News

Supporters Set to Observe World Sickle Cell Day on June 19

From sporting the color red to “shining a light” on sickle cell, supporters are set to mark World Sickle Cell Day June 19 to call attention to the inherited blood disorder that affects millions of people globally and about 100,000 in the U.S. In addition to raising awareness, the event…

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…

UCSF’s Stad Center Will Cater to Pediatric Pain Management

The UCSF Benioff Children’s Hospitals in California has announced the addition of the Stad Center for Pediatric Pain, Palliative & Integrative Medicine, which is tailored for treating pain in children with disorders such as sickle cell disease. Harboring one of the most innovative and comprehensive programs of its kind in…

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…