Researchers at the University of York in the U.K. have been awarded a $3.1 million (£2.3 million) grant to accelerate research supporting stem cell gene therapy to treat sickle cell disease (SCD). The grant was awarded by the nonprofit Bill & Melinda Gates Foundation to track genetic changes in…
Higher doses of FTX-6058, an investigational oral treatment for sickle cell disease (SCD), effectively raised hemoglobin messenger RNA (mRNA) levels above those found at lower doses in healthy volunteers participating in a Phase 1 trial, new data suggests. Increases in hemoglobin mRNA — the molecule cells use as…
IMR-687 (tovinontrine), an experimental oral therapy for sickle cell disease (SCD), reduced the rate of painful vaso-occlusive crisis (VOC), lowered hospitalization due to VOCs, and extended the time to a first VOC in SCD patients, according to new data from an open-label extension of a Phase 2 clinical trial.
Enrollment is underway for a Phase 1/2 trial evaluating the safety and efficacy of EDIT-301, an experimental gene editing cell therapy for people with sickle cell disease (SCD). The trial, called RUBY (NCT04853576), had been cleared for launch by the U.S. Food and Drug Administration earlier this…
Oxbryta (voxelotor), Global Blood Therapeutics’ first-in-class oral therapy for sickle cell disease (SCD), has won the 2021 Prix Galien USA Award for best biotechnology product. The award is among the health industry’s most prestigious honors, and recognizes an outstanding product that has improved patient well-being and impacted human…
The U.S. Food and Drug Administration (FDA) has agreed to consider Global Blood Therapeutics’ (GBT) request to expand the use of Oxbryta (voxelotor) to children as young as 4 with sickle cell disease (SCD). The oral therapy is currently conditionally approved — when given at a daily…
A Promising Innovative Medicine (PIM) designation has been granted to Oxbryta (voxelotor), a treatment to reduce the destruction of red blood cells in people, 12 and older, with sickle cell disease (SCD). The designation, given to promising therapies that are likely to provide major benefit to patients, follows…
Machine learning tools can identify sickle cell disease (SCD) patients at high risk of progressive kidney disease as early as six months in advance, a study shows. The study, “Using machine learning to predict rapid decline of kidney function in sickle cell anemia,” was published in the journal…
A non-viral, CRISPR-based gene-editing tool showed a potential to reverse mutations that cause sickle cell disease (SCD) by successfully editing genes in stem cells within the bone marrow of mice. Data from this proof-of-concept study…
A higher 600 mg daily dose of FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD), was well-tolerated and safe, and improved several measures of red blood cell health after 14 days of treatment, according to…
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