The Technology Transfer Office (TTO) facilitates interactions between NHGRI's research laboratories and other research entities, including universities, non-profit organizations and companies, for the benefit of public health. TTO carries out its mission by assisting in the transfer of NHGRI-developed technologies to the private sector for further development and commercialization and by managing formal relationships with pharmaceutical and life sciences companies through the use of various legal instruments. The TTO also ensures the speedy and efficient exchange of research resources between NHGRI and outside scientific groups and assures compliance with relevant laws and policies. Nine (9) new employee invention reports (EIRs) were evaluated and processed in FY2016 by the TTO. All of these EIRs were for non-patented biological materials, with three still being evaluated. Eleven (11) new patent applications were filed. The titles of the eleven (11) patent applications filed are: Hyposialylation Disorders, Gene Therapy for Cobalamin C Deficiency, Gene Therapy for Combined Methylmalonic Acidemia/Aciduria and Hyperhomocysteinemia/Homocystinuria, Cobalamin C Type, and Deficiency of MMACHC (U.S. provisional, followed by an international PCT application), Sialylation-Increasing Therapies for Diseases Associated with Oxidative Stress, Synthetic Methylmalonyl-COA Mutase Transgene for the Treatment of Mut Class Methylmalonic Acidemia (MMA), Methods for Treating Progeroid Laminopathies Using Oligonucleotide Analogues Targeting Human LMNA, Viral Gene Therapy as Treatment for Cholesterol Storage Disease or Disorder, Tagged Form of Mut Enzyme, Genetic Constructs Incorporating It, and Its Use in Gene Therapy, Oligonucleotide Analogues Targeting Human LMNA, and Cellular Arrays and Methods of Detecting and Using Genetic Disorder Markers. In FY2016, six (6) patents were issued in the NHGRIs portfolio. The titles of the issued patents are: 1) GRIN2A Mutations and Use Thereof for the Diagnosis of Melanoma (U.S. Patent 9,157,125; NHGRI lead inventor Yardena Samuels), 2) N-Acetyl Mannosamine as a Therapeutic Agent (Canadian Patent 2,680,842; NHGRI lead inventor Marjan Huizing), 3) Cellular Arrays and Methods of Detecting and Using Genetic Disorder Markers (U.S. Patent 9,284,610; NHGRI lead inventor Olli Kallioniemi), 4) Hyposialylation Disorders (U.S. Patent 9,341,619; NHGRI lead inventor Marjan Huizing), 5) Methods for Treating Progeroid Laminopathies Using Oligonucleotide Analogues Targeting Human LMNA (U.S. Patent 9,326,992; NHGRI lead inventor Mike Erdos), and 6) Farnesyltransferase Inhibitors for Treatment of Laminopathies, Cellular Aging and Atherosclerosis (European Patent EP1853265; NHGRI lead inventor Francis S. Collins). As of August 2016, NHGRI had forty two (42) active patent families, forty two (42) issued U.S. patents, one hundred and thirteen (113) issued foreign patents, and fifty (50) pending patent applications (U.S. and foreign). In FY2016 NHGRI negotiated and executed three (3) new conditional gift fund agreements with the following organizations: Mucolipidosis Type IV (ML4) Foundation, National Tay-Sachs & Allied Diseases Association, Inc., and United Mitochondrial Disease Foundation Lizzie Piro Research Fund. Our Office handled the electronic submission of several K99/R00 NIH applications on behalf of postdoctoral fellows and we assisted with the submission of several non-NIH grant applications to organizations such as the Life Sciences Research Foundation. New FY2016 executed conditional gift fund agreements and submitted non-NIH research grants focused on the following human disease research topics: Mucolipidosis Type IV (ML4), Tay-Sachs Disease, and Cerebral Florate Deficiency. In FY2016 we negotiated and executed three (3) Cooperative Research and Development Agreements (CRADAs) with the following companies: 1) Escala Therapeutics, Inc.; 2) Moderna Therapeutics, Inc.; and 3) Selecta Biosciences, Inc./Massachusetts Ear and Eye Infirmary. We also negotiated four (4) amendments for two existing CRADAs. In FY2016 we executed eight (8) new license agreements with the following companies and/or organizations: 1) Merck Sharpe & Dohme Corp. (L-083-2016/0) - Fibroblast Cell Lines Homozygous for Glucocerebrosidase (GBA1) Mutation N370S (E-059-2014/0) and Fibroblast Cell Lines from a Patient with Gaucher Genotype L444P/RecNci1 (E-062-2016/0); 2) Ionis Pharmaceuticals, Inc. (L-201-2015/1) - Mouse Model of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Vascular Abnormalities (E-243-2011/0); 3) Applied Biological Materials, Inc. (L-131-2016/0) - hELG-CFP RPE Cell Line for Detection of Genotoxin (E-108-2008/0); 4) Emergent Product Development Gaithersburg Inc. (L-151-2016/0) - Human Cell Lines With Mannosyl Olygosaccharide Glycosidase (MOGS) Defect (E-129-2016/0); 5) Moderna Therapeutics, Inc. (L-149-2016/0) - Novel Mouse Models of Methylmalonic Acidemia (MMA): C57Bl6/Sv129 Mut-/- (Full Knock-Out) and (C57Bl6/Sv129) FvBN Mut -/- (Full Knock-Out) (E-139-2016/0), Novel Mouse Model of Mut- Methylmalonic Acidemia (MMA) Mut-/- Tg CBAMutG715V: Mut Partial-Deficiency (E-140-2016/0), and Novel Mouse Model of Methylmalonic Acidemia (MMA) Mut-/- Tg INS-Mck-Mut (E-141-2016/0); 6) Galyatech LLC (L-186-2016/0) - Adeno-Associated Viral Gene Therapy as a New Treatment for NiemannPick Disease Type C (E-185-2014/0); 7) Escala Therapeutics, Inc. (L-122-2010/2) - Provision Of The Neutral Sugar N-Acetyl Mannosamine (ManNAc) For Increased Intracellular Production Of Sialic Acids (E-217-2007/0); and 8) Ionis Pharamceuticals, Inc. (L-201-2015/2) - Mouse Model of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Vascular Abnormalities (E-243-2011/0). In total, NHGRI currently has forty-five (45) active licenses. In addition, in FY 2016 we processed ninety-three (93) Material Transfer Agreements (MTAs) and Information Transfer Agreements (ITAs). The vast majority of these agreements covered materials and data provided by NHGRI to outside entities and 62% of the MTAs and ITAs involved the transfer of human materials or data.

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Human Genome Research
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