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, as well as 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, as well as with research and academic institutions, 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. The following is the NHGRI technology transfer FY 2025 portfolio as of early August 2025. In FY 2025, three (3) new employee invention reports (EIRs) were evaluated by the TTO, assigned an official record number, classified as biological materials and/or non-patentable or used as a basis for filing a patent application. These EIRs were: 1) Mouse model of metabolism of cobalamin associated B (MMAB)for the study of methylmalonic acidemia (MMA), Lead PI: Charles P. Venditti; 2) Gene therapy for Leigh Syndrome, Lead PIs: Peter J. McGuire and Charles P. Venditti; and 3) Peptides to treat inflammatory diseases, Lead PI: Daniel Kastner. Six (6) new patent applications listing NHGRI inventors were filed, some by third party collaborators. The applications were filed in the U.S, and within the Patent Cooperation Treaty (PCT). The applications were related to virtual reality-based interventions to cognitive skills, gene therapy for Leigh Syndrome, peptides to treat inflammatory diseases, gene therapy to treat Gaucher disease, gene therapy to treat cholesterol storage disease, and gene therapy to treat propionic acidemia. Nine (9) new patents were issued in the NHGRI portfolio, some with third party collaborators. The titles of the patents were: Viral gene therapy as treatment for cholesterol storage disease or disorder (U.S. Patent 12,201,658); Cannabinoid Receptor Mediating Compounds (European Patent No: 3423448, France Patent No: 3423448, Germany Patent No: 3423448, Belgium Patent No: 3423448, United Kingdom Patent No: 3423448, Switzerland Patent No: 3423448, and The Netherlands Patent No: 3423448); and Codon-optimized human NPC1 genes for the treatment of Niemann-Pick Type C1 deficiency and related conditions (U.S. Patent 12,116,577). NHGRI had inventors on one hundred and seventy-six (176) active issued patents (U.S. and foreign, including with third party collaborators and other NIH Institutes) and one hundred and eighty-six (186) filed patent applications (U.S. and foreign, including those led by third party collaborators and other NIH Institutes). Two (2) new license agreements or amendments were executed for: development of RNA-mediated therapies for methylmalonic acidemia; and for mouse models of methylmalonic acidemia. Eight (8) new conditional gift fund (CGF) agreements or amendments were executed to support research for Gaucher disease, rare neuromuscular diseases, rare metabolic diseases, such as methylmalonic acidemia (MMA), and research tools to study neurodegenerative diseases. The following two (2) Cooperative Research and Development Agreements (CRADAs) or Amendments were executed: (1) “Pre-clinical development of RNA-mediated therapies of methylmalonic acidemia” (PI: Charles P. Venditti); (2) “Clinical development of N-acetyl-D-mannosamine (ManNAc) to treat GNE myopathy” (PI: William A. Gahl). A total of one-hundred and fifty (150) various transactional agreements, as well as consult cases, were negotiated and/or executed. Those included one (1) new Research Collaboration Agreement (RCA) and three (3) RCA amendments; fifty-seven (57) Material Transfer Agreements (MTAs) and amendments; twenty-seven (27) Information Transfer Agreements (ITAs)/Data Use Agreements (DUAs) and amendments; two (2) Memoranda of Understanding (MOUs); thirty-four (34) other agreements or various consults; eleven (11) Confidential Disclosure Agreements (CDAs) and amendments; and fifteen (15) video/written works release/speaking agreements.