23 April 2021 (Friday) - ASH Update

The nice people at he American Society of Hematology sent their update today – an update on red blood cell and iron disorders:

First CRISPR Trial for Patients with Severe Sickle Cell Disease to Begin University of California scientists have received FDA approval to launch an early-phase trial of the first CRISPR/CAS9-based gene therapy for sickle cell disease this summer.

Transplant Improves Health-Related Quality of Life for Patients With SCD and Transfusion-Dependent Thalassemia A systematic review published in Blood Advances evaluated recent longitudinal and cross-sectional data for evidence of the effects of both transplant and gene therapy or gene-editing on health-related QoL outcomes in patients with TDT or SCD.

Scientists Say LentiGlobin Not Linked to Cancer Preliminary data linking the gene therapy to development of acute myeloid leukemia and myelodysplastic syndromes have not held up.