Blood flow and oxygen extraction fraction — a measure of how well oxygen is used by tissues — in the brain was improved among children with sickle cell disease (SCD) who underwent a hematopoietic stem cell transplant (HSCT), a small study showed. According to researchers, these findings may…
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…
Naproxcinod, an investigational therapy being developed by Fera Pharmaceuticals as a treatment for sickle cell disease (SCD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). Orphan drug status provides regulatory support and financial benefits to experimental treatments for diseases that affect fewer…
Imara has completed patient enrollment for its Phase 2b Ardent clinical trial, which is testing the safety and efficacy of IMR-687 for the treatment of sickle cell disease (SCD). “We are excited to have enrolled [participants] from across the world, including in Africa, making this a truly global…
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