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. Sixteen (16) new employee invention reports (EIRs) were evaluated in FY2017 by the TTO. Eight (8) are waiting for additional experimental results for OTT filing. Eight (8) were filed in the NIH Office of Technology Transfer (OTT). Eight (8) were for biological materials and eight (8) were for technologies that could be patented or at least evaluated for patenting. Five (5) new patent applications were filed. The titles of the five (5) patent applications filed are: N-Acetyl Mannosamine as a Therapeutic Agent, Oligonucleotide Analogues Targeting Human LMNA, Methods of Detecting and Treating GATM-Related Renal Disorders, Novel Codon-Optimized and Tagged Human NPC1 Genes and AAV Vectors for the Treatment of Niemann-Pick Type C1 Deficiency and Related Conditions, Synthetic Methylmalonyl-COA Mutase Transgene for the Treatment of Mut Class Methylmalonic Acidemia (MMA)and Serum Protein Biomarkers That Predict The Response To Liver Directed Therapy In Methymalonic Acidemia (MMA). In FY2017, three (3) patents were issued in the NHGRI portfolio. The titles of the issued patents are: Methods for Treating Progeroid Laminopathies Using Oligonucleotide Analogues Targeting Human LMNA (Japanese Patent 6132849, NHGRI lead inventor Michael Erdos) and Synthetic Methylmalonyl-COA Mutase Transgene for the Treatment of Mut Class Methylmalonic Acidemia (MMA) (U.S. Patent 9,719,080, and European Patent 2968602, NHGRI lead inventor Charles Venditti). As of August 2017, NHGRI had forty (40) active patent families, fifty (50) issued U.S. patents, one hundred twenty (120) issued foreign patents, and seventy-one (71) pending patent applications (U.S. and foreign). In FY2017, NHGRI negotiated and executed two (2) new conditional gift fund agreements with the following organizations: Moebius Syndrome Foundation, and Ding Lei Professional Medical Corporation. 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 American Diabetes Association (granted), Ara Parseghian Medical Research Foundation (granted), National Tay-Sachs and Allied Diseases Association, Audrey Heimler Special Projects, Saint Baldricks Foundation, Fred Hutchinson Cancer Research Center, New York Stem Cell Foundation, and Johns Hopkins Medical Institutions. New FY2017 executed conditional gift fund agreements and submitted non-NIH research grants focused on the following human disease research topics: Diabetes, Niemann Pick Type 2 Disease, Tay-Sachs, Moebius Syndrome, GM1 Gangliosidosis, and Familial Platelet Disorder. In FY2017 we negotiated and executed two (2) Cooperative Research and Development Agreements (CRADAs) with the following companies: 1) LogicBio Therapeutics; and 2) Pfizer, Inc. We also negotiated six (6) amendments for three existing CRADAs. In FY2017 we executed nine (9) new license agreements with the following companies and/or organizations: 1) Ionis Pharmaceuticals, Inc. (L-201-2015/3) - Mouse Model of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Vascular Abnormalities (E-243-2011/0); 2) LogicBio Therapeutics (L-153-2017/0) - Novel mouse model of methylmalonic acidemia (MMA): Mut-/- Tg INS-Mck-Mut (E-141-2016/0); 3) Ikonisys, Inc. (L-053-2007/1) - Methods for Diagnosis of Cervical Cancer (E-012-1997/0); 4) Selecta Biosciences, Inc. (L-216-2017/0) - A Synthetic Methylmalonyl-CoA Mutase Transgene for the Treatment of Mut Class Methylmalonic Acidemia (MMA) (E-243-2012/0); 5) SK Biopharmaceuticals Co., Ltd. (L-231-2017/0) - Fibroblast Cell Lines Homozygous for Glucocerebrosidase (GBA1) Mutation N370S (E-059-2014/0); 6) LogicBio Therapeutics (L-243-2017/0) - Novel mouse models of methylmalonic acidemia (MMA) (E-139-2016/0); 7) LogicBio Therapeutics (L-244-2017) - Synthetic Methylmalonyl-CoA Mutase Transgene for the Treatment of Mut Class Methylmalonic Acidemia (MMA) (E-243-2012/0); 8) Shire Human Genetic Therapies, Inc. (L-260-2017/0) - Fibroblast Cell Lines Homozygous for Glucocerebrosidase (GBA1) Mutation N370S (E-059-2014/0); and 9) Selecta Biosciences, Inc. (L-272-2017) - Novel Mouse Model of Methylmalonic Acidemia (MMA) Mut-/- Tg INS-Mck-Mut (E-141-2016). In total, NHGRI currently has forty-nine (50) active licenses. In addition, in FY 2017 we processed one hundred forty-five (145) Material Transfer Agreements (MTAs), MTA Amendments and Information Transfer Agreements (ITAs). The vast majority of these agreements covered materials and data provided by NHGRI to outside entities and fifty-six percent (56%) of the MTAs, MTA Amendments and ITAs involved the transfer of human materials or data.
