A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia

  • Post author:
  • Post category:

Sangamo, Inc. is testing genetically engineered blood stem cells for the treatment of beta-thalassemia, a severe form of anemia caused by mutations in the hemoglobin gene. This genetic disorder requires…

Continue ReadingA Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia

Development of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions

  • Post author:
  • Post category:

Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer…

Continue ReadingDevelopment of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions

Development of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions

  • Post author:
  • Post category:

Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer…

Continue ReadingDevelopment of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions

A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia

  • Post author:
  • Post category:

Therapeutic Candidate or Device ST-400 is a gene-edited cell therapy candidate for patients with transfusion-dependent beta-thalassemia Indication Transfusion-dependent beta-thalassemia Therapeutic Mechanism ST-400 is intended to disrupt BCL11A erythroid enhancer in…

Continue ReadingA Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia