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 2022 (as of July 20, 2022) five (5) 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) or used as a basis for filing a patent application. 1) Mouse Model of Osteoglophonic Dysplasia, E-097-2022-0, by inventor Carlos R. Ferreira, research material. 2) GBA1 (glucosylceramidase beta gene) Induced Pluripotent Stem Cell (iPSC) Isogenic Cell Lines (GBA1 N370S/N370S, Knockout/Knockout (KO/KO), and wildtype/wildtype (WT/WT)) for Use in Gaucher Disease and Parkinson Disease Research, E-159-2022-0, by inventors Yu Chen, Chase Chen, Richard Sam, and Ellen Sidransky, research material. 3) Synthetic Genes For The Treatment Propionic Acidemia (PA) Caused By Mutations In PropionylcoA Carboxylase Alpha (PCCA), E-034-2022-0, by inventors Charles Venditti, and Randy Chandler, Patent filing. 4) Viable Murine Model Of Severe Propionic Acidemia (PA) With An Orthologous Human Propionyl-CoA Carboxylase Alpha (PCCA) Missense Muration: C.424C>A (p.T81N), E-141-2022-0, by inventors Charles Venditti, and Randy Chandler, research material. 5) Lentiviral Gene Therapy Vectors To Treat MMUT Type Methylmalonic Acidemia (MMA), E-163-2022-0, Charles Venditti, and Tomoyasu Higashimoto, Patent Filing. As of end of July, 2022, ten (10) new patent application were filed, based on NHGRI EIRs, some filed by third party collaborators. The titles of the ten applications were: Non-disruptive Gene Therapy for the Treatment of MMA (two Hong Kong applications); Gene Therapy Constructs for the Treatment of Propionic Acidemia Caused by Mutations in Propionyl-CoA Carboxylase Alpha (U.S. provisional application); Oligonucleotide Analogues Targeting Human LMNA (Japanese application); Synthetic Genes for the Treatment of Propionic Acidemia Caused by Mutations in Propionyl-CoA Carboxylase Alpha (European and U.S. applications); Cancer Detection and Classification (U.S. application); High Concentration Methylcobalamin or Combination of Methyl- and Hydroxycobalamin for the Treatment of Cobalamin C Deficiency and Related Disorders (PCT application); and Synthetic Genes for the Treatment of Propionic Acidemia (PA) Caused by Mutations in Propionyl-CoA Carboxylase Beta (PCCB) (U.S. and European applications). As of July 20, 2022, six (6) new patents were issued, some with third party collaborators. The titles of the six patents were: Oligonucleotide Analogues Targeting Human LMNA (Japanese, Korean, and Colombian patents); Biomarkers of Acidemia (U.S. patent); N-Acetylmannosamine as a Therapeutic Agent (Canadian patent); and Cannabinoid Receptor Mediating Compounds (U.S. patent). As of July 20, 2022, NHGRI had thirty (30) active patent families (including those being prosecuted by third-party collaborators), seventeen (17) active issued U.S. patents (excluding abandoned and expired), forty-seven (47) issued foreign patents (excluding abandoned and expired), and fifty-nine (59) pending patent applications (U.S. and foreign, including those led by third party collaborators). In FY2022 we negotiated four (4) new conditional gift fund (CGF) agreements and/or grant terms and conditions agreements: two (2) with the Michael J. Fox Foundation (one entitled Development of live-cell GCase activity assays to advance non-inhibitory GCase chaperones for treating Parkinson disease and one entitled Testing and validation of novel anti-GCase, anti-GlcCer, and anti-GlcSph antibodies), 1 with the Neuromuscular Diseases Foundation (NDF) and 1 with the Lewy Body Dementia Association (LBDA) (a Mentorship Award Agreement for a NHGRI trainee) and one (1) CGF agreement amendment with the Salla Treatment and Research (STAR) foundation. In FY2022 we negotiated and executed one (1) Cooperative Research and Development Agreement (CRADA) with Merck Sharp & Dohme (entitled Clinical Development of Miransertib to Treat Proteus Syndrome) and two (2) CRADA Letters of Intent (LOI) with the following companies: Cycle Pharmaceuticals, Ltd. and Selecta Biosciences (1 CRADA and 2 LOIs). We also negotiated five (5) amendments to existing active CRADAs. In FY2022 we executed four (4) new Research Collaboration Agreements (RCA) and one (1) RCA amendment. In FY2022 we executed seven (7) new license agreements with the following companies and/or organizations: Invitae Corporation, Progeria Research Foundation, University of Maryland, Chameleon Biosciences, Inc, Cancer Research Technology Limited (Ximbio), Altos Labs, and Applied Biological Materials, Inc. In FY2022 we processed seventy-two (72) Material Transfer Agreements (MTAs) and MTA Amendments, forty-eight (48) Information Transfer Agreements (ITAs)/Data Use Agreements, and nine (9) fee-for-service or purchase agreement terms and conditions agreements and amendments. We also negotiated eighteen (18) confidentiality disclosure agreements (CDAs) and CDA amendments and twenty-one (21) standard NHGRI video/written works release agreements and one (1) Advisory Board committee participation agreement.