Diogo Pinto,  —

Novartis’ investigational therapy for sickle cell disease (SCD), crizanlizumab (SEG101), was seen to reduce the number of patients reporting a pain crisis — including in those with a recent history of multiple crises — a post-hoc analysis of the SUSTAIN Phase 2 study reports. More patients (35.8%)…

The HRI signaling protein was identified as a key negative regulator of hemoglobin production in red blood cells, unraveling a potential new therapeutic target for sickle cell disease (SCD). Using the gene editing tool CRISPR-Cas9, HRI was found to block the production of fetal hemoglobin, which carries iron…

White blood cells were seen to produce molecules that decrease a specific type of hemoglobin — the protein in red blood cells that’s responsible for the transport of oxygen — in patients with sickle cell disease (SCD). These findings may help in the understanding of disease mechanisms, as well as resistance…

Bluebird bio reported that its investigational gene therapy LentiGlobin showed promising results for severe sickle cell disease (SCD) patients in a Phase 1 clinical trial. Data from the ongoing HGB-206 Phase 1 multicenter study (NCT02140554) were revealed in an oral presentation, “Recent progress in gene therapy for severe…

Sancilio Pharmaceuticals is reporting that its investigational oral compound Altemia (SC411, docosahexaenoic acid) showed an ability to safely treat sickle cell disease (SCD) in a Phase 2 study. Data from the Phase 2 SCOT trial (NCT02973360) trial were given at the 2018 American Society Pediatric Hematology and Oncology (ASPHO)…

Global biotechnology company MaxCyte announced the successful correction of a genetic mutation associated with sickle cell disease (SCD) in a patient’s hematopoietic stem cells (HSCs), the type of cells that give rise to blood cells. The mutation was corrected in the hemoglobin gene using MaxCyte’s non-viral cell engineering…