[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unpublished information.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