoglobin which is resistant to effects of thalassemia and Sickle cell disease. Normally fetal hemoglobin is turned off after birth. Fetal hemoglobin was reactivated in all seven patients. So far it looks like they may have cured Sickle cell disease in two patient with no transfusions or admissions for vast-occlusive crisis. Time and more patients will tell but this is very exciting. Similar results in thalassemia patients.
Paper: Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9–Modified CD34+ Hematopoietic Stem and Progenitor Cells