ARU-1801, an experimental gene therapy for sickle cell disease (SCD), lowered the number of vaso-occlusive crises and led to  blood cells producing fetal hemoglobin in a first group of three treated patients, according to data from an ongoing Phase 1/2 trial. Favorable safety is…
Global Blood Therapeutics (GBT) has been granted exclusive global rights to two small-molecule research programs for sickle cell disease (SCD) as part of a newly announced agreement between GBT and Sanofi. The two early stage programs,…
Graphite Bio has raised $150 million in Series B funding to expand and advance the clinical development of its pipeline of investigational gene-editing therapies — including GPH101, a potentially curative treatment for sickle cell disease (SCD). In addition…
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