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December 3, 2024 News by Marisa Wexler, MS

Trial of gene-editing therapy into SCD’s cause opens in California

Two centers in California are enrolling nine people with severe sickle cell disease (SCD) for a clinical trial of a new gene editing therapy called CRISPR_SCD001. The Phase 1/2 trial (NCT04774536) is open to adolescents and adults, ages 12 through 35, who in the two years before enrollment…

July 27, 2021 News by Marta Figueiredo, PhD

UCSF Awarded Nearly $8.4M for CRISPR_SCD001 Gene Editing Trial

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…

April 1, 2021 News by Marta Figueiredo, PhD

1st CRISPR Gene Editing Trial Slated to Open in Severe SCD Patients

The U.S. Food and Drug Administration approved the start of a first clinical trial of CRISPR_SCD001, the first non-viral and CRISPR/Cas9-based gene editing therapy for sickle cell disease (SCD). Both the therapy and the upcoming Phase 1/2 trial — planned to start this summer — are the result of a…

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