Abstract: The present invention relates to methods and compositions for specifically modulating the Hippo pathway transcription factor TAZ (WWTR1), as a therapeutic target for inhibiting or preventing liver conditions including the progression of steatosis-to-NASH in a patient.
Type:
Grant
Filed:
April 18, 2017
Date of Patent:
April 29, 2025
Assignees:
The Trustees of Columbia University in the City of New York, The Brigham and Women's Hospital, Inc.
Inventors:
Ira Tabas, Xiaobo Wang, Omid Farokhzad, Xiaoding Xu
Abstract: Compositions and methods for fetal or in utero delivery of active agents are provided. The compositions are most typically administered intravenously via the vitelline vein, umbilical vein, or directly into the amniotic cavity of a pregnant mother. Fibroblast growth factor is to correct structural defects of neural tissue.
Type:
Grant
Filed:
December 27, 2023
Date of Patent:
April 8, 2025
Assignee:
YALE UNIVERSITY
Inventors:
W. Mark Saltzman, David H. Stitelman, James Stephen Farrelly, Anthony Bianchi
Abstract: The present invention relates to a pharmaceutical composition comprising a polyribonucleotide (RNA) with a sequence which encodes a bone morphogenetic protein (BMP) for use in (i) treating or preventing a bone disease, bone disorder or bone injury; and/or (ii) inducing or enhancing osteogenic differentiation, osteogenesis, ossification, bone regeneration and/or bone morphogenesis in a patient. The present invention also relates to the respective BMP encoding RNAs (BMP RNAs), in particular in its chemically modified form. The present invention also relates to complexes which comprise or are complexed with the BMP RNA, in particular to the respective transfection complexes like lipofection, magnetofection and magnetolipofection complexes. The present invention further relates to a carrier and carrier body to which the RNA or complex has been loaded and to a pharmaceutical composition comprising said carrier or carrier body.
Type:
Grant
Filed:
November 10, 2015
Date of Patent:
April 1, 2025
Assignee:
ethris GmbH
Inventors:
Elizabeth Balmayor, Carsten Rudolph, Christian Plank
Abstract: A solid cosmetic which contains 1 to 30% by mass of (A) a solid oil component having a melting point of 50 to 120° C., 50 to 95% by mass % of (B) a liquid oil component, 3 to 40% by mass % of (C) a nonionic surfactant having an HLB value of 5 to 13, 0.1 to 20% by mass % of (D) a powder having a volume average particle diameter of 1 to 200 ?m, and 0.1 to 10% by mass of (E) fumed silica is excellent in cleansing property, washing-off property and refreshing feeling after washing, and is also excellent in quality stability such as appearance and uniformity of composition.
Abstract: The present invention provides a nucleic acid conjugate in which a sugar chain ligand is bonded to an oligonucleotide via a linker, the sugar chain ligand having O-bonded mannose at its non-reducing end.
Abstract: The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).
Type:
Grant
Filed:
January 21, 2021
Date of Patent:
March 18, 2025
Assignee:
ModernaTX, Inc.
Inventors:
Paolo Martini, Stephen Hoge, Kerry Benenato, Vladimir Presnyak, Iain McFadyen, Ellalahewage Sathyajith Kumarasinghe, Ding An, Staci Sabnis
Abstract: Provided herein are compositions and methods for the viral gene therapy (e.g., AAV-directed gene therapy) of cholesterol storage diseases or disorders, such as Niemann-Pick disease, Type C.
Type:
Grant
Filed:
September 25, 2020
Date of Patent:
January 21, 2025
Assignee:
The United States of America, as represented by the Secretary, Department of Health and Human Services
Inventors:
Charles P. Venditti, Randy Chandler, William J. Pavan
Abstract: Materials and methods for treating a patient with hereditary hemochromatosis (HHC), both ex vivo and in vivo, and materials and methods for modulating the expression, function, or activity of a haemochromatosis (HFE) gene in a cell by genome editing.
Type:
Grant
Filed:
July 9, 2021
Date of Patent:
January 7, 2025
Assignee:
CRISPR THERAPEUTICS AG
Inventors:
Roman Lvovitch Bogorad, Chad Albert Cowan, Ante Sven Lundberg
Abstract: A compound comprising formula (I): wherein R1 is an alkylamino group or a group containing at least one aromatic group; R2 and R3 are independently an aliphatic group or hydrophobic group; R4 and R5 are independently H, a substituted or unsubstituted alkyl group, an alkenyl group, an acyl group, an aromatic group, polymer, a targeting group, or a detectable moiety; a, b, c, and d are independently an integer from 1 to 10; and pharmaceutically acceptable salts thereof.
Type:
Grant
Filed:
September 28, 2021
Date of Patent:
December 10, 2024
Assignee:
CASE WESTERN RESERVE UNIVERSITY
Inventors:
Zheng-Rong Lu, Anthony Malamas, Maneesh Gujrati, Da Sun
Abstract: The invention relates to mRNA therapy for the treatment of Alagille syndrome (ALGS), mRNAs for use in the invention, when administered in vivo, encode JAGGED 1 (JAG1), isoforms thereof functional fragments thereof, and fusion proteins comprising JAG1, mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of JAG1 expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient JAG1 activity in subjects.
Type:
Grant
Filed:
May 18, 2017
Date of Patent:
October 29, 2024
Assignee:
ModernaTX, Inc.
Inventors:
Kerry Benenato, Stephen Hoge, Paolo Martini, Iain Mcfadyen, Vladimir Presnyak, Ding An, Ellalahewage Sathyajith Kumarasinghe
Abstract: Disclosed are a sustained-release injection formulation containing a biodegradable polymer double microcapsule that contains a conjugate of poly-L-lactic acid (hereinafter referred to as “PLLA”) filler and hyaluronic acid (hereinafter referred to as “HA”) and is capable of controlling the release rate of PLLA, and a method of preparing the same.
Abstract: A concentrate composition comprising: (a) at least 0.1 wt % of a surfactant compound; and (b) at least 10 wt % one or more ingredients selected from aldehydes, succinimidyl esters, chelating agents and amine salts of carboxylic acids.
Type:
Grant
Filed:
September 29, 2017
Date of Patent:
September 3, 2024
Assignee:
Innospec Limited
Inventors:
Nicholas John Dixon, Matthew Robert Giles, Kimberley Elizabeth Griffiths, Tony Gough, Ian Malcolm McRobbie
Abstract: The present invention relates to oligomers, polymers and lipidoids comprising characteristic oligo(alkylene amine) moieties which are useful as vehicles for transfecting a cell with RNA. The present invention furthermore relates to a composition comprising at least a nucleic acid and an oligomer or polymer or a lipidoid comprising such oligo(alkylene amine) moieties and to a method of transfecting a cell using said composition. Furthermore, the present invention relates to pharmaceutical compositions and uses.
Type:
Grant
Filed:
June 27, 2014
Date of Patent:
August 20, 2024
Assignee:
ethris GmbH
Inventors:
Christian Dohmen, Christian Plank, Carsten Rudolph, Christian Koch
Abstract: Methods of producing a protein in cell culture comprising an anti-senescence compound, such as the antioxidant carnosine, are provided. According to teachings of the present invention, cells grown in a cell culture medium comprising an anti-senescence compound exhibit increased viability and productivity. Furthermore, cell cultures grown in the presence of an anti-senescence compound exhibit decreased levels of high molecular weight aggregates in the cell culture medium.
Type:
Grant
Filed:
April 22, 2008
Date of Patent:
August 6, 2024
Assignee:
WYETH LLC
Inventors:
Jose Manuel Gomes, Yen-Tung Luan, Gregory Walter Hiller, Wenge Wang
Abstract: Compositions are provided, the compositions comprising: (1) a nanoparticle; (2) optionally, a linker and/or masking agent; and (3) a ligand configured to activate peri-tumoral cells to induce scarring by the peri-tumoral cells. In some aspects, administration of the compositions to a subject may generate an environment capable of walling-off and containing invasive tumors.
Type:
Grant
Filed:
November 26, 2019
Date of Patent:
June 4, 2024
Assignee:
DUKE UNIVERSITY
Inventors:
Tarun Saxena, Ravi Bellamkonda, Nassir Mokarram-Dorri
Abstract: The present invention is directed to hemostatic compositions comprising at least partially integrated agglomerated ORC fibers, fibrinogen, and thrombin and methods of forming a powdered hemostatic composition, comprising the steps of: forming a suspension of a mixture comprising particles of fibrinogen, thrombin, ORC fibers in a non-aqueous low boiling solvent; spraying the suspension through a nozzle onto a substrate, allowing the non-aqueous solvent to evaporate; separating from the substrate and sieving the composition.
Type:
Grant
Filed:
December 13, 2021
Date of Patent:
June 4, 2024
Assignee:
Guangzhou Bioseal Co., Ltd.
Inventors:
Shuang Chen, Yufu Li, Dengmin Feng, Xiang Wan
Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
Type:
Grant
Filed:
August 3, 2020
Date of Patent:
June 4, 2024
Assignee:
TRANSLATE BIO, INC.
Inventors:
Frank DeRosa, Braydon Charles Guild, Michael Heartlein
Abstract: The disclosure relates to ovarian cancer neoantigens, polynucleotides encoding them, vectors, host cells, recombinant virus particles, vaccines comprising the neoantigens, proteinaceous molecules binding the ovarian cancer neoantigens, and methods of making and using them.
Type:
Grant
Filed:
February 12, 2021
Date of Patent:
April 2, 2024
Assignee:
Janssen Biotech, Inc.
Inventors:
Vipul Bhargava, Vinod Krishna, David J. Pocalyko, Pegah Safabakhsh, Manuel Alejandro Sepulveda
Abstract: Compositions and methods for targeted treatment of cancer are disclosed. In particular, the invention relates to methods of targeting anti-cancer therapy to cells exhibiting aberrant signaling associated with cancer pathogenesis by administering synthetic signaling proteins that couple detection of an oncogenic signal to release of therapeutic agents into cancerous cells.
Type:
Grant
Filed:
May 1, 2019
Date of Patent:
March 26, 2024
Assignee:
The Board of Trustees of the Leland Stanford Junior University