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 using 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 six (6) new employee invention reports (EIRs) were evaluated in FY2019 by the TTO. Three (3) were filed with the NIH Office of Technology Transfer (OTT) and assigned an official record number. Three (3) were for biological materials and three (3) were for technologies that could be patented or at least evaluated for patenting (one filing was done by a third-party CRADA collaborator). Twenty (20) new patent applications were filed in FY2019. The titles of the twenty (20) patent applications filed are: Isotopic Biomarkers of Organic Acidemias (E-148-2017, PCT application filed on October 31, 2018); Non-Disruptive Gene Therapy for the Treatment of MMA (E-034-2019, PCT Application filed on October 30, 2018); Oligonucleotide Analogues Targeting Human LMNA (E-044-2013, (U.S. application filed on October 25, 2018), (European application filed on November 2, 2018), (Chinese application filed on December 28, 2018), (Australian application filed on October 25, 2018), (Brazilian application filed on October 29, 2018), (Canadian application filed on October 25, 2018), (Colombian application filed on November 27, 2018), (Indian application filed on November 19, 2018), (South Korean application filed on November 28, 2018), (Mexican application filed on October 26, 2018), (New Zealand application filed on October 25, 2018), (Singapore application filed on October 26, 2018), (Eurasian application filed on November 28, 2018), and (Japanese application filed on October 26, 2018), Detecting Protein Interaction Sites in Nucleic Acids (E-031-2018, PCT application filed on January 9, 2019), Synthetic Methylmalonyl-CoA Mutase Transgene for the Treatment of Mut Class Methylmalonic Acidemia (MMA) (E-243-2012, U.S. application filed on April 22, 2019), N-Acetyl Mannosamine as a Therapeutic Agent (E-217-2007, U.S. application filed on May 20 2019), and Synthetic Genes for the Treatment of Propionic Acidemia Caused by Mutations in Propionyl-COA Carboxylase Alpha (E-104-2019, U.S. application filed on June 27, 2019). In FY2019, two (2) patents were issued in the NHGRI portfolio. The titles of the issued patents are: Synthetic Methylmalonyl-COA Mutase Transgene for the Treatment of Mut Class Methylmalonic Acidemia (MMA) (U.S. Patent 10,307,469, NHGRI lead inventor Charles Venditti); and N-Acetyl Mannosammine as a Therapeutic Agent (U.S. Patent 10,335,426, NHGRI lead inventor Marjan Huizing). As of the end of July 2019, NHGRI had thirty-eight (38) active patent families, fifty-five (55) issued U.S. patents (including abandoned and expired), one hundred and forty-nine (149) issued foreign patents (including abandoned and expired), and sixty-two (62) pending patent applications (U.S. and foreign). In FY2019, NHGRI negotiated and executed two (2) new conditional gift fund agreements with the following organizations: Neuromuscular Disease Foundation (Research Project A Randomized, Double-Blinded, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy of N-acetyl-D-mannosamine (ManNAc) in Subjects with GNE Myopathy; NHGRI PI is William Gahl, M.D., Ph.D.), and the Salla Treatment and Research Foundation (for ongoing scientific research focused on Salla disease; NHGRI PI is William Gahl, M.D., Ph.D.). 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 Michael J. Fox Foundation for Parkinsons Research (Research Project Development of a Novel TRPML1 Activator for Treatment of Parkinson's Disease, NHGRI PI is Ellen Sidransky, M.D. (granted)). New FY2019 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), Parkinsons Disease and propionic acidemia. In FY2019 we negotiated and executed one (1) Cooperative Research and Development Agreements (CRADAs) with the following companies: Axovant Sciences Inc. (Axovant) and the University of Massachusetts (UMass). Under this three-party CRADA, entitled Development of Gene Therapy for GM1 Gangliosidosis, Dr. Cynthia Tifft at NHGRI will use a gene therapy vector from UMass to conduct a clinical study at NIH. During the clinical trial, pediatric patients with a rare lysosomal storage disorder called GM1 gangliosidosis will be treated with gene therapy, and the resulting data will be used to support FDA filings and commercialization of the gene therapy. We also negotiated three (3) amendments for existing CRADAs. In FY2019 we executed five (5) new license agreements with the following companies and/or organizations: 1) LogicBio Therapeutics, Inc. (L-069-2019/0) - A Synthetic Methylmalonyl-CoA Mutase Transgene for the Treatment of Mut Class Methylmalonic Acidemia (MMA) (E-243-2012/0); 2) Lysosomal Therapeutics, Inc. (L-055-2015/1) ; 3) HemoShear Therapeutics, LLC (L-128-2019/0) - Glucocerebrosidase Activators as a Treatment for Gaucher Disease (E-257-2010/0); 4) Agios Pharmaceuticals, Inc. (L-167-2019/0) - Novel Mouse Model of Mut- Methylmalonic Acidemia (MMA) Mut-/- Tg CBAMutG715V: Mut Partial-Deficiency (E-140-2016/0); and 5) LifeMax Laboratories, Inc. (L-207-2019/0) Novel Mouse Model of Methylmalonic Acidemia (MMA) (E-141-2016/0). In addition, in FY 2019 we processed one hundred seventy-six (176) 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.
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