Margarida Maia, PhD, science writer —

Vertex Pharmaceuticals and CRISPR Therapeutics are planning a November launch for a biologics license application (BLA) for their gene-editing therapy exagamglogene autotemcel — known as exa-cel — seeking its approval for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The U.S. Food and Drug Administration (FDA)…

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…

The total lifetime medical costs of living with sickle cell disease (SCD) total about $1.6 million for women and $1.7 million for men younger than 65 who hold private health insurance in the U.S., a study found. Researchers concluded that some of these expenses — which are roughly four…

A renowned stem cell biologist has been honored with the 2022 Canada Gairdner International Award for his work that set the grounds for a new approach to treating sickle cell disease (SCD) and beta-thalassemia. Stuart H. Orkin, MD, is the David G. Nathan Distinguished Professor of Pediatrics at…

An enzyme known to play a key role in blood vessel health is present at low levels in the cells lining the lung blood vessels of patients with sickle cell disease (SCD), which may affect how these cells stick to their supporting matrix, a study has found. Lack of…

A researcher will use a four-year, $1.63 million grant from the National Institutes of Health (NIH) to help set up a quality control system for CRISPR-based therapies aimed at certain genetic disorders, starting with sickle cell disease (SCD). The grant was awarded to Kiana Aran, PhD, an associate…

Adakveo (crizanlizumab), by Novartis, is now available on the U.K.’s National Health Service (NHS) for sickle cell disease (SCD) patients 16 and older. The new treatment, which is delivered by an into-the-vein infusion, can be used alone or as an add-on to hydroxyurea to help reduce the frequency of…

Biopharmaceutical company Brooklyn ImmunoTherapeutics has launched a new center focused on research and development (R&D) of gene editing and cell-based therapies to treat cancer and rare blood disorders, including sickle cell disease. “Our new R&D facility will provide us with the ability to advance our research and development…