Quark Pharmaceuticals, Inc.

Nitto-denko Corporation

Other

Quark Biotech, Inc.

Silence Therapeutics Ag

Washington University

Hadasit Medical Research Services and Development Ltd.

Insight Biopharmaceuticals Ltd.

Rutgers, the State University of New Jersey

The United States Government As Represented by the Department of Veterans Affairs

Elena Feinstein

Igor Mett

Hagar Kalinski

Sharon Avkin-Nachum

Yoshiro Niitsu

Yasunobu Tanaka

Rami Skaliter

Shai Erlich

Svetlana Adamsky

Wenbin Ying

Joseph E Payne

Israel Vlodavsky

Iris Pecker

Mohammad Ahmadian

Victor Knopov

Richard P Witte

Violetta Akopian

Loren A Perelman

Kenjiro Minomi

Shai Ehrlich

Yun Liu

Sharon Avkin-Nahum

Klaus Giese

Evgenia Alpert

Igor Spivak

Elizabeth C Squiers

Lei Yu

Paz Einat

Hirokazu Takahashi

Amir Bar-Ilan

Orna Mor

Daniel Zurr

Ning Chen

Xiaomei Jin

Leonid Beigelman

Aviv Regev

Andrew E Gelman

Roni Wechsler

James D Thompson

Orit Segev

Natanja Slager

Huseyin Aygun

James A McSwiggen

Jörg Kaufmann

Priya Prakash Karmali

John Gaudette

Zheng Hou

Bruce Molitoris

Sridhar C Nagarajan

Alexander Faerman

Martin Grumet

Daniel J Odenheimer

Rabia Ozden

Dganit Bar

Daniel Rothenstein

Svetlana Gorodin

Sylvia G Kachalsky

Aviva Samach

Netanja Slager

Ron Lahav

Nir Sharon

Sharon Avkin-nachum

Joerg Kaufmann

Yun Lui

Jörg c/o Atugen Ag Kaufmann

The present application relates to nucleic acid compounds, compositions comprising same and methods of use thereof for treatment of various diseases, disorders and conditions. The compounds are preferably chemically synthesized and modified double-stranded nucleic acid molecules which down regulate expression of a p53 gene.

Double-stranded oligonucleotide molecules targeting p53 and methods of use thereof

The present disclosure relates to methods of treating a patient suffering from or at risk of developing an ocular disease, disorder or injury, and includes treatment regimens using a double-stranded RNA compound that down-regulates CASP2 expression, or a pharmaceutically acceptable salt thereof.

Double-stranded RNA compounds to CASP2 and uses thereof

Provided herein are double stranded nucleic acid molecules, compositions comprising same and methods of use thereof for the treatment of a subject wherein expression of DDIT4 is associated with the etiology or progression of a disease or disorder in the subject. The compounds are preferably chemically synthesized and modified dsRNA molecules.

Double-stranded oligonucleotide molecules to DDIT4 and methods of use thereof

What is described is a method for treating a fibrotic disease by administering a pharmaceutical composition comprising a drug carrier, which comprises a lipid and a retinoid, and a double-stranded nucleic acid molecule, which comprises an antisense sequence to mRNA encoding human hsp47.

Method for treating fibrosis using siRNA and a retinoid-lipid drug carrier

What is described is a pharmaceutical composition for treating a fibrotic disease comprising a drug carrier, which comprises a lipid and a retinoid, and a double-stranded nucleic acid molecule, which comprises an antisense sequence to mRNA encoding human hsp47.

Retinoid-lipid drug carrier

Disclosed herein are double stranded RNA molecules which have been modified to exhibit one of the following, increased activity, enhanced nuclease stability, reduced off target activity and or reduced immunogenicity, to pharmaceutical compositions comprising such compounds and to methods of use. Further disclosed is a method for the synthesis of threose nucleic acid phosphoramidites and methods of use thereof.

Double stranded oligonucleotide compounds comprising positional modifications

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.

Modulation of HSP47 expression

The present invention provides antibodies or antigen-binding fragments thereof that specifically bind the ENDO180 polypeptide and are internalized thereby, to conjugates comprising the molecules, to compositions comprising the antibodies and conjugates and to methods of using the same for delivery of therapeutic agents to cells that express the ENDO180 polypeptide on the surface of the cell for treating cell proliferative diseases or disorders and fibrosis, and for controlling (modulating) tumor progression.

Composition of anti-ENDO180 antibodies and methods of use for the treatment of cancer and fibrotic diseases

Double-stranded oligonucleotide molecules to P53 and methods of use thereof

The invention, in some embodiments, relates to compounds and methods for the prevention of ischemia reperfusion injury (IRI) in organs, and in particular to IRI in organs aged 35 years and older. Specific uses include prevention of IRI in native organs in vivo, in reimplantations and in transplantations of donor organs aged 35 years and older. Additional embodiments include the prophylaxis of delayed graft function (DGF) and reduction in the frequency, amount and duration of dialysis in recipients of deceased donor kidney transplantations. The methods entail contacting the organ in vivo or ex vivo with a temporary p53 inhibitor. Novel temporary dsNA p53 inhibitors are further provided.

Methods and compositions for preventing ischemia reperfusion injury in organs

Disclosed herein are therapeutic methods for treating lung diseases, disorders and injury in a mammal, including treatment of acute respiratory distress syndrome (ARDS), acute lung injury, pulmonary fibrosis (idiopathic), bleomycin induced pulmonary fibrosis, mechanical ventilator induced lung injury, chronic obstructive pulmonary disease (COPD), chronic bronchitis, emphysema, bronchiolitis obliterans after lung transplantation and lung transplantation-induced acute graft dysfunction, including treatment, prevention or prevention of progression of primary graft failure, ischemia-reperfusion injury, reperfusion injury, reperfusion edema, allograft dysfunction, pulmonary reimplantation response, bronchiolitis obliterans after lung transplantation and/or primary graft dysfunction (PGD) after organ transplantation, in particular in lung transplantation, comprising down-regulating the TLR2 gene or both the TLR2 gene and TLR4 gene. Provided herein are compositions, methods and kits for treating lung diseases, disorders and injury.

Compositions and methods for treating lung disease and injury

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 Toll like receptor 4.

Oligonucleotide modulators of the toll-like receptor pathway

Disclosed herein are methods and kits useful for providing neuroprotection to neurons in the inner ear and to methods of treating inner ear diseases and disorders, including tinnitus and Mnire's disease.

Methods and compositions for neuroprotection

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.

Methods for treating eye disorders

Growing community of inventors

Rehovot, Israel

Elena Feinstein