[Federal Register Volume 81, Number 203 (Thursday, October 20, 2016)]
[Notices]
[Pages 72595-72596]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-25343]


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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 invention listed below is owned by an agency of the U.S. 
Government and is available for licensing in the U.S. 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.

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

[[Page 72596]]

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be required to receive any unpublished information.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: Technology description follows.

Capsid-Free AAV Vectors for Gene Delivery and Their Use for Gene 
Therapy

    Description of Technology: The invention concerns novel capsid-free 
AAV vectors that can be used for gene delivery and gene therapy 
applications. The invention provides for a linear nucleic acid molecule 
comprising in this order: A first adeno-associated virus (AAV) inverted 
terminal repeat (ITR), a nucleotide sequence of interest, and a second 
AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid 
protein coding sequences. The said nucleic acid molecule can be applied 
to a host at repetition without eliciting an immune response. Methods 
of producing and purifying this nucleic acid molecule, as well as its 
use for gene transfer and gene therapy are also described.
    Potential Commercial Applications: The commercial applications of 
the technology relate to the field of gene therapy. It may offer 
significant advantages compared to existing methods of gene delivery 
and gene therapy.
    Competitive Advantages:
     The AAV vectors described in the invention devoid the AAV 
capsid proteins and thus are not exposed to the adverse effects caused 
by immunogenicity.
     In contrast to the use of plasmid DNA for gene delivery, 
the AAV DNA of the invention seems to confer greater stability in cell 
nuclei, allowing prolonged expression compared to plasmid DNA.
     The vector DNA of the invention is not limited in size to 
the packageable size genome.
     The production of the AAV DNA vector is economical, simple 
and provides high yields.
    Development Stage: Early-stage; In vitro data available
    Inventors: Drs. Luis Garcia, Cyriaque Beley, and Thomas Voit 
(INSERM Paris); Drs. Robert M. Kotin and Lina Li (NHLBI).
    Publication: Li L, Dimitriadis EK, Yang Y, Li J, Yuan Z, Qiao C, 
Beley C, Smith RH, Garcia L, Kotin RM. Production and characterization 
of novel recombinant adeno-associated virus replicative-form genomes: A 
eukaryotic source of DNA for gene transfer. PLoS One. 2013 Aug 
1;8(8):e69879. doi: 10.1371/journal.pone.0069879.
    Intellectual Property: NIH Reference No. E-241-2010/0--US Patent 
Application No. 14/004,379 (Publication No. 2014-0107186 A), and its 
foreign counterparts in Europe (11 157986.8; 12 708035.6), Canada 
(2,829,518), Australia (2012228376), Brazil (BR 1 1 2013 023185 8), 
China (201280022523.5), Israel (228328), India (8000/DELNP/2013), Japan 
(2013-557138), and South Korea (10-2013-7026982).
    Licensing Contact: Uri Reichman, Ph.D., M.BA.; Phone: 301-435-4616; 
Email: [email protected].

    Dated: October 13, 2016.
Uri Reichman,
Senior Advisor for Licensing, Office of Technology Transfer and 
Development, National Heart, Lung, and Blood Institute.
[FR Doc. 2016-25343 Filed 10-19-16; 8:45 am]
 BILLING CODE 4140-01-P