[Federal Register Volume 82, Number 1 (Tuesday, January 3, 2017)]
[Notices]
[Pages 132-133]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-31834]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

National Institutes of Health


Prospective Grant of Exclusive License: Development, Manufacture 
and Commercialization of Gene Therapy Products for Human Gene Therapy 
Use To Treat and/or Prevent Methylmalonic Acidemia (MMA)

AGENCY: National Institutes of Health (NIH).

ACTION: Notice.

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SUMMARY: The National Human Genome Research Institute (NHGRI), an 
institute of the National Institutes of Health, Department of Health 
and Human Services, is contemplating the grant of an exclusive 
commercialization patent license to practice the inventions embodied in 
the Patent Applications listed in the Supplementary Information section 
of this notice License to Selecta Biosciences (``Selecta'') located in 
Watertown, Massachusetts.

[[Page 133]]


DATES: Only written comments and/or applications for a license which 
are received by the NCI Technology Transfer Center on or before January 
18, 2017 will be considered.

ADDRESSES: Requests for copies of the patent application, inquiries, 
comments, and other materials relating to the contemplated exclusive 
license should be directed to: Eggerton Campbell Ph.D., Licensing and 
Patenting Manager, Technology Transfer Office (TTO) National Human 
Genome Research Institute, National Institutes of Health, 5635 Fishers 
Lane, Suite 3058, MSC 9307, Bethesda, MD 20892-9307. Telephone: 301-
402-1648. Fax: 301-402-9722. email: [email protected].

SUPPLEMENTARY INFORMATION: 

Intellectual Property

1. US Provisional Patent Application No.: 61/792,081
    HHS Ref. No.: E-243-2012/0-US-01
2. PCT Patent Application No.: PCT/2014/028045
    HHS Ref. No.: E-243-2012/0-PCT-02
3. EP Patent Application 14729502.6
    HHS Ref. No.: E-243-2012/0-EP-03
4. US Patent Application No.: 14/773,885
    HHS Ref. No.: E-243-2012/0-US-04
5. US Patent Application No.: 15/070,787
    HHS Ref. No.: E-243-2012/1-US-01

and all continuing applications and foreign counterparts. The patent 
rights in these inventions have been assigned to the Government of the 
United States of America.
    The prospective exclusive license territory may be worldwide and 
the field of use may be limited to the use of Licensed Patent Rights 
for the following:

Development, manufacture and commercialization of gene therapy 
products for human gene therapy use to treat and/or prevent 
Methylmalonic Acidemia (MMA) comprised of the following: all of or 
fragments of the synthetic methylmalonyl-CoA mutase (MUT) human 
polynucleotide (synMUT) and/or recombinant synMUT constructs, in 
combination with the following:
    the Anc80 vector or vectors derived from the Anc80 vectors, 
wherein the derived Anc80 vectors have capsid sequences possessing 
90% or greater sequence identity to the Anc80 capsid sequences.

    For purposes of clarity, the above gene therapy products may be 
combined with Selecta's synthetic vaccine particles (SVPTM) 
technology encapsulating an immunomodulator.
    The subject technology discloses a synthetic codon-optimized human 
methylmalonyl-CoA mutase (MUT) cDNA gene (co-MUT) encoding human MUT 
protein, co-MUT constructs and uses thereof for treatment of MMA 
disorders. Such uses, may include the administration of 
immunomodulator(s) in order to maximize the advantage of the gene 
therapy, with fewer side effects. MMA is an autosomal recessive 
disorder caused by defects in the mitochondria-localized enzyme 
methylmalonyl-CoA mutase (MUT). MUT deficiency, the most common cause 
of MMA, is characterized by the accumulation of methylmalonic acid. MMA 
can lead to metabolic instability, seizures, strokes, and kidney 
failure, and can be lethal even when patients are being properly 
managed. If successfully developed, this invention would be a first of 
its kind therapy for MMA, by administering the disclosed nucleic acid, 
vector, or recombinant virus to a subject, optionally with an 
immunomodulator.
    This notice is made in accordance with 35 U.S.C. 209 and 37 CFR 
part 404. The prospective Exclusive Patent License will be royalty 
bearing and may be granted unless within fifteen (15) days from the 
date of this published notice, the National Human Genome Research 
Institute receives written evidence and argument that establishes that 
the grant of the license would not be consistent with the requirements 
of 35 U.S.C. 209 and 37 CFR part 404.
    Complete applications for a license in the prospective field of use 
that are timely filed in response to this notice will be treated as 
objections to the grant of the contemplated Exclusive Patent License. 
Comments and objections submitted to this notice will not be made 
available for public inspection and, to the extent permitted by law, 
will not be released under the Freedom of Information Act, 5 U.S.C. 
552.

    Dated: December 27, 2016.
Claire T. Driscoll,
Director, NHGRI Technology Transfer Office.
[FR Doc. 2016-31834 Filed 12-30-16; 8:45 am]
 BILLING CODE 4140-01-P