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 2023 (as of August 16, 2023) three (3) 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. These EIRs were: Synthetic MMAB genes and AAV vectors to treat cobalamin B deficiency (cblB), Ref No.: E-134-2023, by inventors Charles Venditti and Eun-Young Choi, Patent Filing; MMAB knockout cell lines and uses to assay potency of MMAB gene-based medicines (expression constructs), EIR Ref. No: E-150-2023, by inventors Charles Venditti and Eun-Young Choi, Research Material; and Serum samples from patients with Adenosine Deaminase 2 Deficiency (DADA2), EIR Ref. No: E-205-2023, by inventors Ivona Aksentijevich and Daniel Kastner, Research Material. As of August 16, 2023, seven (7) new patent application were filed, based on NHGRI EIRs, some filed by third party collaborators. The titles of the applications were: Aberrant post-translational modifications (PTMS) in methyl- and propionic acidemia and a mutant sirtuin (SIRT) to metabolize PTMS (US application); Gene therapy for combined methylmalonic acidemia/aciduria and hyperhomocysteinemia/homocystinuria, cobalamin C type, and deficiency of MMACHC (US application); Gene therapy constructs for the treatment of propionic acidemia (US application); Novel adeno-associated viral (AAV) vectors to treat hereditary methylmalonic acidemia (MMA) caused by methylmalonyl-CoA mutase (MMUT) deficiency (US Application); High concentration methylcobalamin or combination of methyl- and hydroxycobalamin for the treatment of cobalamin C deficiency disorders (US Application); Synthetic MMAB genes and AAV vectors to treat cobalamin B deficiency (US Application); and Oligonucleotide analogues targeting human LMNA (Macao application). As of August 16, 2023, two (2) new patents were issued, some with third party collaborators. The titles of the two patents were: Gene therapy for combined methylmalonic acidemia/aciduria and hyperhomocysteinemia/homocystinuria, cobalamin C type, and deficiency of MMACHC (US Patent); and N-Acetyl Mannosamine as a therapeutic agent (Israeli Patent). As of August 16, 2023, NHGRI had inventors on one hundred and fifty-seven (157) active issued patents (U.S. and foreign, including with third party collaborators and other NIH Institutes) and on one hundred and eighty (180) filed patent applications (U.S. and foreign, including those led by third party collaborators and other NIH Institutes). In FY2023 we executed five (5) new license agreements or amendments with the following companies and/or organizations: Poseida Therapeutics, Inc., General Hospital Corporation, Leadiant Biosciences, Inc. (Amendment), LogicBio Therapeutics, and Purdue Research Foundation. In FY2023, we executed a total of one hundred and ninety-six (196) of various transactional agreements, as well as consult cases. In FY2023 we negotiated two (2) new conditional gift fund (CGF) agreements or amendments and/or grant terms and conditions agreements with: Neuromuscular Disease Foundation (Amendment 1), titled GNE Myopathy Research Fund; and Salla Treatment and Research Foundation (Amendment 2), titled Free Sialic Acid Storage Disorder (FSASD). In FY2023 we negotiated and executed nine (9) Cooperative Research and Development Agreements (CRADAs) or amendments with: Selecta Biosciences, Inc., titled Phase 1/2, open-label, single dose study to evaluate the safety, tolerability, and pharmacodynamics of SEL-302 (MMA 101 following administration of SEL-110) in pediatric subjects with mut subtype isolated methylmalonic acidemia (MMA); Cellphire Therapeutics, Inc., titled Preclinical development of allogeneic platelet replacement therapy for Hermansky-Pudlak Syndrome (HPS); Cellphire Therapeutics, Inc., Amendment #1, titled Preclinical development of allogeneic platelet replacement therapy for Hermansky-Pudlak Syndrome (HPS); Sanofi Genzyme, Amendment #1, titled A Multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2 (AMETHIST); Cycle Pharmaceuticals Ltd., titled Development of Nityr (nitisinone) treatment for alkaptonuria (AKU) in the United States; C-RCA with Mass General Brigham and Johns Hopkins University, titled Generalized Arterial Calcification of Infancy (GACI) Natural History and Lead In Prospective Biomarkers and Outcome Study; Floreo, Inc., Amendment #2, titled Clinical Development of Virtual Reality-based Interventions to Strengthen Cognitive Skillsets Related to Attention Deficit Hyperactivity Disorder (ADHD); Novartis Gene Therapies, Amendment 1, titled Pre-clinical development of AAV9-based gene therapy to treat Niemann-Pick Disease Type C; and Leadiant Biosciences, Inc., Amendment #5, titled Clinical Development of N-acetyl-D-mannosamine (ManNAc) to Treat GNE Myopathy. In FY2023 we executed ten (10) new Research Collaboration Agreements (RCAs) and one (1) RCA amendment. In FY2023 we processed eighty-eight (88) Material Transfer Agreements (MTAs) and MTA Amendments, thirty-three (33) Information Transfer Agreements (ITAs)/Data Use Agreements, two (2) service agreement terms and conditions, and three (3) Memoranda of Understanding (MOUs). One of the ITAs was a Master Data Use and Registration Agreement with Vanderbilt University for NHGRI investigators to have access (with specific approval process) to All of Us Research Program Research Hub for biomedical and health research. We also negotiated twenty-two (22) confidential disclosure agreements (CDAs) and CDA amendments and sixteen (16) standard NHGRI video/written works release agreements.