News

2 GBT Therapies Win FDA Orphan Drug, Rare Pediatric Designations

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to inclacumab and GBT601, two experimental therapies for sickle cell disease (SCD) being developed by Global Blood Therapeutics (GBT). These designations confer benefits to therapy developers that are designed to speed…

2 Proteins That Silence Fetal Hemoglobin May Be Therapy Target

Interfering with two newly identified proteins — NFIA and NFIX — involved both directly and indirectly in silencing fetal hemoglobin in red blood cells, could be a new therapeutic approach for sickle cell disease (SCD), according to researchers. Their hypothesis is that inhibiting the production or activity of these…

Phase 1 Trial Supporting GBT601 as Next-gen Therapy to Oxbryta

Multiple daily doses of GBT601, Global Blood Therapeutics (GBT)’s experimental oral therapy for sickle cell disease (SCD), were generally well tolerated and showed promising pharmacological and efficacy signals in six patients in a Phase 1 trial. These results support further evaluation of GBT601 as the potential successor to GBT’s…

FDA Grants Fast Track Status to SCD Gene-editing Therapy GPH101

Graphite Bio’s GPH101, an investigational gene-editing therapy that aims to correct the genetic mutation that causes sickle cell disease (SCD) and potentially cure the condition, has been awarded fast track designation by the U.S. Food and Drug Administration (FDA). The fast track process is designed to accelerate the…