[Federal Register Volume 84, Number 26 (Thursday, February 7, 2019)]
[Notices]
[Page 2540]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-01496]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

National Institutes of Health


Government-Owned Inventions; Availability for Licensing

AGENCY: National Institutes of Health, HHS.

ACTION: Notice.

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SUMMARY: The inventions listed below are owned by an agency of the U.S. 
Government and are available for licensing in the U.S. to achieve 
expeditious commercialization of results of federally-funded research 
and development.

FOR FURTHER INFORMATION CONTACT: Licensing information may be obtained 
by emailing the indicated licensing contact: Michael Shmilovich; 
National Heart, Lung, and Blood, Office of Technology Transfer and 
Development Office of Technology Transfer, 31 Center Drive Room 4A29, 
MSC2479, Bethesda, MD 20892-2479; telephone: 301-402-5579. A signed 
Confidential Disclosure Agreement may be required to receive any 
unpublished information.

SUPPLEMENTARY INFORMATION: Technology description follows.

Sickle Cell Anemia Treatment Through RIOK3 Inhibition

    Beta-globinopathy is a common inherited single-gene disorder of 
beta-globin synthesis that results in an abnormal structure of one 
globin chain of the hemoglobin molecule. Common hemoglobinopathies 
include sickle-cell disease and beta-thalassemia. The efficacy of bone 
marrow transplantation is limited due to high cost and the requirement 
for HLA-matched donors. Increasing fetal hemoglobin expression above a 
certain threshold is potentially curative in the beta-globinopathies. 
The inventors identified Rio-Kinase 3 (RIOK3) as a key negative 
regulator of fetal hemoglobin expression in primary human erythroid 
progenitor cells. Their work shows that lentiviral mediated shRNA 
knockdown of RIOK3 in primary human erythroid progenitor cells 
increased fetal hemoglobin expression above 55% of total b-like globin 
expression, thus, RIOK3 is a promising novel therapeutic target to 
increase fetal hemoglobin expression.
    Potential Commercial Applications: Inhibition of RIOK3 through 
genetic manipulation or by using orally administered kinase inhibitors 
will be a novel and cost-effective treatment strategy in beta-
globinopathies.
    Development Stage: The potential clinical use of this novel 
invention will depend on collaborating with interested companies for 
efficiently inhibiting RIOK3 through (1) designing lentiviral vectors 
encoding shRNA to RIOK3, (2) gene editing using endonucleases such as 
CRISPR/Cas9 and (3) by developing orally administered RIOK3 specific 
kinase inhibitor drugs.
    Inventors: Bjorg Gudmundsdottir, Laxminath Tumburu, John Tisdale 
(all of NHLBI).
    Intellectual Property: HHS Reference No. E-200-2018; U.S. 
Provisional Patent Application 62/756,497 filed November 6, 2018.
    Licensing Contact: Michael Shmilovich, Esq., CLP; 301-435-5019; 
[email protected].

    Dated: December 26, 2018.
Michael A. Shmilovich,
Senior Licensing and Patenting Manager, National Heart, Lung, and Blood 
Institute, Office of Technology Transfer and Development.
[FR Doc. 2019-01496 Filed 2-6-19; 8:45 am]
 BILLING CODE 4140-01-P