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…
News
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…
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…
Treatment with hydroxyurea at the time of conception may be safe, but its use by women with sickle cell disease (SCD) during pregnancy was linked to twofold higher risks of both miscarriage and stillbirth, a U.S. study reported. Pregnant women taking hydroxyurea — used to reduce the frequency…
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…
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…
ARU-1801, Aruvant Sciences’ experimental gene therapy for sickle cell disease (SCD), led to the production of fetal hemoglobin in red blood cells and significantly lowered the number of vaso-occlusive crises (VOCs) in a group of five people with severe SCD, according to data from the ongoing Phase…
CRISPR Therapeutics and Vertex have launched two Phase 3 trials to assess the safety and effectiveness of CTX001, an experimental gene-editing cell therapy, one in children with sickle cell disease (SCD) and another for those with transfusion-dependent beta thalassemia (TDT). Both Phase 3 studies will enroll up to…
Hemex Health’s portable point-of-care device, Gazelle, is being enhanced to more precisely monitor fetal hemoglobin levels. According to Hemex, this could be useful for monitoring people with sickle cell disease (SCD) who are being treated with hydroxyurea. The company announced the software upgrade on May 8…
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…
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