Therapeutic

METHODS FOR PROGRAMMING DIFFERENTIATED CELLS INTO HEMATOPOIETIC STEM CELLS

This technology defines a specific combination of transcription factors that can program hemogenesis in fibroblasts. The blood system is continuously replenished from a rare population of hematopoietic stem cells (HSCs) that balance self-renewal and differentiation. Transplantation of HSCs is utilized cell therapy for a range of genetic and acquired disorders. However, allogeneic transplantation depends on genetic matching to avoid graft vs. host disease as well as graft rejection, and even matched grafts are still associated with high risk. Furthermore, there are also limited quantities of available material especially in cord blood transplants and for various ethnic groups. Therefore, alternative sources of patient-specific transplantable HSCs are needed. This technology could provide an unlimited patient-specific source for cell replacement and genetic correction therapies.

Current Development Status

  • Research and development

Applications

  • Directly programmed HSCs from somatic cells could provide an unlimited patient-specific source for cell replacement and genetic correction therapies

Advantages

  • This technology provides a platform for the generation of patient-specific therapeutics and blood products.
  • Allogeneic transplantation depends on genetic matching to avoid graft vs. host disease as well as graft rejection, and even matched grafts are still associated with high risk

Publications

  • Not available

Patent Status

  • International Application PCT/US2013/023803 filed January 30, 2013
  • Status: Published. International Publication No. WO 2013/116307
  • US Application 14/374,639 filed July 25, 2014
  • Status: Published. US Publication No. US-2015-0004145-A1

Contact

Felipe Araujo, PhD
Director, Blue Mountain Technologies
Mount Sinai Innovation Partners | Icahn School of Medicine at Mount Sinai
Phone: (212) 731-7039