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PRESENCE Study
PRESENCE is a national study testing a digital CBT program to help 16 to 30-year-olds with sickle cell manage chronic pain, stress, and the emotional toll it can take.
16 to 30 years old
Survey
Yes
All types of SCD
United States
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
The goal of this study, conducted by UCSF and UCLA, is to determine if a potential gene therapy, called CRISPR_SCD001, is safe for and improves the health of individuals with SCD.
12-35 years
Clinical
Yes
1/2
SCD type SS
United States of America
01 Jul 2026
The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exa-cel) for patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT). This is the first gene therapy approved for patients aged 2 years and older with SCD.
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20 Apr 2026
Phase 3 results showed a 27% reduction in VOC events, delayed time to first VOC, and improvements in hemoglobin.
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01 Apr 2026
Results of base editing of the HBG1/HBG2 promoters to increase fetal hemoglobin in sickle cell disease.
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