| Genzyme Corporation Patent applications |
| Patent application number | Title | Published |
|---|
| 20120121704 | Aliphatic Amine Polymer Salts for Tableting - The tablets, compositions and methods of the present invention, comprising a carbonate salt of an aliphatic amine polymer and s monovalent anion can prevent or ameliorate acidosis, in particular acidosis in patients with renal disease. The tablets and compositions of the present invention maintain a disintegration time of no greater than 30 minutes at 37° C. and at pH of at least 1 for a period of at least ten weeks at 60° C. Furthermore, the tablets are stable for extended periods of time without the need for specialized storage conditions. | 05-17-2012 |
| 20120115926 | ANTISENSE MODULATION OF APOLIPOPROTEIN B EXPRESSION - Methods for the rapid and long-term lowering of lipid levels in human subjects and for the treatment of conditions associated with elevated LDL-cholesterol and elevated apolipoprotein B are provided. | 05-10-2012 |
| 20120100152 | ANTI-HUMAN CD52 IMMUNOGLOBULINS - The present invention relates to humanized immunoglobulins, mouse monoclonal antibodies and chimeric antibodies that have binding specificity for human CD52. The present invention further relates to a humanized immunoglobulin light chain and a humanized immunoglobulin heavy chain. The invention also relates to isolated nucleic acids, recombinant vectors and host cells that comprise a sequence which encodes a humanized immunoglobulin or immunoglobulin light chain or heavy chain, and to a method of preparing a humanized immunoglobulin. The humanized immunoglobulins can be used in therapeutic applications to treat, for example, autoimmune disease, cancer, non-Hodgkin's lymphoma, multiple sclerosis and chronic lymphocytic leukemia. | 04-26-2012 |
| 20120088721 | Cartilage Repair - This invention relates to compositions, methods of preparation thereof, and use thereof for cartilage repair. | 04-12-2012 |
| 20120070408 | METHODS AND COMPOSITIONS FOR TREATING LUPUS - The invention provides methods of treating lupus in a patient with an anti-CD52 antibody. Also includes are methods of increasing infiltration of regulatory T cells to affected sides of the patient's body, methods of reducing urine protein and/or albumin levels and methods of depleting lymphocytes to alleviate lupus symptoms. | 03-22-2012 |
| 20120064147 | AGENT FOR IMPROVING TISSUE PENETRATION - The invention concerns a pharmaceutical preparation which improves penetration of active substances through the tissue membrane or barrier of the target organ. | 03-15-2012 |
| 20120058082 | METHODS AND COMPOSITIONS FOR TREATMENT - The invention provides methods for improving the efficacy and reducing side effects of anti-CD52 antibody treatment. The methods can be used to treat patients who are in need of immunoregulation such as lymphocyte depletion and patients who have cancer. Also included are compositions useful for these methods. | 03-08-2012 |
| 20120027805 | Controlled-Released Peptide Formulations - Described herein are methods and compositions for modulating the release and/or drug loading characteristics of encapsulated bioactive agents in polymer-based delivery systems via direct modification of the isoelectric point and/or net charge of the bioactive agent. | 02-02-2012 |
| 20120022126 | Method Of Treating Diabetes Mellitus - The invention provides methods of treating a diabetic subject comprising administering a glucosylceramide synthase inhibitor to the subject. | 01-26-2012 |
| 20120004235 | METHODS TO TREAT CANCER - The invention provides methods and pharmaceutical compositions for treating certain cancers with compounds of formula (I) wherein A, B, W, Y, Z, and R | 01-05-2012 |
| 20110319469 | SNPs of Apolipoprotein B and Modulation of Their Expression - Compounds, compositions and methods are provided for modulating the expression of apolipoprotein B. The compositions comprise oligonucleotides, targeted to nucleic acid encoding apolipoprotein B. Methods of using these compounds for modulation of apolipoprotein B expression and for diagnosis and treatment of diseases and conditions associated with expression of apolipoprotein B are provided. | 12-29-2011 |
| 20110262516 | AUTOLOGOUS CELLS ON A SUPPORT MATRIX FOR TISSUE REPAIR - The present invention relates to a method for the effective treatment of tissue defects and for tissue regeneration. The method includes seeding autologous cells on a support matrix and implanting the cell-seeded support matrix into a site of transplantation. The present invention also relates to various tissue repair structures that include cells seeded onto a cell-free membrane. The present invention also provides methods for cultivation, seeding, and implantation of autologous cells. | 10-27-2011 |
| 20110262350 | BREAST ENDOTHELIAL CELL EXPRESSION PATTERNS - To gain a better understanding of breast tumor angiogenesis, breast endothelial cells (ECs) were isolated and evaluated for gene expression patterns. When transcripts from breast ECs derived from normal and malignant breast tissues were compared, genes that were specifically elevated in tumor-associated breast endothelium were revealed. These results confirm that neoplastic and normal endothelium in human breast are distinct at the molecular level, and have significant implications for the development of anti-angiogenic therapies in the future. | 10-27-2011 |
| 20110245265 | CXCR4 ANTAGONISTS FOR KIDNEY INJURY - Methods to treat or prevent acute kidney injury and chronic kidney injury in subjects using CXCR4 antagonists are disclosed. | 10-06-2011 |
| 20110212086 | GITR Antibodies For The Treatment of Cancer - The present invention provides compositions and methods for inhibiting the growth of a GITR-expressing cancer cell which cells may include, but are not limited to cells of epithelial origin such as NSCLC, prostate cancer, breast cancer, colon cancer and ovarian cancer and to treat or ameliorate the symptoms associated with the presence of these cells in a subject. Suitable compositions for use in these methods are antibodies that selectively recognize and bind to GITR (Glucocorticoid-induced TNFR-related protein) present on these cancer cells. The antibodies can be either polyclonal or monoclonal antibodies. | 09-01-2011 |
| 20110183936 | REGIMENS FOR INTRA-ARTICULAR VISCOSUPPLEMENTATION - The invention provides viscosupplementation methods for treating osteoarthritis and joint injury with HA-based viscosupplements, particularly viscosupplements with an intra-articular residence half-life shorter than 3 weeks. Viscosupplements for use in the methods of the invention may be further characterized in that they contain less than 20 mg/ml HA, at least 5% (w/w) of which is in a gel form, such as, e.g., hylan B. In an illustrative embodiment, hylan G-F 20 (Synvisc®) is administered in a single intra-articular knee injection of 6±2 ml. | 07-28-2011 |
| 20110142818 | COMBINATION ENZYME REPLACEMENT AND SMALL MOLECULE THERAPY FOR TREATMENT OF LYSOSOMAL STORAGE DISEASES - This invention provides various combinations of enzyme replacement therapy, gene therapy, and small molecule therapy for the treatment of lysosomal storage diseases. | 06-16-2011 |
| 20110129448 | METHODS TO MOBILIZE PROGENITOR/STEM CELLS - Methods to elevate progenitor and stem cell counts in animal subjects using compounds which bind to the chemokine receptor CXCR4 are disclosed. Preferred embodiments of such compounds are of the formula | 06-02-2011 |
| 20110118145 | COPY NUMBER ANALYSIS OF GENETIC LOCUS - Systems and methods for analyzing copy number of a target locus, detecting a disease associated with abnormal copy number of a target gene or a carrier thereof. | 05-19-2011 |
| 20110104117 | METABOLICALLY ACTIVATED RECOMBINANT VIRAL VECTORS AND METHODS FOR THEIR PREPARATION AND USE - Recombinant viral vectors, especially parvovirus vectors such as adeno-associated virus (AAV) vectors; capable of enhanced expression of heterologous sequences, and methods for their construction and use, are provided. The vectors have a structure, or are capable of rapidly adopting a structure, which involves intrastrand base pairing of at least one region in a heterologous sequence. | 05-05-2011 |
| 20110091892 | Methods for the Diagnosis and Treatment of Lung Cancer - The present invention provides methods for aiding in the diagnoses of the neoplastic condition of a lung cell and methods of screening for a potential therapeutic agent for the reversal of the neoplastic condition. | 04-21-2011 |
| 20110052585 | COMPOSITIONS AND METHODS FOR INHIBITING INTERLEUKIN PATHWAYS - Fusion proteins including an IL-17 receptor with a multimerization domain, or an IL-23 receptor and a multimerization domain, and recombinant viral vectors encoding such fusions, are described. The fusion proteins and vectors encoding such fusions, alone or in combination, can be used in methods for modulating the IL-17 and IL-23 signaling pathways and for treating or preventing diseases mediated by interleukin-17 and interleukin-23, such as immune-related and inflammatory diseases. | 03-03-2011 |
| 20110034546 | METHODS TO PRODUCE ROD-DERIVED CONE VIABILITY FACTOR (RDCVF) - The invention is related to methods of producing rod-derived cone viability factor (RdCVF). This invention also relates to the treatment of an ocular disease in a mammal using RdCVF. Also provided are expression vectors for high secreted expression of RdCVF of using nucleotide sequences encoding heterologous signal proteins and optionally markers for furin cleavage. | 02-10-2011 |
| 20110008364 | ANTIBODIES TO TGF-BETA - The present invention relates to antibody molecules, in particular antibody molecules that bind Transforming Growth Factor beta (TGFβ), and uses thereof. More particularly, the invention relates to antibody molecules that bind and preferably neutralise TGFβ1, TGFβ2 and TGFβ3, so-called “pan-specific” antibody molecules, and uses of such antibody molecules. Preferred embodiments within the present invention are antibody molecules, whether whole antibody (e.g. IgG, such as IgG1 or IgG4) or antibody fragments (e.g. scFv, Fab, dAb). | 01-13-2011 |
| 20110008296 | MUSCLE-DERIVED CELLS HAVING DIFFERENTIATION CAPACITIES - The present application relates to cell populations having differentiation capacities, which are obtainable by isolation from a muscle tissue, more particularly from a skeletal and/or cardiac muscle tissue, preferably from endomysial and/or cardiac tissue, more preferably from endomysial tissue. The cell populations of the invention comprise ALDH-positive cells, and notably have myogenic and/or adipogenic and/or osteogenic differentiation capacities. | 01-13-2011 |
| 20100331390 | EFFECTS OF APOLIPOPROTEIN B INHIBITION ON GENE EXPRESSION PROFILES IN ANIMALS - Methods are provided for modulating the expression of genes involved in lipid metabolism, useful in the treatment of conditions associated with cardiovascular risk. Antisense oligonucleotides targeted to apolipoprotein B reduce the level of apolipoprotein B mRNA, lower serum cholesterol and shift liver gene expression profiles from those of an obese animal towards those of a lean animal. Further provided are methods for improving the cardiovascular risk of a subject through antisense inhibition of apolipoprotein B. Also provided are methods for employing antisense oligonucleotides targeted to apolipoprotein B to modulate a cellular pathway or metabolic process. | 12-30-2010 |
| 20100298366 | CHEMOKINE RECEPTOR BINDING COMPOUNDS - The present invention relates to chemokine receptor binding compounds, pharmaceutical compositions and their use. More specifically, the present invention relates to modulators of chemokine receptor activity, preferably modulators of CCR5. These compounds demonstrate protective effects against infection of target cells by a human immunodeficiency virus (HIV). | 11-25-2010 |
| 20100297105 | ADMINISTERING ANTISENSE OLIGONUCLEOTIDES COMPLEMENTARY TO HUMAN APOLIPOPROTEIN B - Methods for long-term lowering of lipid levels in human subjects and for the treatment of conditions associated with elevated LDL-cholesterol and elevated ApoB are provided. | 11-25-2010 |
| 20100267812 | GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS - This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. | 10-21-2010 |
| 20100266526 | Combination therapy for treating hypercholesterolemia - The invention relates to methods for treating hypercholesterolemia and atherosclerosis, and reducing serum cholesterol in a mammal. The methods of the invention comprise administering to a mammal a first amount of a bile acid sequestrant compound which is an unsubstituted polydiallylamine polymer and a second amount of a cholesterol-lowering agent. The first and second amounts together comprise a therapeutically effective amount. | 10-21-2010 |
| 20100254935 | Amine condensation polymers as phosphate sequestrants - Disclosed is a polymer or physiologically acceptable salt thereof. The polymer comprises a polymerized multifunctional amine monomer. The amine monomer comprises at least two amine groups and at least two acyclic nitrogen atoms that are connected through a —CH | 10-07-2010 |
| 20100248252 | METHODS FOR ANALYSIS OF MOLECULAR EVENTS - Methods and compositions are provided for detecting the presence of nucleic acid sequence variants in a subpopulation of nucleic acid molecules in a biological sample. These methods are particularly useful for identifying individuals with mutations indicative of cancer. | 09-30-2010 |
| 20100234309 | Dendrimer Compositions - Amine compounds, amine polymers, crosslinked amine polymers and pharmaceutical compositions comprising the same may include polyhydroxy-containing cores that may be substituted with amine groups and may be used to treat hyperphosphatemia or to remove ions from the gastrointestinal tract of animals, including humans. | 09-16-2010 |
| 20100233158 | INDUCTION OF IMMUNE TOLERANCE - Methods of inducing immune tolerance by administering an immunosuppressive agent and a compound represented by Formula (I) are disclosed: | 09-16-2010 |
| 20100216709 | SYSTEMIC INSULIN-LIKE GROWTH FACTOR-1 THERAPY REDUCES DIABETIC PERIPHERAL NEUROPATHY AND IMPROVES RENAL FUNCTION IN DIABETIC NEPHROPATHY - The present invention provides methods of treatment of patients suffering from the complications of blood sugar disorders: diabetic peripheral neuropathy and diabetic nephropathy by administration of IGF-1 via protein therapy or gene therapy. It relates to methods of treating an individual having a diabetic disorder or a hyperglycemic disorder, comprising administering to the individual an effective amount of a DNA vector expressing IGF-1Eb or IGF-1Ec in vivo or an effective amount of at the IGF-1Eb or IGF-1Ec protein in the early hyperalgesia stage or in patients that have advanced to the hyposensitivity stage. Treatment at the early hyperalgesia stage prevents subsequent hyposensitivity with increases or maintenance of sensory nerve function. IGF-1Eb or IGF-1Ec treatment also increases muscle mass and improves overall mobility, which indicates a treatment-related improvement in motor function. Treatment with IGF-1Eb or IGF-1Ec at the hyposensitivity stage reverses hyposensitivity and improves muscle mass and overall health. Systemic IGF-1 provides a therapeutic modality for treating hyposensitivity associated with DPN. In addition, IGF-1Eb or IGF-1Ec provides a therapeutic modality for treating diabetic nephropathy. IGF-1Eb or IGF-1Ec improves renal function as evidenced by a modulation in serum albumin concentration and a reduction in urine volume and protein levels. IGF-1Eb or IGF-1Ec also reduces diabetic glomerulosclerosis. | 08-26-2010 |
| 20100178271 | Combination Therapy - Methods to mobilize progenitor and/or stem cells from the bone marrow to the bloodstream by administering a combination of at least one CXCR | 07-15-2010 |
| 20100173979 | GENE THERAPY FOR LYSOSOMAL STORAGE DISEASES - This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease. | 07-08-2010 |
| 20100173320 | Methods for Detecting Nucleic Acids Indicative of Cancer - The invention provides methods for screening tissue or body fluid samples for nucleic acid indicia of cancer or precancer. | 07-08-2010 |
| 20100172861 | Anionic Polymers as Toxin Binders and Antibacterial Agents - The present invention relates to a method of inhibiting a toxin in an animal, such as a human, by administering to the animal a therapeutically effective amount of a polymer having a plurality of pendant acid functional groups which are directly attached to the polymer backbone or attached to the polymer backbone by a spacer group. The spacer group can have a length in the range from 0 to about 20 atoms. The toxin is, typically, an exotoxin secreted by a pathogenic microorganism, such as a bacterium. | 07-08-2010 |
| 20100151478 | METHODS FOR DISEASE DETECTION - The present invention provides methods for detecting disease by analysis of a patient sample to determine the integrity of nucleic acids in the sample. | 06-17-2010 |
| 20100143297 | METHODS OF ENHANCING LYSOSOMAL STORAGE DISEASE THERAPY BY MODULATION OF CELL SURFACE RECEPTOR DENSITY - Methods of modulating uptake of extracellular lysosomal enzymes by administering a pharmaceutical agent and methods of treating a lysosomal storage disease (such as Gaucher disease, Pompe disease, Fabry disease or Niemann-Pick disease) or enhancing enzyme replacement therapy or gene therapy, comprising administering a pharmaceutical agent such as dexamethasone, glucose or insulin, are provided. | 06-10-2010 |
| 20100135950 | Iron(II)-Containing Treatments for Hyperphosphatemia - A pharmaceutical composition comprises a pharmaceutically acceptable ferrous iron compound; an amine polymer or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier. Alternatively, a pharmaceutical composition comprises a pharmaceutically acceptable ferrous iron compound and pharmaceutically acceptable carrier, wherein the ferrous iron compound is selected from the group consisting of iron(II) acetate, iron(II) citrate, iron(II) ascorbate, iron(II) oxalate, iron(II) oxide, iron(II) carbonate, iron(II) carbonate saccharated, iron(II) formate, iron(II) sulfate, iron(II) chloride, iron(II) acetylacetonate and combinations thereof. A method of treating a subject with hyperphosphatemia comprises administering to the subject an effective amount of a pharmaceutical composition as described above. | 06-03-2010 |
| 20100124542 | AMINE DENDRIMERS - Ion binding compounds and compositions may include compounds, polymers and compositions that include amine moieties. Ion binding polymers may be crosslinked amine polymers and may be used to remove ions, such as phosphate ions, from the gastrointestinal tract of animals, such as humans. Such compounds, polymers and compositions may be used therapeutically to treat a variety of medical conditions, such as hyperphosphatemia. | 05-20-2010 |
| 20100104537 | Methods of treating Parkinson's disease using viral vectors - Methods of delivering viral vectors, particularly recombinant AAV virions, to the CNS are provided. Also provided are methods of treating Parkinson's Disease. | 04-29-2010 |
| 20100093857 | AMIDO-AMINE POLYMER COMPOSITIONS - Compounds, polymers, crosslinked polymers and pharmaceutical compositions comprising the same may be derived from multi-amine monomers and multi-functional monomers having two or more amine reactive groups. Such compounds, polymers, crosslinked polymers and compositions may be used to treat hyperphosphatemia or to remove ions from the gastrointestinal tract of animals, including humans. | 04-15-2010 |
| 20100092981 | METHODS OF DETECTING HYPERMETHYLATION - Aspects of the invention relate to methods of detecting cancer, such as colon cancer. In aspects of the invention, methods for detecting the presence of hypermethylated genomic DNA in a biological sample are disclosed. Methods of the invention relate to detecting small amounts of hypermethylated nucleic acids in a biological sample. | 04-15-2010 |
| 20100083523 | Method of Drying A Material Having A Cohesive Phase - A method for drying a material such as a polymer hydrogel which passes through a cohesive phase as it dries is disclosed. The method comprises agitating a composition while removing liquid until the solids content of the composition reaches a level at which the composition enters a cohesive phase, halting agitation, removing liquid from the composition in the absence of agitation, and resuming agitation. Practice of the present invention can eliminate the problems associated with adhesion of a material to itself and to process equipment during the cohesive phase. | 04-08-2010 |
| 20100069500 | AMIDE DENDRIMER COMPOSITIONS - Amide compounds, amide polymers, compositions including amide compounds and amide polymers may be used to bind target ions, such as phosphorous-containing compounds in the gastrointestinal tract of animals. In some cases, amide compounds and amide polymers may include a core derived from an amide polyol and an organic polyacid or ester. | 03-18-2010 |
| 20100068167 | Low Salt Forms of Polyallylamine - Disclosed is a pharmaceutical composition comprising a stable polyallylamine hydrochloride polymer in which between about 4% to about 12% by weight of the polymer is a chloride anion and a pharmaceutically acceptable carrier or diluent. | 03-18-2010 |
| 20100062443 | Methods for the Diagnosis and Treatment of Lung Cancer - The present invention provides methods for aiding in the diagnoses of the neoplastic condition of a lung cell and methods of screening for a potential therapeutic agent for the reversal of the neoplastic condition. | 03-11-2010 |
| 20100062002 | Brain Endothelial Cell Expression Patterns - To gain a better understanding of brain tumor angiogenesis, new techniques for isolating brain endothelial cells (ECs) and evaluating gene expression patterns were developed. When transcripts from brain ECS derived from normal and malignant colorectal tissues were compared with transcripts from non-endothelial cells, genes predominantly expressed in the endothelium were identified. Comparison between normal- and tumor-derived endothelium revealed genes that were specifically elevated in tumor-associated brain endothelium. These results confirm that neoplastic and normal endothelium in human brains are distinct at the molecular level, and have significant implications for the development of anti-angiogenic therapies in the future. | 03-11-2010 |
| 20100047225 | OLIGOSACCHARIDES COMPRISING AN AMINOOXY GROUP AND CONJUGATES THEREOF - The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group. The invention further provides oligosaccharides comprising an aminooxy group, methods for coupling oligosaccharides comprising an aminooxy group to glycoproteins, and oligosaccharide-protein conjugates. Also provided are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-protein conjugate. | 02-25-2010 |
| 20100041151 | Compositions and methods for treating lysosomal storage disease - The present invention provides recombinant viral and non-viral vectors comprising a transgene encoding a biologically active human lysosomal enzyme that are able to infect and/or transfect and sustain expression of the biologically active human lysosomal enzyme transgene in mammalian cells deficient therein. In addition, methods are provided for providing a biologically active human lysosomal enzyme to cells deficient therein, which comprises introducing into the cells a vector comprising and expressing a transgene encoding the biologically active human lysosomal enzyme, wherein the vector is taken up by the cells, the transgene is expressed and biologically active enzyme is produced. The cells may be infected and/or transfected by the vector, dependent upon whether the vector is a viral vector and/or plasmid or the like. The invention also provides a method of supplying a biologically active human lysosomal enzyme to other distant cells deficient therein wherein the transfected and/or infected cells harboring the vector secrete the biologically active enzyme which is then taken up by the other deficient cells. In a preferred embodiment the present invention provides for sustained production of biologically human active α-galactosidase A in cells of Fabry individuals that are deficient in said enzyme. | 02-18-2010 |
| 20100003224 | Combination Therapy - Methods to mobilize progenitor and/or stem cells from the bone marrow to the bloodstream by administering a combination of at least one CXCR4 inhibitor and at least one VLA-4 inhibitor are described. The combinations may also be used to treat multiple myeloma. | 01-07-2010 |
| 20090325206 | Method for assaying the activity of lysosomal enzymes - A method, and associated kit, for assaying the activity of lysosomal enzymes present in dried bodily fluids and cell tissue samples, such as α-L-iduronidase, β-D-galactosidase, β-D-glucosidase, chitotriosidase, total α-D-galactosidase and α-D-galactosidase A, hexosaminidase A and B, α-D-mannosidase, β-D-mannosidase, α-L-fucosidase, N-acetyl-α-galactosaminidase, arylsulfatases, sphingomyelinase, β-galactocerebrosidase, iduronate- | 12-31-2009 |
| 20090324609 | METHOD OF TREATING AUTOIMMUNE DISEASE WITH MESENCHYMAL STEM CELLS - Methods and compositions for treating an autoimmune disease, such as new onset type 1 diabetes (T1D) in a subject using autologous or allogeneic mesenchymal stem cells administered to the subject prior to autoimmune-induced complete depletion of insulin-producing pancreatic beta cells, e.g., within six months of new onset type 1 diabetes (T1D) diagnosis or prior to the onset of disease in a subject determined to be at high risk for T1D. | 12-31-2009 |
| 20090312440 | AGENT FOR IMPROVING TISSUE PENETRATION - The invention concerns a pharmaceutical preparation which improves penetration of active substances through the tissue membrane or barrier of the target organ. | 12-17-2009 |
| 20090304623 | Once A Day Formulation for Phosphate Binders - A method for reducing serum phosphate in a subject in need thereof comprising administering once per day to said subject a phosphate binder, wherein the phosphate binder has a phosphate binding capacity of at least 52 mmole. | 12-10-2009 |
| 20090291135 | Direct compression polymer tablet core - The present invention provides a tablet core which comprises at least about 95% by weight of an aliphatic amine polymer. The invention also provides a method of producing a tablet core comprising at least about 95% by weight of an aliphatic amine polymer resin The method comprises the step of compressing the aliphatic amine polymer to form the tablet core. The tablet core can further include one or more excipients. In this embodiment, the method of producing the tablet core comprises the steps of: (1) hydrating the aliphatic amine polymer to the desired moisture level; (2) blending the aliphatic amine polymer with the excipients in amounts such that the polymer comprises at least about 95% by weight of the resulting blend; and (3) compressing the blend to form the tablet core. The present invention further relates to a coated tablet comprising an aliphatic amine polymer core wherein the coating is a water based coating. | 11-26-2009 |
| 20090286857 | GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS - This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant viral vector containing the transgene to the spinal cord. The viral vector delivers the transgene which expresses the encoded recombinant viral gene product. The viral gene product comprises HIF1-alpha. Also provided are compositions for delivery of a transgene product to a subject's spinal cord. | 11-19-2009 |
| 20090280081 | JAK/STAT pathway inhibitors and uses thereof - The role of the JAK/STAT signal transduction pathway cellular mechanisms that lead to the onset and progression of degenerative joint diseases or disorders such as osteoarthritis (OA) is disclosed. Certain known effective OA therapeutics such as hymenialdisine, debromohymenialdisine, and its variants and derivatives are shown to function as JAK3-specific inhibitors, which downregulate steady state mRNA levels of key cellular components involved in cartilage degradation. Another JAK3-specific inhibitor, not previously known as an OA therapeutic, is shown to downregulate steady state mRNA levels of various cellular components involved in cartilage degradation in a manner identical to that of the known OA therapeutics. | 11-12-2009 |
| 20090270265 | Molecular Characteristics of Non-Small Cell Lung Cancer - We used hierarchical clustering to examine gene expression profiles generated by serial analysis of gene expression (SAGE) in a total of nine normal lung epithelial cells and non-small cell lung cancers (NSCLC). Separation of normal and tumor samples, as well as histopathological subtypes, was evident using the 3,921 most abundant transcript tags. This distinction remained when just 115 highly differentially expressed transcript tags were used. Furthermore, these 115 transcript tags clustered into groups that were suggestive of the unique biological and pathological features of the different tissues examined. Adenocarcinomas were characterized by high-level expression of small airway-associated or immunologically related proteins, while squamous cell carcinomas overexpressed genes involved in cellular detoxification or antioxidation. The messages of two p53-regulated genes, p21 | 10-29-2009 |
| 20090263389 | Use of TGF-Beta Antagonists to Treat or to Prevent Chronic Transplant Rejection - Effective use of a TGF-β antagonist to treat or to prevent loss of transplant function is described herein. Use of a TGF-β antagonist is demonstrated to effectively prevent loss of organ function in a host due to chronic rejection in which TGF-β-mediated fibroproliferation is a characteristic. Expression in situ of a TGF-β antagonist in the form of a recombinant receptor, i.e., TGF-β type III receptor (TGFBIIIR) showed prevention of bronchiolitis obliterans in comparison to untreated controls in a rat lung transplant model. This provides an effective method for preventing or inhibiting chronic rejection of transplant organs such as lung, kidney, liver and heart in vertebrate hosts including human hosts. | 10-22-2009 |
| 20090192079 | PROLONGED DELIVERY OF HEPARIN-BINDING GROWTH FACTORS FROM HEPARIN-DERIVATIZED COLLAGEN - The present invention relates to a heparin-derivatized collagen matrix comprising a fragment of heparin covalently linked to a collagen scaffold, wherein the fragment of heparin has molecular weight of less than about 15 kDa, and at least one heparin-binding growth factor (HBGF) or heparin-binding adeno-associated virus (HB-AAV) or a combination thereof and methods for promoting bone growth, bone repair, cartilage repair, bone development, neo-angiogensis, wound healing, tissue engraftment and muscle tissue regeneration and/or tissue augmentation comprising administering a heparin-derivatized collagen matrix that includes at least one heparin-binding growth factor or heparin-binding adeno-associated virus or a combination thereof. | 07-30-2009 |
| 20090155368 | Pharmaceutical compositions - The present invention relates to crosslinked polyamine particles and/or pharmaceutical compositions comprising, at least in part, crosslinked polyamine particles and aggregates of such particles (including cured aggregates of crosslinked polyamine particles). The compositions may be in the form of tablets comprising, for example, particles larger than 500 μm, and used for treating patients, for example, patients with hyperphosphatemia. | 06-18-2009 |
| 20090155201 | Alkylated poly(allylamine) polymers and methods of use - The invention relates to a method for removing bile salts from a patient in need thereof and compositions useful in the method. The method comprises administering to the patient a therapeutically effective amount of a salt of an alkylated and crosslinked polymer. The alkylated and crosslinked polymer salt comprises the reaction product of crosslinked polymers, or salts and copolymers thereof having amine containing repeat units, with at least one aliphatic alkylating agent. | 06-18-2009 |
| 20090130079 | INTRAVENTRICULAR ENZYME DELIVERY FOR LYSOSOMAL STORAGE DISEASES - Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs. | 05-21-2009 |
| 20090123451 | SLOW INTRAVENTRICULAR DELIVERY - Neurological diseases, including lysosomal storage diseases, can be successfully treated using intraventricular delivery of the therapeutic agents to bypass the blood-brain barrier. Similarly, diagnostic agents and anesthetic agents can be delivered to the brain in this manner. The administration can be performed slowly to achieve maximum effect. Such administration permits greater penetration of distal portions of the brain. | 05-14-2009 |
| 20090117156 | GENE THERAPY FOR NIEMANN-PICK DISEASE TYPE A - This disclosure pertains to methods and compositions for tolerizing a mammal's brain to exogenously administered acid sphingomyelinase polypeptide by first delivering an effective amount of a transgene encoding the polypeptide to the mammal's hepatic tissue and then administering an effective amount of the transgene to the mammal's central nervous system (CNS). | 05-07-2009 |
| 20090117038 | Breast Endothelial Cell Expression Patterns - To gain a better understanding of breast tumor angiogenesis, breast endothelial cells (ECs) were isolated and evaluated for gene expression patterns. When transcripts from breast ECs derived from normal and malignant breast tissues were compared, genes that were specifically elevated in tumor-associated breast endothelium were revealed. These results confirm that neoplastic and normal endothelium in human breast are distinct at the molecular level, and have significant implications for the development of anti-angiogenic therapies in the future. | 05-07-2009 |
| 20090105142 | TREATMENT WITH KALLIKREIN INHIBITORS - Methods, kits and compositions are disclosed that include a non-naturally occurring kallikrein inhibitor and an optional additional gout therapeutic for the treatment of gout, such as acute gout. | 04-23-2009 |
| 20090105141 | INTRAVENTRICULAR PROTEIN DELIVERY FOR AMYOTROPHIC LATERAL SCLEROSIS - Amyotrophic Lateral Sclerosis can be successfully treated using intraventricular delivery of a neurotrophic growth factor, IGF-1. The administration can be performed slowly to achieve maximum effect. Effects are seen on both sides of the blood-brain barrier, making this a delivery means for Amyotrophic Lateral Sclerosis which affects both brain and skeletal muscle. | 04-23-2009 |
| 20090075887 | Treatment with Kallikrein Inhibitors - Methods, kits and compositions are disclosed that include a non-naturally occurring kallikrein inhibitor and optionally a viscosupplement for the treatment of joint pathology. | 03-19-2009 |
| 20090069261 | GENE THERAPY FOR SPINAL CORD DISORDERS - This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain. | 03-12-2009 |
| 20080292593 | GENE THERAPY FOR SPINAL CORD DISORDERS - The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site. | 11-27-2008 |
| 20080248512 | Methods for detection of lysosomal storage disease - The present invention provides compositions for performing assays of enzyme activity associated with lysosomal storage diseases. The invention further provides methods for determining enzyme activity, and methods for the screening for lysosomal storage disease in an individual. | 10-09-2008 |
| 20080226658 | TARGETING OF GLYCOPROTEIN THERAPEUTICS - Methods of making ligand-decorated polymer conjugates of therapeutic glycoproteins are described. Improved targeting of glycoproteins to specific tissues is achieved by masking the natural carbohydrate and other surface determinants with high molecular weight polymers, such as, e.g., PEG, polysialic acid, etc., which in turn are decorated with target-specific ligands. In some embodiments, acid-labile linkages in such conjugates or rapidly degradable masking groups allow for the intracellular release of the polymer from the glycoprotein, for example, under conditions found in lysosomes. | 09-18-2008 |
| 20080199442 | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors - The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved. | 08-21-2008 |
