Fetal Hemoglobin Induction for Treating Sickle Cell Disease and Thalassemias


Sickle cell disease and thalassemia are hereditary disorders marked by a disruption in the pathways responsible for carrying oxygen to erythrocytes. It has been shown that mutations involved in the development of fetal to adult hemoglobin contribute to the pathophysiology underlying these blood diseases. As a result, there has been increased focus on treatments that promote the induction of fetal hemoglobin to improve clinical symptoms and ameliorate the severity of the diseases.

Researchers at the National Institute of Diabetes and Digestive and Kidney Diseases have discovered that erythroid-specific expression of IGF2BP1 in adult human erythroid cells causes more than adequate expression of fetal hemoglobin for the treatment of sickle cell anemia and beta thalassemia and have developed a system for increasing fetal hemoglobin by using a lentiviral vector containing erythroid specific promoters with the IGF2BP1 coding region in the absence of the IGF2BP1 3'UTR. Early ex vivo results have shown that the technology increased gamma-globin mRNA to 90% of total levels as well as increased fetal hemoglobin to 80%. This technology could lead to the development of novel fetal hemoglobin induction therapies that reduce the aberrant pathologies associated with these diseases.

Potential Commercial Applications: Competitive Advantages:
  • Ex vivo and in vivo therapeutics for treatment of sickle-cell anemia and beta thalassemias.
  • Potential use in combination with other transduction methods for unique therapeutic strategies.
 
  • Reduced production of symptom-associated adult hemoglobin.
  • Novel target of fetal hemoglobin induction.
  • Improved safety and reduced toxicity as a result of erythroid-specific expression.


Development Stage:
Ex vivo data available

Related Invention(s):
E-456-2013-2


Inventors:

Jeffery Miller (NIDDK)  ➽ more inventions...

Yuanwei Lee (NIDDK)  ➽ more inventions...

Colleen Byrnes (NIDDK)  ➽ more inventions...

Laxminath Tumburu (NIDDK)  ➽ more inventions...


Intellectual Property:
US Application No. 62/250,815
PCT Application No. PCT/US2016/060084

Collaboration Opportunity:

The NIDDK Technology Advancement Office is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize Sickle Cell Disease and Thalassemias Treatment. For collaboration opportunities, please contact Agnes Rooke at agnes.rooke@nih.gov.


Licensing Contact:
Carolyn Buller,
Email: carolyn.buller@nih.gov
Phone: 301-443-5605

OTT Reference No: E-038-2016-0
Updated: May 10, 2018