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Sickle hemoglobin is produced by a β-point mutation in the globin gene, which is less soluble than normal fetal or adult hemoglobin.
sickle cell disease (SCD) is rarely seen in blacks.
about 10 percent of black Americans have a sickle cell disease gene (that is, they have sickle cellular symptoms).
who have sickle cellular symptoms do not necessarily develop sickle cell disease, but their risk of certain complications, such as urine and blood, does increase.
about 0.3% of blacks carry two genes.
Biopharmaceutical company Aruvant Sciences announced today that the European Medicines Agency (EMA) has granted ARU-1801 priority drug eligibility (PRIME) for the treatment of sickle cell disease.
ARU-1801 is a research gene therapy for sickle cell disease.
Will Chou, chief executive of Aruvant, said: "PRIME highlights the medical need for sickle cell disease.
with PRIME, we were able to work closely with the EMA to develop the ARU-1801, and our aim is to bring this potential treatment to SCD patients in Europe quickly."
based on clinical data from phase I/II clinical trials (MOMENTUM studies), ARU-1801 treats severe sickle cell disease and can reduce the burden of disease in a lasting way.
