The company has been previously granted orphan drug designation and rare pediatric disease designations for the therapy for the treatment of sickle cell disease by the U.S. Food and Drug Administration.

ARU-1801 is a one-time potentially curative investigational gene therapy for individuals living with sickle cell disease. The therapy incorporates a patented modified gamma-globin into autologous stem cells, with the goal of restoring red blood cell function through increased fetal hemoglobin. ARU-1801 engraftment is achieved with only reduced intensity conditioning.

The EMA's decision was based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. Preliminary clinical data from the Momentum study, an ongoing phase I/II trial, demonstrated durable reductions in disease burden.

Intellia to advance CRISPR treatment for sickle cell
Intellia Therapeutics has received a grant from the Bill & Melinda Gates Foundation to research in vivo sickle cell disease treatments using its CRISPR/Cas9...

Survey queries informed consent for genome editing clinical trials
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