Durable mechanistic effects and a good safety profile were found in patients who took mitapivat for sickle cell disease (SCD). Taking 2 doses of mitapivat daily was found to improve treatment of ...

Scribe Therapeutics Co-Founder, President and CEO Benjamin L. Oakes, PhD A growing landscape for sickle cell disease (SCD) treatments, a growing relationship between the companies, and the potential ...

Here, we describe the first two patients, one with TDT and the other with SCD, who were infused with CTX001 (autologous CRISPR-Cas9–edited CD34+ HSPCs that were genetically edited to reactivate the ...

Conversion of saturated fatty acids to monounsaturated fatty acids by the enzyme stearoyl-Co-A-desaturase (SCD-1) is emerging as a major factor in promoting carcinogenesis including breast cancer. The ...