Technology ID
TAB-3358
Potential Treatment for sickle-cell disease and thalassemia
E-Numbers
E-200-2018-0
Lead Inventor
Tisdale, John (NHLBI)
Co-Inventors
Gudmundsdottir, Bjorg (NHLBI)
Tumburu, Laxminath (NHLBI)
Applications
Therapeutics
Development Stages
Pre-Clinical (in vitro)
Lead IC
NHLBI
ICs
NHLBI
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.
Commercial Applications
- Designing lentiviral vectors to genetically target RIOK3
- Gene editing using endonucleases such as CRISPR/Cas9
- Developing orally administered RIOK3 specific kinase inhibitor drugs
Competitive Advantages
- A novel and cost-effective treatment strategy in beta-globinopathies
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