Genetic blood disorders can be caused by mutations in genes that are important for normal blood cell function.

Our scientists developed a method for treating genetic blood disorders by engineering pluripotent stem cells to create cell therapies that can restore the function of the hematopoietic system.

Pluripotent Stem Cell-Derived Therapies for the Treatment of Genetic Blood Disorders is protected by a pending U.S. patent (with additional patents pending in other countries).

Example Applications

  • Replace lost or damaged cells following immune cell depletion treatment
  • Develop a single product that can treat genetic blood diseases with a single administration and independent of specific disease-causing mutations
  • Gene edit somatic cells to avoid the risk of germline transmission
  • Combine with Factor’s Combined mRNA Cell Reprogramming & Gene Editing for streamlined manufacturing
  • Combine with Factor’s Gene-Edited Allogeneic Cell Therapies Technology to create “off-the-shelf” therapies