Technology ID
TAB-4736

Treatment of the beta-globinopathies through inhibition of RIOK3 activity

E-Numbers
E-127-2021-2
Lead Inventor
Tisdale, John (NHLBI)
Co-Inventors
Gudmundsdottir, Bjorg (NHLBI)
Tumburu, Laxminath (NHLBI)
Applications
Therapeutics
Therapeutic Areas
Immunology
Development Stages
Pre-Clinical (in vitro)
Development Status
Pre-Clinical (in vivo - animal)
Research Products
Plasmids/Vectors
ICs
NHLBI

Disorders of adult beta-globin synthesis, which include sickle cell disease (SCD) and beta-thalassemia, are the most common monogenic disorders in the world. While the curative potential of bone marrow transplantation has been demonstrated, this approach is limited to a small fraction of affected patients due to the requirement for an HLA-matched donor, the highly specialized approach that requires critical infrastructure, and the high cost.

Scientists at the National Heart, Lung, and Blood Institute (NHLBI), have developed lentiviral vectors encoding shRNA to Rio-Kinase 3 (RIOK3) that decrease expression of RIOK3, leading to more robust fetal hemoglobin expression.

Commercial Applications
  • Treatment of beta-globinopathies such as Sickle Cell Disease.
Competitive Advantages
  • Increase fetal hemoglobin expression.
  • Novel and more cost-effective treatment.
Licensing Contact:
Kolesnitchenko, Vincent
vk5q@nih.gov