Potential Treatment for sickle-cell disease and thalassemia

The technology addresses treatment options for diseases such as sickle-cell and thalassemia. Traditionally, such beta-globinopathies are treated through bone marrow transplantation. However, this method is limited due to high treatment costs and finding a matched-donor. This relies on increasing fetal hemoglobin to potentially cure the disease. NIH inventors have identified a protein called Rio-Kinase 3 (RIOK3), that inhibits the production of fetal hemoglobin. Their work shows that inhibiting RIOK3 increases the production of fetal hemoglobin. Thus, RIOK3 is a promising novel therapeutic target to increase fetal hemoglobin expression.

Potential Commercial Applications: Competitive Advantages:
  • Designing lentiviral vectors to genetically target RIOK3
  • Gene editing using endonucleases such as CRISPR/Cas9
  • Developing orally administered RIOK3 specific kinase inhibitor drugs
  • A novel and cost-effective treatment strategy in beta-globinopathies


John Tisdale (NHLBI)  ➽ more inventions...

Bjorg Gudmundsdottir (NHLBI)  ➽ more inventions...

Laxminath Tumburu (NHLBI)  ➽ more inventions...

Intellectual Property:
US Application No. 62/756,497
US Application No. 16/676,369
PCT Application No. PCT/US2019/060151

Gudmundsdottir B., et al. PMID: 29898395

Collaboration Opportunity:

The NHLBI is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize use of RIOK3 as therapeutic target in the treatment of beta-globinopathies. For collaboration opportunities, please contact Mr. Michael Shmilovich at michael.shmilovich@nih.gov

Licensing Contact:
Denise Crooks,
Email: crooksd@mail.nih.gov
Phone: 301-435-0103

OTT Reference No: E-200-2018-0
Updated: Jan 17, 2019