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 p...
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 oligonucleotid...
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 characteri...
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 s...
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 ce...
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

Nucleic Acid Nanoparticles for Triggering RNA Interference
RNA interference (RNAi) is a naturally occurring cellular post-transcriptional gene regulation process that utilizes small double-stranded RNAs to trigger and guide gene silencing. By introducing synthetic RNA duplexes called small-interfering RNAs (siRNAs), we can harness the RNAi machinery for therapeutic gene control and the treatment of various diseases.  NCI researchers created RNA, RNA-DNA,  or DNA-RNA hybrid nanocubes consisting of a DNA or RNA core (composed of six strands) with attached RNA or DNA hybrid duplexes. The nanocubes can induce the reassociation of the RNA duplexes, which can then be processe...
Source: NIH OTT Licensing Opportunities - April 11, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Detection of Novel Endocrine-Disrupting Chemicals in Water Supplies
There is a growing interest in the cancer risk posed by endocrine-disrupting chemicals (EDCs)in our environment. Steroidal EDCs interfere with the normal function of the endocrine system and have been associated with cancer. Currently, detection and monitoring of water sources for steroidal contamination of water relies on a laborious analysis of their chemical structures. Considering that many natural steroids are rapidly metabolized, their derivatives are frequently not present in the currently existing libraries and thus cannot be identified. In addition, it is unclear whether EDCs detected by chemical methods can elici...
Source: NIH OTT Licensing Opportunities - April 11, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

A Novel Genetically Encoded Inhibitor of Hippo Signaling Pathway to Study YAP1/TAZ-TEAD Dependent Events in Cancer
In conclusion, TEADi is a valuable research tool for studying YAP1/TAZ and the Hippo pathway in cancer and other pathologies, with improved advantages that include rapid and simple inhibition of TEAD transcription and specific blockage of nuclear events mediated by both YAP1 and TAZ without affecting structural or cytoplasmic functions of these proteins.The NCILaboratory of Cellular and Molecular Biologyis seeking statements of capability or interest from parties interested in licensing this novel inhibitor of the Hippo signaling pathway.IC: NCINIH Ref. No.: E-108-2019Advantages: Rapid and simple inhibition ...
Source: NIH OTT Licensing Opportunities - April 2, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Substrate Reduction Therapy for Smith-Lemli-Opitz Syndrome and Related Disorders
Smith-Lemli-Opitz Syndrome (SLOS) is a rare autosomal recessive genetic disorder affecting the final step of cholesterol biosynthesis. SLOS is characterized by slow growth before and after birth, mental retardation, and multiple congenital disabilities. There is no FDA approved treatment for SLOS. Patients may benefit moderately from palliative care through an increase in dietary cholesterol to compensate for the endogenous block in cholesterol biosynthesis. However, dietary change offers only limited clinical benefit in mental improvement because the level of cholesterol or 7-DHC in the cerebrospinal fluid is not signific...
Source: NIH OTT Licensing Opportunities - March 21, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Methods of Producing T-cell Populations Using P38 MAPK Inhibitors
Adoptive cell therapy (ACT) uses cancer reactive T-cells to effectively treat patients. However, several obstacles inhibit the successful use of ACT for cancer treatment.   Current approaches for the expansion of T-cells may produce T-cells with a terminally differentiated phenotype that is associated with diminished anti-tumor activity and poor capacity for long-term persistence. Studies have shown that improving metabolic properties and persistence of T-cells duri ng ex vivo expansion could improve anti-tumor efficacy of T-cells. Thus, there is a need for improved methods of obtaining an isolated population of effec...
Source: NIH OTT Licensing Opportunities - March 20, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Ebola Virus Glycoprotein-Specific Monoclonal Antibodies and Uses Thereof
Ebola virus is a large, negative-strand RNA virus composed of 7 genes encoding viral proteins, including a single glycoprotein (GP). The virus is responsible for causing Ebola virus disease (EVD), formerly known as Ebola hemorrhagic fever (EHF), in humans. In particular, Bundibugyo (BDBV), Zaire (EBOV), and Sudan (SUDV) species have been associated with large outbreaks of EVD in Africa and reported case fatality rates of up to 90%. Transmission of Ebola virus to humans is not yet fully understood but is likely due to incidental exposure to infected animals. EVD spreads through human-to-human transmission, with infection re...
Source: NIH OTT Licensing Opportunities - March 9, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Fluorescent Primer(s) Creation for Nucleic Acid Detection and Amplification
CDC researchers have developed technology that consists of a simple and inexpensive technique for creating fluorescent labeled primers for nucleic acid amplification. Fluorescent chemical-labeled probes and primers are extensively used in clinical and research laboratories for rapid, real-time detection and identification of microbes and genetic sequences. During nucleic acid amplification, the " UniFluor " primer is incorporated into newly synthesized double stranded DNA. As a consequence, quenching of the dye's fluorescent signal occurs decreasing the fluorescence of the sample several fold. The decrease in flu...
Source: NIH OTT Licensing Opportunities - March 9, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Nucleic Acid Amplification Technique for Rapid Diagnostic Analysis
CDC researchers developed a simple target-specific isothermal nucleic acid amplification technique, termed Genome Exponential Amplification Reaction (GEAR). The method employs a set of four primers (two inner and two outer). The outer primer pair targets three specific nucleic acid sequences at a constant 60 °C, while the inner pair of primers accelerates and improves the sensitivity of the assay.The GEAR technique is an improvement over loop-mediated isothermal amplification (LAMP) in three ways. First, the GEAR method uses two Tab primers which target three genomic regions (corresponding LAMP primers target four regi...
Source: NIH OTT Licensing Opportunities - March 9, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Photoinduced Electron Transfer Fluorescent Primer for Nucleic Acid Amplification
CDC scientists have developed a rapid and cost-efficient method for generating fluorescently labeled primers for PCR and real-time PCR. At present, fluorescent primers are useful for detecting and identifying microbes and specific nucleic acid sequences, amplifying nucleic acids for pyro-sequencing, determining the levels of gene expression, and many other uses. However, problems exist with current techniques used to create fluorescent primers. For one, labeling is not one hundred percent efficient, leading to inaccurate results. Further, it is expensive and time consuming for researchers to make and label their own unique...
Source: NIH OTT Licensing Opportunities - March 9, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Reprogrammed Tumor Infiltrated Lymphocytes for Efficient Identification of Tumor-Antigen Specific T-Cell Receptors
Adoptive T Cell Therapy (ACT) is a form of cancer immunotherapy. It consists of harvesting Tumor Infiltrated Lymphocytes (TIL), screening for TIL which display tumor antigen-specific T-cell receptors (TCR), expanding these in vitro, and reinfusing into the patient for treatment. While ACT has proven effective in treating various cancer types, it is a laborious procedure as the harvested TIL population has generally limited expansion and a finite lifespan.Researchers at the National Cancer Institute (NCI) Surgery Branch improved identification and isolation of the tumor antigen-specific TCR by reprogramming TIL into induced...
Source: NIH OTT Licensing Opportunities - March 4, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Monoclonal Antibodies Against Bacillus Anthracis Antigens
Anthrax, whether resulting from natural or bioterrorist-associated exposure, is a constant threat to human health. Bacillus anthracis is the causative agent of anthrax. It is surrounded by a polypeptide capsule of poly-gamma-D-glutamic acid (gamma-D-PGA), which is essential for virulence, is poorly immunogenic and has anti-phagocytic properties. Antibodies to the capsule have been shown to enhance phagocytosis and killing of encapsulated bacilli. The lethality of anthrax is primarily the result of the effects of anthrax toxin, which has 3 components: a receptor-binding protein known as " protective antigen " (PA)...
Source: NIH OTT Licensing Opportunities - February 25, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Handwipe Disclosing Method for Detecting the Presence of Lead
Lead (Pb) exposure can cause serious health concerns including abdominal pain, headaches, loss of appetite, memory loss, weakness, and other symptoms. Lead residues on human skin, especially on the hands of workers can be a significant health risk since such residues may be ingested during normal activities (e.g. eating, drinking, and smoking). A key component to reducing lead exposure is being able to identify areas of lead contamination.US Centers for Disease Control and Prevention (CDC), National Institute for Occupational Safety and Health (NIOSH) researchers developed a method to detect lead on surfaces, including ski...
Source: NIH OTT Licensing Opportunities - February 13, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Wipes and Methods for Removal of Lead and Other Metal Contamination from Surfaces
Exposure to lead (Pb) has long posed serious health risks. Ingestion of lead from skin exposure can adversely impact every organ in the body; the kidneys, blood, nervous, and reproductive systems are most affected. Washing skin with soap and water is not sufficient to remove lead residues. To prevent adverse impacts from Pb exposure, exposed individuals need cleaning methods that will effectively remove Pb ions from the skin to less than the limit of identification (i.e., 10 µg or less).Centers for Disease Control and Prevention (CDC), National Institute for Occupational Safety and Health (NIOSH) researchers develope...
Source: NIH OTT Licensing Opportunities - February 13, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Therapeutic and Diagnostic Targets for Severe RSV Infection
Respiratory Syncytial Virus (RSV) infects nearly all children by their second birthday. RSV usually causes mild respiratory illness, however, a subset of patients experience severe infection that require hospitalization. Successful host defense against viral pathogens requires rapid recognition of the virus and activation of both innate and adaptive immunity. Toll-Like Receptors (TLRs) are responsible for mounting an innate immune response and genetic variations within TLRs modulate severity of infection. Researchers at NIEHS have identified a single nucleotide polymorphism (SNP) in TLR8 that is associated with RSV disease...
Source: NIH OTT Licensing Opportunities - February 6, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

T-Cell Therapy Against Patient-Specific Cancer Mutations
Human cancers contain genetic mutations that are unique to each patient. Some of the mutated peptides are immunogenic, can be recognized by T cells, and therefore, may serve as therapeutic targets.Scientists at theNational Cancer Institute's Surgery Branch developed a method to identify T cells that specifically recognize immunogenic mutations expressed only by cancer cells. The scientists identified cancer-specific mutations from a patient with widely metastatic cholangiocarcinoma by sequencing tumor samples and comparing with normal cells. Using tandem minigene constructs encoding all of the mutations expressed by a pati...
Source: NIH OTT Licensing Opportunities - February 3, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

A Viral Exposure Signature to Define and Detect Early Onset Hepatocellular Carcinoma
Early detection of liver cancer, such as hepatocellular carcinoma (HCC), is key to improve cancer-related mortality. More than 800,000 people are diagnosed with this cancer each year throughout the world. Liver cancer is also a leading cause of cancer deaths worldwide, accounting for more than 700,000 deaths each year. Currently, millions of Americans and possibly billions in the world are considered at risk for developing liver cancer. Individuals are considered at risk for developing liver cancer if they have underlying chronic liver diseases such as fibrosis and cirrhosis which in turn may be caused by viral infections ...
Source: NIH OTT Licensing Opportunities - January 15, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Parental A2780 Ovarian Cancer Cell Line and Derivative Cisplatin-resistant and Adriamycin-resistant A2780 Cell Lines
Ovarian cancer is one of the most common and lethal types of gynecological malignancies worldwide, accounting for approximately 295,000 new cases and 185,000 deaths annually. The high lethality rate is due to multiple reasons, including recurrence and the resistance of recurrent tumors to chemotherapy. Cell line models are crucial for preclinical cancer studies, to identify mechanisms of disease, to study drug resistance, and to screen for candidate therapeutics.  Researchers at the National Cancer Institute (NCI), Laboratory of Cellular and Molecular Biology have derived a cell line, A2780, from a patient with metast...
Source: NIH OTT Licensing Opportunities - January 15, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

A Preclinical Model for Mutant Human EGFR-driven Lung Adenocarcinoma
Previously described epidermal growth factor receptor- (EGFR) driven tumor mouse models develop diffuse tumors, which are dissimilar to human lung tumor morphology and difficult to measure by CT and MRI scans. Scientists at the National Cancer Institute (NCI) have developed and characterized a genetically engineered mouse (GEM) model of human EGFR-driven tumor model (hEGFR-TL) that recapitulates the discrete lung tumor nodules similar to those found in human lung tumor morphology. Individual tumor nodules can be easily measured by live animal imaging and the nodules can be harvested and isolated from surrounding lung tissu...
Source: NIH OTT Licensing Opportunities - January 15, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

A Preclinical Orthotopic Model for Glioblastoma Multiforme that Represents Key Pathways Aberrant in Human Brain Cancer
Current therapies for glioblastoma multiforme (GBM), the highest grade malignant brain tumor, are mostly ineffective, and better preclinical model systems are needed to increase the successful translation of drug discovery efforts into the clinic. Scientists at the National Cancer Institute (NCI) have developed and characterized an orthotopic genetically engineered mouse (GEM)-derived model of GBM that closely recapitulates various human GBM subtypes and is useful for preclinical evaluation of candidate therapeutics. The GEM-derived GBM model harbors perturbations in the receptor tyrosine kinase (RTK), phosphoinositide 3-k...
Source: NIH OTT Licensing Opportunities - January 15, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Molecular Classification of Primary Mediastinal Large B Cell Lymphoma Using Formalin-Fixed, Paraffin-Embedded Tissue Specimens
Primary mediastinal B-cell lymphoma (PMBCL) is an aggressive type of non-Hodgkin lymphoma that mostly occurs in people between the ages of 30-40. It accounts for 5-7% of all aggressive lymphomas. The diagnosis of PMBCL is challenging as the histological features of PMBCL overlap with diffuse large B-cell lymphoma (DLBCL), another most common type of non-Hodgkin lymphoma. Available evidence suggests that PMBCL responds much more favorably to the DA-EPOCH-R chemotherapy regimen than to the standard R-CHOP regimen used to treat DLBCL. The diagnostic uncertainty of PMBCL can result in delayed and/or inappropriate treatment, se...
Source: NIH OTT Licensing Opportunities - January 15, 2020 Category: Research Authors: ajoyprabhu3 Source Type: research

Engineered T Cells that Target Tumor-Specific Mutations
Scientists at the National Cancer Institute'sSurgery Branch have developed a method to identify and generate T-cell receptor (TCR) engineered T- cells for personalized cancer therapy. The TCR is a complex of integral membrane proteins that recognizes antigens and activates T cells. Human cancers contain genetic mutations that are unique in each patient. The researchers found cancer-specific mutations by sequencing tumors and comparing with normal cells. Using tandem minigene constructs encoding all of the patient's tumor mutations, they first identified T cells that were reactive with the unique mutated antigens expressed ...
Source: NIH OTT Licensing Opportunities - December 14, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

Nanoparticle delivery of lung cancer therapeutic
Non-small cell lung cancer (NSCLC) is the leading cause of cancer-related deaths in developed countries.   Despite the availability of several synergistic, targeted therapy regiments, the 5-year survival rate for NSCLC is only 15%.  The poor prognosis of NSCLS is due in part to limitations of current treatments, which do not trigger an immune response against NSCLC, nor can they be directly delivere d into the lungs.  Researchers at NCI ’sLaboratory of Experimental Immunologyhave developed a novel method for synthesizing polyketal nanoparticles with adsorbed CpG oligonucleotides (NP-CpG) that poss...
Source: NIH OTT Licensing Opportunities - December 14, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

Broadly Protective Influenza Vaccine Comprising a Cocktail of Inactivated Avian Influenza Viruses
There is a great need for broadly protective, “universal” influenza virus vaccines given the antigenic drift and shift of influenza viruses and the variable protective efficacy of the current influenza vaccines. This technology relates to a broadly protective, “universal” influenza vaccine candidate composed of a cocktail of different l ow pathogenicity avian influenza virus subtypes inactivated by betapropiolactone (BPL). Vaccinating animals with BPL-inactivated whole virus vaccine comprising influenza virus strains belonging to four or more different low pathogenicity avian influenza hemagglutinin...
Source: NIH OTT Licensing Opportunities - December 5, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

A High-Yield Perfusion-Based Transient Gene Expression Bioprocess
Currently, fed-batch processes are the most commonly used bioprocesses in transient gene expression (TGE) vaccine manufacturing. However, because fed-batch processes keep all the cells and protein product in the vessel throughout the run, some limitations are intrinsic. First, waste products like cell debris or other unwanted small molecules accumulate in the vessel with a potential to disrupt the cell growth, protein production, and the stability of the generated protein of interest. Second, necessary buffer exchange and/or cell concentration steps must be performed outside of the culturing vessel. These steps are more in...
Source: NIH OTT Licensing Opportunities - December 2, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

T-Cell Therapy Against Patient-Specific Cancer Mutations
Human cancers contain genetic mutations that are unique to each patient. Some of the mutated peptides are immunogenic, can be recognized by T cells, and therefore, may serve as therapeutic targets.Scientists at the National Cancer Institute'sSurgery Branch developed a method to identify T cells that specifically recognize immunogenic mutations expressed only by cancer cells. The scientists identified cancer-specific mutations from a patient with widely metastatic cholangiocarcinoma by sequencing tumor samples and comparing with normal cells. Using tandem minigene constructs encoding all of the mutations expressed by a pati...
Source: NIH OTT Licensing Opportunities - November 22, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

Engineered T Cells that Target Tumor-Specific Mutations
Scientists at the National Cancer Institute'sSurgery Branch have developed a method to identify and generate T-cell receptor (TCR) engineered T- cells for personalized cancer therapy. The TCR is a complex of integral membrane proteins that recognizes antigens and activates T cells. Human cancers contain genetic mutations that are unique in each patient. The researchers found cancer-specific mutations by sequencing tumors and comparing with normal cells. Using tandem minigene constructs encoding all of the patient's tumor mutations, they first identified T cells that were reactive with the unique mutated antigens expressed ...
Source: NIH OTT Licensing Opportunities - November 22, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

Design and Biological Activity of Novel Stealth Polymeric Lipid Nanoparticles for Enhanced Delivery of Hydrophobic Photodynamic Therapy Drugs
Nanoparticles such as lipid-based nanoparticles (LNPs) represent a relatively new era of targeted drug delivery systems wherein these biocompatible particles can carry the drug(s) of interest to a specific tumor site. The new generation of nanoparticles, known as stealth nanoparticles, are engineered to have a coating of polyethylene glycol polymer (PEG) or other glycolipids that enable them to evade the immune system and have a longer circulation lifespan as well as improved bioavailability to diseased tissue and reduced non-specific toxicity.  Scientists at the National Cancer Institute (NCI) have developed the for...
Source: NIH OTT Licensing Opportunities - November 19, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

Newly Improved Method and Composition for Treating Genetically Linked Diseases of the Eye
X-linked retinoschisis (XLRS) is an inherited ocular disease caused by mutations in the RS1 gene which results in the abnormal splitting of the retinal layers. It is the leading cause of macular degeneration in males, and approximately 1:15,000 individuals in the US are affected by this condition.   XLRS causes progressive vision loss, and affected individuals are unable to perform simple daily activities such as reading, writing and driving. This condition can lead to vitreous hemorrhage and retinal detachment in up to 40% of patients – resulting in total blindness.A current treatment option includes a tissue-s...
Source: NIH OTT Licensing Opportunities - November 19, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

Improvement of Broadly HIV-Neutralizing Antibodies; Anti-HIV-1 Antibody VRC01.23 for Prevention or Treatment of HIV Infection
Scientists at NIAID have developed broadly neutralizing antibodies (bNAbs) with enhanced neutralizing activity against HIV-1. Specifically, previously unknown gp120 interactions with a newly elucidated quaternary receptor (CD4)-binding site in the HIV-1 envelope have been discovered by engrafting the extended heavy-chain framework region 3 (FR3) loop of VRC03 onto several potent bNAbs (including VRC01, VRC07 and N6). The new antibodies show improved binding with CD4 by interacting with both binding sites and as a result show improved neutralization of various HIV-1 strains. Furthermore, they show reduced autoreactivity and...
Source: NIH OTT Licensing Opportunities - September 25, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

Biofluorescent Colon Cancer Cell Line for Evaluation of Anti-Cancer Agents
Colorectal cancer (cancer that starts in the colon or rectum) is the third most commonly diagnosed cancer and the second leading cause of cancer death in the United States. There is a significant unmet need for improved therapies against this deadly disease – despite early detection increasing survival rates. Mouse models resembling the biological situation are necessary in the evaluation of chemotherapeutic or immunotherapeutic agents against colon cancer. The murine colon adenocarcinoma cell line, MC-38, was used to create the MC-38 cell line-derived xenograft model, which is widely used to study the prolifera...
Source: NIH OTT Licensing Opportunities - September 18, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

In vitro Generation of an Autologous Thymic Organoid from Human Pluripotent Stem Cells
The thymus is an integral part of the adaptive immune system as it generates T cells. Its function diminishes rapidly as the body ages, leading to a compromise of the immune system in the elderly. Reconstitution of adaptive immunity through mass production of different T cell types is therefore a therapeutic need in immunocompromised populations. Furthermore, production of T cells with specific receptors targeting cancer cells is an important cancer immunotherapy approach. However, current in vitro methods are not efficient in producing conventional CD4 or CD8 positive T cells, as they result in production of immature, CD4...
Source: NIH OTT Licensing Opportunities - September 18, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

Genetically Engineered Myeloid Cells (GEMys) as a Platform to Enhance Anti-Tumor Immunity
Cancer cells can spread to various regions in the body in a process called metastasis, which is associated with difficulty in treatment and thus reduced survival. Identifying metastatic biomarkers have been a major concern in the field of cancer diagnosis and therapy. Interestingly, research has shown that there is an increase in myeloid progenitors and myeloid cells at various stages of metastasis in tumors and tissues, as the immune system attempts to suppress cancer cells. This presents a unique opportunity for cancer immunotherapy.Researchers at National Cancer Institute (NCI) developed a platform to culture myeloid ce...
Source: NIH OTT Licensing Opportunities - September 18, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

Efficient Methods to Prepare Hematopoietic Progenitor Cells in vitro for Therapeutic Use
Hematopoietic progenitor cells (HPC) are multi-potent hematopoietic lineage cells that can differentiate into any type of blood cell, including but not limited to erythrocytes, T cells, B cells, and natural killer cells. As such, they have high therapeutic potential in the fields of regenerative medicine and cancer immunotherapy, especially when generated from patient-derived induced pluripotent stem cells (iPSC). Currently, the most efficient protocol to produce HPCs is co-culturing human iPSCs (hiPSC) with mouse stromal cells as a two-dimensional (2D) monolayer. However, animal cells are not suitable for clinical applica...
Source: NIH OTT Licensing Opportunities - September 18, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

A Novel Transgenic Zebrafish Line Reporting Dynamic Epigenetic Changes
Currently, there is no other whole-animal reporter for epigenetic regulation established in any vertebrate.  The inventors generated this novel zebrafish line using a transgene construct containing dazl gene silencing sequences (CpG island) fused to a destabilized GFPd2 gene driven by the ubiquitously expressing ef1alpha promoter. The resulting transgenic line permits detailed tissue- or cellular- level visualization of dynamic changes in GFPd2 expression in response to changes in DNA methylation or downstream epigenetic regulation in developing or adult animals. GFPd2 is “off” in the fertilized egg but tu...
Source: NIH OTT Licensing Opportunities - September 18, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

T Cell Receptors (TCRs) Specific for Mutant p53
Mutations in tumor protein p53 are expressed in a variety of human cancers such as colon, pancreatic, breast, and non-small cell lung cancer. P53 acts as a tumor suppressor by regulating cell division and DNA repair. Mutations of p53 reduce or eliminate its regulatory functions, contributing to cancer formation and progression. Novel therapeutics are needed that specifically target p53 mutations, as small molecule inhibitors lack sequence specificity.T cell receptors (TCRs) are proteins expressed on the surface of T lymphocytes that can recognize peptide antigens from infected and malignant cells in the context of human le...
Source: NIH OTT Licensing Opportunities - September 17, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research

Floxed Targeted Mouse Strain for Use in Conditional Deletion of the Irf8 Gene
IRF8, a member of interferon regulatory factor (IRF) family of transcription factors is a novel intrinsic transcriptional inhibitor of TH17-cell differentiation. TH17-cells are believed to be involved in the pathogenesis of various autoimmune/inflammatory diseases. These materials could be used to help define patterns of gene expression important for the development and function of cells including possible contributions to understanding: normal immune responses, inflammatory conditions, autoimmunity and anti-viral responses.IC: NIAIDNIH Ref. No.: E-062-2012-0Advantages:  Mice with established germ line trans...
Source: NIH OTT Licensing Opportunities - August 27, 2019 Category: Research Authors: ajoyprabhu3 Source Type: research