Assay for Predicting the Time of Onset of Niemann-Pick Disease Type C (NPC)
Niemann-Pick Disease, type C (NPC) is a rare, autosomal recessive, neurodegenerative disease. Approximately 95% of patients with NPC have mutations in NPC1, a gene implicated in intracellular cholesterol trafficking. Mutation of NPC1 causes intracellular accumulation of unesterified cholesterol in late endosomal/lysosomal structures and marked accumulation of glycosphingolipids, especially in neuronal tissue. Thus, NPC patients generally present with hepatosplenomegaly (enlargement of liver and spleen) and neurological degeneration.NPC is highly heterogeneous in both mutations and time of onset. Most mutations in individua...
Source: NIH OTT Licensing Opportunities - May 29, 2020 Category: Research Authors: ott-admin Source Type: research
Codon-Optimized Gene Therapy for Niemann-Pick Disease Type C
Niemann Pick Disease Type C (NPC) is a rare and fatal, autosomal recessive, neurodegenerative disease that can present in infants, children, or adults. Most patients with NPC have mutations in NPC1, a gene implicated in intracellular cholesterol trafficking, which results in intracellular accumulation of unesterified cholesterol in late edosomal/lysosomal structures and of glycosphingolipids, especially in neuronal tissue. No curative therapy exists at present.Adding to their previous work and patent portfolio of NPC gene constructs, NHGRI investigators have generated improved and codon-optimized gene vectors. These new ad...
Source: NIH OTT Licensing Opportunities - May 29, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
Gene Therapy for Niemann-Pick Disease Type C
Investigators at the National Human Genome Research Institute (NHGRI) at the National Institutes of Health (NIH) are seeking collaborators to further develop viral gene therapy to treat Niemann-Pick Disease Type C (NPC). NPC is a rare and fatal, autosomal recessive, neurodegenerative disease that can present in infants, children, or adults. Most patients with NPC have mutations in NPC1, a gene implicated in intracellular cholesterol trafficking, which results in intracellular accumulation of unesterified cholesterol in late edosomal/lysosomal structures and of glycosphingolipids, especially in neuronal tissue. Thus, NPC pa...
Source: NIH OTT Licensing Opportunities - May 29, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
A Rapid Ultrasensitive Assay for Detecting Prions Based on the Seeded Polymerization of Recombinant Normal Prion Protein (rPrP-sen)
Prion diseases are neurodegenerative diseases of great public concern as humans may either develop disease spontaneously or, more rarely, due to mutations in their prion protein gene or exposures to external sources of infection. Prion disease is caused by the accumulation in the nervous system of abnormal aggregates of prion protein. This technology enables rapid, economical, and ultrasensitive detection of disease-associated forms of prion protein. Specifically, prion aggregates (contained in a biological sample) seed the polymerization of recombinant, monomeric prion protein (rPrP-sen) and the polymerized product is det...
Source: NIH OTT Licensing Opportunities - May 29, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
Alpha-Synuclein RT-QuIC: An Ultrasensitive Assay for the Detection of Alpha-Synuclein Seeding Activity Associated with Synucleinopathies
Synucleinopathies are a category of neurodegenerative diseases defined by the abnormal aggregation and accumulation of misfolded alpha-synuclein protein molecules within the brain. These aggregates are of particular concern to humans as they are a primary cause of Parkinson ’s disease, dementia with Lewy bodies, and other neurological disorders. This technology enables rapid, economical and ultrasensitive detection of disease-associated forms of alpha-synuclein as biomarkers or indicators of synucleinopathy in a biological sample. Specifically, alpha-synuclein aggreg ates (contained in a biological sample) seed the polym...
Source: NIH OTT Licensing Opportunities - May 29, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
Assay for Predicting the Time of Onset of Niemann-Pick Disease Type C (NPC)
Niemann-Pick Disease, type C (NPC) is a rare, autosomal recessive, neurodegenerative disease. Approximately 95% of patients with NPC have mutations in NPC1, a gene implicated in intracellular cholesterol trafficking. Mutation of NPC1 causes intracellular accumulation of unesterified cholesterol in late endosomal/lysosomal structures and marked accumulation of glycosphingolipids, especially in neuronal tissue. Thus, NPC patients generally present with hepatosplenomegaly (enlargement of liver and spleen) and neurological degeneration.NPC is highly heterogeneous in both mutations and time of onset. Most mutations in individua...
Source: NIH OTT Licensing Opportunities - May 29, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
Inducible Activation Nucleic Acid Hybrid Switch for Conditional Generation of Oligonucleotides
Gene therapy research has yielded FDA-approved treatments for an array of diseases. However, challenges facing nucleic-acid based therapeutics include non-specific delivery and degradation of the nanoparticles. NCI investigators have developed a solution to address these challenges in their novel nucleic-based therapy based on the conditional activation strategy. The inducible activation nucleic acid hybrid switch overcomes the drawbacks of current technologies through its unique design. The implementation of nucleic acid logic elements in these constructs circumvents off-target effects. The functional oligonucleotide co...
Source: NIH OTT Licensing Opportunities - May 1, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
Induced Pluripotent Stem Cells Derived from Patients with CEP290-associated Ciliopathies and Unaffected Family Members
Approximately one-third of non-syndromic retinal dystrophies involve a defect in a ciliary protein. Non-syndromic retinal ciliopathies include retinitis pigmentosa, cone dystrophy, cone-rod dystrophy, macular dystrophy, and Leber-congenital amaurosis (LCA). Many CEP290-LCA patients also exhibit auditory and olfactory defects. Induced pluripotent stem cells (iPS) cells were derived from patients with LCA and unaffected relatives.
The National Eye Institute (NEI) seeks research collaborations and/or licensees for the use of these iPS cells.IC: NEINIH Ref. No.: E-100-2020Advantages: Extensive characterizati...
Source: NIH OTT Licensing Opportunities - April 30, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
T-Cell Receptors Targeting Epstein Barr Virus Latent Membrane Protein 2 for Treatment of Lymphomas and Epithelial Cancer
Epstein Barr Virus (EBV) is one of the most common human viruses in the world and often leads to persistent latent infection. EBV-related cancers have the common feature of harboring latent EBV within the cancer cells, and include cancer such as lymphomas (Hodgkin lymphoma, T/NK cell lymphoma, and post-transplant lymphoproliferative disorders) and certain incurable epithelial cancers (nasopharyngeal cancer and gastric cancer). In such cases, the EBV Latent Membrane Protein 2 (EBV-LMP2), a transmembrane protein, is highly expressed by cancer cells and not in life-essential tissues, making EBV-LMP2 an attractive target for c...
Source: NIH OTT Licensing Opportunities - April 28, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
EGFRvIII Antibodies for the Treatment of Human Cancer
Epidermal growth factor receptor variant III (EGFRvIII) is a variant of EGFR that is an excellent target for immunotherapy because of its expression in cancer cells and not in normal cells. Inventors from the National Cancer Institute (NCI) have isolated seven mouse monoclonal antibodies that bind to the human EGFRvIII but not wildtype EGFR. These EGFRvIII antibodies can be used as either independent agents or targeting domains in recombinant immunotoxins (RITs), antibody-drug conjugates (ADCs), bispecific antibodies, and chimeric antigen receptors (CARs). Significantly, RITs using one of the antibodies (40H3) have shown...
Source: NIH OTT Licensing Opportunities - April 28, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
Recombinant Prefusion Measles and Mumps F and F –HN (H) Glycoproteins for Vaccine Development
The Measles virus (MeV) and Mumps virus (MuV) are highly contagious paramyxoviruses that can be transmitted by respiratory droplets from or on direct contact with an infected person. The resulting diseases can lead to serious complications or death among children. The existing vaccines for MeV and MuV are live attenuated virus vaccines which are administered in two subcutaneous doses at 1 year of age and as early as one month later. Two doses of a combination measles, mumps and rubella vaccine are 97% effective against measles and 88% against mumps. A single dose of a combination measles, mumps, and rubella vaccine is 93% ...
Source: NIH OTT Licensing Opportunities - April 24, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
Use of the Intracellular Signaling Domain of Receptor CD28H as a Component of Chimeric Antigen Receptors to Overcome Inhibition of Cytotoxic Lymphocytes by Checkpoint Receptors
Engineered chimeric antigen receptors (CARs) that are expressed in cytotoxic T cells and natural killer (NK) cells have been used to specifically target tumor cells. However, CAR-T and CAR-NK cells are still subject to downregulation by their inhibitory receptors after injection into patients.Scientists at NIAID have developed CAR constructs that overcome inhibition of NK cells by receptors for human major histocompatibility complex molecules HLA-E and HLA-C, based on in vitro studies. The CAR contains an antigen binding domain of receptor CD28 homolog (CD28H), a CD28H transmembrane domain (TM), a CD28H signaling domain, a...
Source: NIH OTT Licensing Opportunities - April 20, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
Peptide Hydrogels for Rate-Controlled Delivery of Therapeutics
Hydrogels represent an attractive controlled drug-delivery system that have been used in various clinical applications, such as: tissue engineering for wound healing, surgical procedures, pain management, cardiology, and oncology. High-water content of hydrogels confers tissue-like physical properties and the crosslinked fibrillar network enables encapsulation of labile small molecule drugs, peptides, proteins, nucleic acids, proteins, nanoparticles, or cells. The porosity of the mesh-like network contributes to enhanced protection and controlled release of therapeutics compared with the rapid clearance and degradation of ...
Source: NIH OTT Licensing Opportunities - April 20, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
Combined PIKFYVE and p38 MAP Kinase Inhibition for Treating Cancer
Cancer cells can upregulate autophagy – recycling of components – as a response to chemotherapy. Investigators in Dr. Melvin DePamphilis’ laboratory at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) have shown that PIKFYVE inhibitors and p38 MAP kinase inhibitors work synergistically in vitro to kill cancer cells (e.g., colon adenocarcinomas, non-small cell lung carcinoma, glioblastoma, breast adenocarcinoma and osteosarcoma), but are not toxic to normal cells. Cancer cells with a BRAF mutation are especially dependent on autophagy. Treatment of cancer cells containing t...
Source: NIH OTT Licensing Opportunities - April 20, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
Human Monoclonal Antibodies Against Dengue Viruses
Dengue viruses cause dengue outbreaks and major epidemics in most tropical and subtropical areas where Aedes albopictus and Aedes aegypti mosquitoes are abundant. Among the arthropod-borne flaviviruses, the four dengue virus serotypes, dengue type 1 virus (DENV-1), dengue type 2 virus (DENV-2), dengue type 3 virus (DENV-3), and dengue type 4 virus (DENV-4) are most important in terms of human morbidity and geographic distribution.A safe and effective vaccine against dengue is currently not available. Passive immunization with monoclonal antibodies from non-human primates or humans represents a possible alternative to vacci...
Source: NIH OTT Licensing Opportunities - April 11, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research
