[Federal Register Volume 83, Number 61 (Thursday, March 29, 2018)]
[Notices]
[Pages 13494-13495]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-06364]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

National Institutes of Health


Government-Owned Invention; 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.

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Disclosure Agreement may be required to receive any unpublished 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

SUPPLEMENTARY INFORMATION: The following inventions are available for

[[Page 13495]]

licensing in accordance with 35 U.S.C. 209 and 37 CFR part 404 to 
achieve expeditious commercialization of results of federally-funded 
research and development. Technology description follows.

Lentiviral Protein Delivery System for RNA-Guided Genome Editing

    Description of Technology: This invention provides an HIV-1-based 
lentiviral vector system for gene correction strategies involving a 
homologous recombination with a variation of the CRISPR/Cas9 system. 
Such systems are being explored as potential therapies for certain 
hereditary diseases. This system comprises (a) a lentivirus vector 
particle comprising a lentiviral genome which encodes at least one 
guide RNA sequence that is complementary to a first DNA sequence in a 
host cell genome, (b) a Cas9 protein, and optionally (c) a donor 
nucleic acid molecule comprising a second DNA sequence. In addition, 
the invention provides a host cell comprising the foregoing system, as 
well as a method of altering a DNA sequence in a host cell comprising 
contacting a host cell with the foregoing system. Alternatively, the 
invention also provides a fusion protein comprising a Cas9 protein and 
a cyclophilin A (CypA) protein, wherein the fusion protein binds to the 
lentivirus vector particle, as well as a lentiviral vector particle 
comprising such a fusion protein. Other such lentivirus-based vectors 
encode a guide RNA, which contains a specific sequence that recognizes 
a target gene, and a Cas9 endonuclease, which cuts at the specific 
site. However, such systems present some problems due to constitutive 
expression of Cas9 endonuclease in lentiviral vector-transduced cells 
and the large size of the Cas9 gene. The variation of this invention 
delivers the Cas9 endonuclease directly, instead of the gene encoding 
the protein.
    Potential Commercial Applications: Clinical trials for hereditary 
diseases such as sickle-cell disease and beta-thalassemia are good 
market opportunities. Gene correction using the disclosed lentiviral 
vector system are being tested with respect to the beta-globin gene and 
the BCL11A gene to treat sickle-cell disease and will be used for 
induced pluripotent stem cell (iPS) generation.
    Development Stage: Early-stage. In vitro data in cell-line models 
available.
    Inventors: Naoya Uchida, Juan J. Haro Mora and John F. Tisdale 
(NHLBI).
    Intellectual Property: US Application No. 62/236,223, filed October 
2, 2015 and PCT/US2016/054759, filed September 30, 2016, (NIH Reference 
No. E-165-2015/0,1).
    Publications: Lentiviral protein delivery system for RNA-guided 
genome editing, PCT Publication No. WO/2017/059241, published April 6, 
2017.
    Licensing Contact: Cristina Thalhammer-Reyero, Ph.D., M.B.A.; 301-
435-4507; [email protected].
    Collaborative Research Opportunity: The National Heart, Lung and 
Blood Institute is seeking statements of capability or interest from 
parties interested in collaborative research to further develop, 
evaluate or commercialize this technology. For collaboration 
opportunities, please contact Denise Crooks at [email protected].

    Dated: March 22, 2018.
Cristina Thalhammer-Reyero,
Senior Licensing and Patenting Manager, Office of Technology Transfer 
and Development, National Heart, Lung, and Blood Institute.
[FR Doc. 2018-06364 Filed 3-28-18; 8:45 am]
 BILLING CODE 4140-01-P