News

Global Blood Therapeutics (GBT) has begun a Phase 2/3 trial to study the safety, tolerability, effectiveness, and pharmacological properties of its experimental oral therapy GBT601 in people with sickle cell disease (SCD). Initial data from the study (NCT05431088) are expected before the end of the year, according…

Precision BioSciences and Novartis are teaming up to develop and commercialize a custom gene therapy as a potential treatment approach for certain inherited blood disorders, including beta thalassemia and sickle cell disease (SCD). Under their new exclusive license and collaboration agreement, the companies will combine Precision’s proprietary ARCUS genome…

A comprehensive integrated sickle cell disease (SCD) clinic in southern California — funded by a $500,000 state grant — has been established to support the “highly affected” SCD patient population in San Bernardino County, according to a press release. The community clinic is housed at Loma Linda University…

Aflac, a U.S. supplemental health insurance company, has given $100,000 to Children’s National Hospital in Washington, D.C., to support research into sickle cell disease (SCD) and its treatments. The donation was made to honor World Sickle Cell Awareness Day, observed each June 19. “We are honored for Aflac’s partnership to…

To mark World Sickle Cell Day, Marja Hurley, a University of Connecticut (UConn) physician-researcher, has been awarded nearly $3 million in research funding to investigate bone loss in people with sickle cell disease (SCD). A four-year $2.21 million endowment comes from the National Institutes of Health (NIH) to Hurley…

Exagamglogene autotemcel (exa-cel), an experimental gene-editing cell therapy, increased fetal hemoglobin levels and prevented vaso-occlusive crises (VOCs) in 31 people with severe sickle cell disease (SCD), according to new data from the CLIMB-SCD-121 trial. Vertex Pharmaceuticals and CRISPR Therapeutics, which are jointly developing the therapy, recently launched…

Novartis is partnering with the American Society of Hematology (ASH) to roll out an app in six sub-Saharan African countries to track babies who receive a diagnosis of sickle cell disease (SCD) through newborn screening. The app is already in use in Ghana, where it works to collect…

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…

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…

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…