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. Approximately ten (10) new employee invention reports (EIRs) were evaluated in FY2018 by the TTO. Eight (8) were filed with the NIH Office of Technology Transfer (OTT) and assigned an official record number. Eight (8) were for biological materials and two (2) were for technologies that could be patented or at least evaluated for patenting. Fifteen (15) new patent applications were filed in FY2018. The titles of the fifteen (15) patent applications filed are: Detecting Protein Interaction Sites in Nucleic Acids (E-031-2018, U.S. provisional application filed on January 9, 2018); Oligonucleotide Analogues Targeting Human LMNA (E-044-2013, PCT application filed on April 28, 2017 and U.S. application filed on October 6, 2017); Tagged Form of MUT Enzyme, Genetic Constructs Incorporating It, and Its Use in Gene Therapy (E-090-2015, European application filed on October 6, 2017); Novel Codon-Optimized and Tagged Human NPC1 Genes and AAV Vectors for the Treatment of Niemann-Pick Type C1 Deficiency and Related Conditions, (E-100-2017, PCT application filed on June 20, 2018); Methods of Detecting and Treating GATM-Related Renal Disorders (E-110-2017; PCT application filed on May 31, 2018); Serum Protein Biomarkers That Predict The Response To Liver Directed Therapy In Methylmalonic Acidemia (MMA) (E-148-2017; U.S. provisional application filed on October 31, 2017); A Method of Detecting Molecules in Proximity to a Target Molecule in a Sample (E-161-2015; European application filed on March 21, 2018; and U.S. application filed on February 28, 2018); Cancer Detection Methods (E-177-2015, U.S. application filed on March 12, 2018, and European application filed on March 14, 2018); Viral Gene Therapy as Treatment for Cholesterol Storage Disease or Disorder (E-185-2014; Canadian application filed on October 6, 2017; European application filed on November 8, 2017; and U.S. application filed on October 6, 2017); Cellular Arrays and Methods of Detecting and Using Genetic Disorder Markers (E-007-1999, Japanese application filed on October 13, 2017). In FY2018, eight (8) patents were issued in the NHGRI portfolio. The titles of the issued patents are: Hyposialylation Disorders (European Patent 2972390, UK Patent 2972390, French Patent 2972390, and German Patent 602014016875.2, NHGRI lead inventor Marjan Huizing); N-Acetyl Mannosamine as a Therapeutic Agent (Japanese Patent 6283298, NHGRI lead inventor Marjan Huizing); Methods for Treating Progeroid Laminopathies Using Oligonucleotide Analogues Targeting Human LMNA (European Patent 2788488, NHGRI lead inventor Francis Collins); Synthetic Methylmalonyl-COA Mutase Transgene for the Treatment of MuT Class Methylmalonic Acidemia (MMA) (U.S. Patent 9,944,918, NHGRI lead inventor Charles Venditti); and Compositions and Methods for Predicting Onset of a Lysosomal Storage Disease or a Disease Associated with a Lysosomal Defect (U.S. Patent 9,983,200, NHGRI lead inventor William Pavan). As of the end of July 2018, NHGRI had thirty-eight (38) active patent families, fifty-four (54) issued U.S. patents (including abandoned and expired), one hundred and forty-four (144) issued foreign patents (including abandoned and expired), and fifty-five (55) pending patent applications (U.S. and foreign). In FY2018, NHGRI negotiated and executed two (2) new conditional gift fund agreements with the following organizations: Neuromuscular Disease Foundation and the Propionic Acidemia Foundation. 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 Propionic Acidemia Foundation (granted). New FY2018 executed conditional gift fund agreements and submitted non-NIH research grants focused on the following human disease research topics: neuromuscular disorders (such as GNE myopathy) and propionic acidemia. In FY2018 we negotiated and executed five (5) Cooperative Research and Development Agreements (CRADAs) with the following companies: 1) Synlogic, Inc., 2) StrideBio, Inc., 3) Inozyme Pharma, Inc., 4) Leadiant Biosciences, Inc., and 5) Translate Bio, Inc. We also negotiated one (1) amendment for an existing CRADA. In FY2018 we executed nine (9) new license agreements with the following companies and/or organizations: 1) Selecta Biosciences, Inc. (L-017-2018/0) - Novel Mouse Model of Methylmalonic Acidemia (MMA) Mut-/- Tg INS-Mck-Mut (E-141-2016/0); 2) Selecta Biosciences, Inc. (L-075-2018/0) - Novel Mouse Model of Methylmalonic Acidemia (MMA) Mut-/- Tg INS-Mck-Mut (E-141-2016/0); 3) Emergent Product Development Gaithersburg Inc. (L-151-2016/1) - Human Cell Lines with Mannosyl Olygosaccharide Glycosidase (MOGS) Defect (E-129-2016/0); 4) StrideBio, Inc. (L-110-2018/0) Viral Gene Therapy as Treatment for Cholesterol Storage Disease or Disorder (E-185-2014/0 and E-110-2017/0); 5) Leadiant Biosciences, Inc. (L-146-2018/0) - N-Acetyl Mannosamine as a Therapeutic Agent (E-217-2007/0); 6) Moderna Therapeutics, Inc. (L-168-2018/0) - 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); 7) Ionis Pharmaceuticals, Inc. (L-201-2015/4) - Mouse Model of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Vascular Abnormalities (E-243-2011/0); 8) Chameleon Biosciences, Inc. (L-204-2018/0) - Viral Gene Therapy as Treatment for Cholesterol Storage Disease or Disorder (E-185-2014/0); and 9) StrideBio, Inc. (L-216-2018/0) Viral Gene Therapy as Treatment for Cholesterol Storage Disease or Disorder (E-185-2014/0) and Codon-Optimized Human NPC1 Genes for the Treatment of Niemann-Pick Type C1 Deficiency and Related Conditions (E-100-2017/0). In total, NHGRI currently has fifty-five (55) active licenses. In addition, in FY 2018 we processed one hundred twenty-eight (128) 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.
