NHGRI/DIR Technology Transfer Program

National Human Genome Research Institute

Investigators

Abstract

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. In FY 2021 (as of August 12, 2021) two (2) new employee invention reports (EIRs) were evaluated by the TTO, filed with the NIH Office of Technology Transfer (OTT), assigned an official record number, and classified as biological materials (non-patentable). 1) Mouse Model of GM1 Gangliosidosis, E-136-2021-0, by inventors Cynthia Tifft and Elena-Raluca Nicoli 2) Mouse Model of Free Sialic Acid Storage Disorder (FSASD or Salla Disease), E-098-2021-0, by inventors Marjan Huizing, May Christine Vergara, John Burke, and Mary Hackbarth As of August 12, 2021, six (6) new patent application were filed, based on NHGRI EIRs. The titles of the six applications were: Synthetic Genes for the Treatment of Propionic Acidemia Caused by Mutations in Propionyl-CoA Carboxylase Beta; High Concentration Methylcobalamin (Me-Cbl) Or Combination Of Methyl- and Hydroxocobalamin (Me/OH-Cbl) For The Treatment Of Cobalamin C Deficiency And Related Disorders, Targeted Integration Into The Albumin (Alb) Locus As A Treatment For Hereditary Methylmalonic Acidemia (MMA) Caused By Methylmalonyl-coA Mutase (MUT) Deficiency, Cancer Detection Methods, LAMP1 Therapy in the Treatment of Lysosomal Storage Disorders, and Aberrant Post-Translational Modifications (PTMS) in Methyl- and Propionic Acidemia and a Mutant Sirtuin (SIRT) to Metabolize PTMS. As of August 12, 2021, three (3) patents were issued in the NHGRI portfolio. The titles of the issued patents were: Cancer Detection Methods, Synthetic Methylmalonyl-CoA Mutase Transgene for the Treatment of MUT Class Methylmalonic acidemia (MMA), and N-Acetyl Mannosamine as a Therapeutic Agent. As of August 12, 2021, NHGRI had thirty-four (34) active patent families, sixty (60) issued U.S. patents (including abandoned, expired and filed by third parties), one hundred and sixty-eight (168) issued foreign patents (including abandoned, expired and filed by third parties), and sixty-three (63) pending patent applications (U.S. and foreign), including third party leads. 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)). We 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 FY2021 we negotiated one (1) new conditional gift fund (CGF) agreement with the Hermansky-Pudlak Syndrome Network and two (2) CGF agreement amendments with A Cure for Clark and the Salla Treatment and Research (STAR). In FY2021 we negotiated and executed three (3) new Cooperative Research and Development Agreements (CRADAs) and one (1) CRADA Letter of Intent with the following companies: Novartis Gene Therapies, Asklepios BioPharmaceutical (2) and Selecta Biosciences. We also negotiated two (2) amendments to existing active CRADAs. In FY2021 we executed five (5) new Research Collaboration Agreements (RCAs) and one (1) RCA amendment. In FY2021 we executed nine (9) new license agreements with the following companies and/or organizations: Novartis Gene Therapies, GT Gain Therapeutics, Genespire, LogicBio, Asklepios BioPharmaceutical, Inc. (2), Selecta Biosciences (3). In FY2021 we processed one hundred thirty (130) Material Transfer Agreements (MTAs), MTA Amendments, Information Transfer Agreements (ITAs), Data Use Agreements, and various fee-for-service or purchase agreement terms and conditions agreements and amendments. We also negotiated thirty (30) confidentiality disclosure agreements (CDAs) and amendments and forty-three (43) standard NHGRI video/written works release agreements and one (1) Advisory Board committee participation agreement.