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UCSF Awarded Nearly $8.4M for CRISPR_SCD001 Gene Editing Trial

Research Update: 🔬🧫 The University of California, San Francisco (UCSF) has been awarded $8.39 million to fund a Phase 1/2 clinical trial of CRISPR_SCD001, the first non-viral and CRISPR-Cas9-based gene editing therapy for sickle cell disease (SCD). Researchers will use the gene-editing technology to attempt to correct a genetic mutation in the blood stem cells of patients with severe SCD.

The grant comes from the California Institute for Regenerative Medicine or CIRM, which was created by the state’s residents to accelerate the process of getting stem cell treatments to patients. CIRM previously funded CRISPR_SCD001’s preclinical development — including studies to secure the needed approval to conduct the first-in-human trial in the U.S. The National Heart, Lung, and Blood Institute (NHLBI) will conduct the trial along with UCSF.

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