Patent application number | Description | Published |
20090156524 | Novel siRNAS and methods of use thereof - The invention relates to compounds, in particular siRNAs, which inhibit the expression of specific human genes. The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier. The present invention also provides a method of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions comprising administering to a subject in need of treatment for such disease or condition and/or symptom the compound or the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject. The invention also provides antibodies which inhibit specified human polypeptides and pharmaceutical compositions comprising one or more such antibodies. | 06-18-2009 |
20100222409 | COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF PRO-APOPTOTIC GENES - The invention relates to one or more inhibitors, in particular siRNA compounds, which down-regulate the expression of a pro-apoptotic gene selected from the group consisting of TP53; HTRA2; KEAP1; SHC1-SHC, ZNHIT1, LGALS3 and HI95. The invention also relates to a pharmaceutical composition comprising the compound, and a pharmaceutically acceptable carrier. The present invention further provides methods of treating a subject afflicted with a disease or a condition associated with those genes, comprising administering to the subject a pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject. | 09-02-2010 |
20100273854 | COMPOSITIONS AND METHODS FOR INHIBITING NADPH OXIDASE EXPRESSION - One or more inhibitors, in particular siRNAs, which down-regulate the expression of a NOX gene selected from the group consisting of NOX4, NOXI, NOX2 (gp91phox, CYBB), NOX5, DUOX2, NOXOI, NOXAI and NOXA2 (p67phox) is disclosed. Also provided is a vector capable of expressing the compound A method is provided for treating or preventing the incidence or seventy of various diseases or conditions associated with NOX gene compĪsing administering to the patient the inhibitor in a pharmaceutical composition | 10-28-2010 |
20110028531 | NOVEL SIRNA COMPOUNDS FOR INHIBITING RTP801 - The present invention provides chemically modified siRNA compounds that target RTP801 and pharmaceutical compositions comprising same useful for treating microvascular disorders, eye diseases, hearing impairment, neurodegenerative diseases and disorders, spinal cord injury and respiratory conditions. | 02-03-2011 |
20110034534 | siRNA compounds and methods of use thereof - The present invention relates to compounds, pharmaceutical compositions comprising same and methods of use thereof for the inhibition of certain genes, including SOX9, ASPP1, CTSD, CAPNS1, FAS and FAS ligand. The compounds and compositions are useful in the treatment of subjects suffering from diseases or conditions and or symptoms associated with diseases or conditions in which gene expression has adverse consequences. | 02-10-2011 |
20110105591 | siRNA COMPOUNDS FOR INHIBITING NRF2 - The present invention provides chemically modified siRNA compounds that target the Nrf2 gene and pharmaceutical compositions comprising same useful for treating or preventing the incidence or severity of a cancerous disease, particularly various lung cancers. | 05-05-2011 |
20110178157 | MODULATION OF HSP47 EXPRESSION - Provided herein are compositions, methods and kits for modulating expression of target genes, particularly heat shock protein 47 (hsp47). The compositions, methods and kits may include nucleic acid molecules (for example, short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA) or short hairpin RNA (shRNA)) that modulate a gene encoding hsp47, for example, the gene encoding human hsp47. The composition and methods disclosed herein may also be used in treating conditions and disorders associated with hsp47 such as liver fibrosis, pulmonary fibrosis, peritoneal fibrosis and kidney fibrosis. | 07-21-2011 |
20110190380 | METHODS FOR DELIVERY OF SIRNA TO BONE MARROW CELLS AND USES THEREOF - The present invention relates to a method for the delivery of therapeutic oligonucleotides to bone marrow, and in particular delivery of siRNA to a subset of bone marrow cells. The method comprises systemically administering siRNA to a subject in need thereof, to reduce or inhibit expression of a gene associated with a disease or disorder or to symptoms associated with a disease or disorder associated with the cells. The invention further relates to chemically modified siRNA compounds, to pharmaceutical compositions comprising such compounds and to methods of using such compounds and compositions in the treatment of disease. | 08-04-2011 |
20110229557 | METHODS FOR TREATING EYE DISORDERS - The present invention relates to compositions and methods for inhibiting loss of a retinal ganglion cell in a subject, comprising non-invasively applying to the surface of the eye of the subject an ophthalmic composition comprising a therapeutically effective amount of at 5 least one siRNA which down regulates expression of a target gene associated with loss of the retinal ganglion cell, thereby inhibiting loss of the retinal ganglion cell in the subject. The methods of the invention also relate to the use of chemically modified siRNA compounds possessing structural motifs which down-regulate the expression of human genes expressed in retinal tissue in the mammalian eye. | 09-22-2011 |
20110251260 | NOVEL siRNAS AND METHODS OF USE THEREOF - The invention relates to compounds, in particular siRNAs, which inhibit the expression of specific human genes. The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier. The present invention also provides a method of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions comprising administering to a subject in need of treatment for such disease or condition and/or symptom the compound or the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject. The invention also provides antibodies which inhibit specified human polypeptides and pharmaceutical compositions comprising one or more such antibodies. | 10-13-2011 |
20110288155 | SIRNA COMPOUNDS AND METHODS OF USE THEREOF - The present application relates to double stranded oligonucleotide inhibitors of target genes, pharmaceutical compositions comprising same and the use of such molecules to treat, inter alia, neurodegenerative disorders including Alzheimer's disease and Amyotrophic Lateral Sclerosis, eye diseases including glaucoma and ION, acute renal failure, hearing loss, acute respiratory distress syndrome and in preventing or treating ischemia-reperfusion injury in organ transplant patients. | 11-24-2011 |
20120136044 | Compositions And Methods Of Inhibiting NADPH Oxidase Expression - The invention relates to one or more inhibitors, in particular siRNAs, which down-regulate the expression of a NOX gene selected from the group consisting of NOX4, NOX1, NOX2 (gp91phox, CYBB), NOX5, DUOX2, NOXO1, NOXA1 and NOXA2 (p67phox). The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the compound, and a pharmaceutically acceptable carrier. The present invention also contemplates a method of treating or preventing the incidence or severity of various diseases or conditions associated with NOX gene comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient. | 05-31-2012 |
20120142754 | MODULATION OF TIMP1 AND TIMP2 EXPRESSION - Provided herein are compositions, methods and kits for modulating expression of target genes, particularly of tissue inhibitor of metalloproteinase 1 and of tissue inhibitor of metalloproteinase 2 (TIMP1 and TIMP2, respectively). The compositions, methods and kits may include nucleic acid molecules (for example, short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA) or short hairpin RNA (shRNA)) that modulate a gene encoding TIMP1 and TIMP2, for example, the gene encoding human TIMP1 and TIMP2. The composition and methods disclosed herein may also be used in treating conditions and disorders associated with TIMP1 and TIMP2 including fibrotic diseases and disorders including liver fibrosis, pulmonary fibrosis, peritoneal fibrosis and kidney fibrosis. | 06-07-2012 |
20130030034 | MODULATION OF TIMP1 AND TIMP2 EXPRESSION - Provided herein are compositions, methods and kits for modulating expression of target genes, particularly of tissue inhibitor of metalloproteinase 1 and of tissue inhibitor of metalloproteinase 2 (TIMP1 and TIMP2, respectively). The compositions, methods and kits may include nucleic acid molecules (for example, short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA) or short hairpin RNA (shRNA)) that modulate a gene encoding TIMP1 and TIMP2, for example, the gene encoding human TIMP1 and TIMP2. The composition and methods disclosed herein may also be used in treating conditions and disorders associated with TIMP1 and TIMP2 including fibrotic diseases and disorders including liver fibrosis, pulmonary fibrosis, peritoneal fibrosis and kidney fibrosis. | 01-31-2013 |
20130071467 | RETINOID-LIPOSOMES FOR TREATING FIBROSIS - What is described are pharmaceutical compositions comprising a double-stranded nucleic acid molecule comprising a sense strand and an antisense strand wherein the sense and antisense strands are selected from the oligonucleotides described as SERPINH1_2 (SEQ ID NOS: 60 and 127), SERPINH1_45a (SEQ ID NOS: 98 and 165), and SERPINH1_51 (SEQ ID NOS: 101 and 168), and drug carrier comprising a mixture of a retinoid and a lipid vesicle, and methods of using these pharmaceutical compositions to treat a disease associated with hsp47 expression, including fibrosis. | 03-21-2013 |
20130071478 | RETINOID-LIPOSOMES FOR ENHANCING MODULATION OF HSP47 EXPRESSION - What is described are pharmaceutical compositions comprising a double-stranded nucleic acid molecule comprising a sense strand and an antisense strand wherein the sense and antisense strands are selected from the oligonucleotides described as SERPINH | 03-21-2013 |
20130123334 | NOVEL SIRNA STRUCTURES - The invention relates to siRNA compounds possessing novel sequences and structural motifs which down-regulate the expression of specific human genes. The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier. The present invention also provides a method of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions comprising administering to a subject in need of treatment for such disease or condition and/or symptom the compound or the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject. | 05-16-2013 |
20130131143 | RTP801L SIRNA COMPOUNDS AND METHODS OF USE THEREOF - The invention provides chemically modified siRNA oligonucleotides that target RTP801L, compositions comprising same and to the use of such molecules to treat, inter alia, respiratory diseases including acute and chronic pulmonary disorders, eye diseases including glaucoma and ION, microvascular disorders, angiogenesis- and apoptosis-related conditions, and hearing impairments. | 05-23-2013 |
20130137750 | DOUBLE STRANDED RNA COMPOUNDS TO RHOA AND USE THEREOF - The present invention relates to compounds, pharmaceutical compositions comprising same, methods of use thereof and kits for the down-regulation of RhoA gene. The compounds, compositions, methods and kits are useful in the treatment of subjects suffering from diseases or conditions and or symptoms associated with diseases or conditions in which RhoA expression has adverse consequences and for conferring neuroprotection. | 05-30-2013 |
20130267578 | COMPOSITIONS AND METHODS FOR INHIBITING NADPH OXIDASE EXPRESSION - The invention relates to one or more inhibitors, in particular siRNAs, which down-regulate the expression of a NOX gene selected from the group consisting of NOX4, NOX1, NOX2 (gp91phox, CYBB), NOX5, DUOX2, NOXO1, NOXA1 and NOXA2 (p67phox). The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the compound, and a pharmaceutically acceptable carrier. The present invention also contemplates a method of treating or preventing the incidence or severity of various diseases or conditions associated with NOX gene comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient. | 10-10-2013 |
20140005253 | COMPOSITIONS AND METHODS FOR TREATING LUNG DISEASE AND INJURY | 01-02-2014 |
20140235695 | MODULATION OF HSP47 EXPRESSION - Provided herein are compositions, methods and kits for modulating expression of target genes, particularly heat shock protein 47 (hsp47). The compositions, methods and kits may include nucleic acid molecules (for example, short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA) or short hairpin RNA (shRNA)) that modulate a gene encoding hsp47, for example, the gene encoding human hsp47. The composition and methods disclosed herein may also be used in treating conditions and disorders associated with hsp47 such as liver fibrosis, pulmonary fibrosis, peritoneal fibrosis and kidney fibrosis. | 08-21-2014 |
20140329878 | OLIGONUCLEOTIDE MODULATORS OF THE TOLL-LIKE RECEPTOR PATHWAY - Disclosed herein are double stranded nucleic acid molecules and pharmaceutical compositions comprising same useful in the treatment of, inter alia, acute and chronic inflammation, neuropathic pain, primary graft dysfunction (PGD) after lung transplantation in a subject in need thereof. The compounds are preferably chemically synthesized and modified dsRNA compounds, which down regulate or inhibit expression of a Toll like receptor genes. | 11-06-2014 |
20140350068 | RTP801L SIRNA COMPOUNDS AND METHODS OF USE THEREOF - The invention provides chemically modified siRNA oligonucleotides that target RTP801L, compositions comprising same and to the use of such molecules to treat, inter alia, respiratory diseases including acute and chronic pulmonary disorders, eye diseases including glaucoma and ION, microvascular disorders, angiogenesis- and apoptosis-related conditions, and hearing impairments. | 11-27-2014 |
20140356413 | RETINOID-LIPOSOMES FOR TREATING FIBROSIS - What is described are pharmaceutical compositions comprising a double-stranded nucleic acid molecule comprising a sense strand and an antisense strand wherein the sense and antisense strands are selected from the oligonucleotides described as SERPINH1_2 (SEQ ID NOS: 60 and 127), SERPINH1_45a (SEQ ID NOS: 98 and 165), and SERPINH1_51 (SEQ ID NOS: 101 and 168), and drug carrier comprising a mixture of a retinoid and a lipid vesicle, and methods of using these pharmaceutical compositions to treat a disease associated with hsp47 espresssion, including fibrosis. | 12-04-2014 |
20150018404 | DOUBLE-STRANDED OLIGONUCLEOTIDE COMPOUNDS FOR TREATING HEARING AND BALANCE DISORDERS - The present application relates to double stranded nucleic acid compounds, compositions comprising same and methods of use thereof for the treatment of hearing loss in a subject in need thereof. The compounds are preferably chemically synthesized and modified dsRNA molecules which inhibit expression of a gene expressed selected from the group consisting of HES1, HES5, HEY1, HEY2, ID1, ID2, ID3, CDKN1B, and NOTCH1. | 01-15-2015 |
20150050328 | METHODS FOR TREATING EYE DISORDERS - The present invention relates to compositions and methods for inhibiting loss of a retinal ganglion cell in a subject, comprising non-invasively applying to the surface of the eye of the subject an ophthalmic composition comprising a therapeutically effective amount of at least one siRNA which down regulates expression of a target gene associated with loss of the retinal ganglion cell, thereby inhibiting loss of the retinal ganglion cell in the subject. The methods of the invention also relate to the use of chemically modified siRNA compounds possessing structural motifs which down-regulate the expression of human genes expressed in retinal tissue in the mammalian eye. | 02-19-2015 |