Abstract: The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g. Danon disease.
Type:
Grant
Filed:
December 27, 2019
Date of Patent:
July 7, 2020
Assignee:
Rocket Pharmaceuticals, Ltd.
Inventors:
Annahita Keravala, Simon Moore, David Ricks
Abstract: The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends optimized functional CRISPR-Cas enzyme systems, wherein the guide sequence is modified by secondary structure to increase the specificity of the CRISPR-Cas system and whereby the secondary structure can protect against exonuclease activity and allow for 5? additions to the guide sequence.
Type:
Grant
Filed:
June 12, 2017
Date of Patent:
June 30, 2020
Assignees:
THE BOARD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
Inventors:
Feng Zhang, Omar O. Abudayyeh, James E. Dahlman, Patrick Hsu, David A. Scott
Abstract: This invention provides methods for transducing a ciliated cell with a recombinant serotype 2 adeno-associated virus (AAV) vector. Additionally, the invention provides methods of treating diseases associated with a mutated gene by transducing a ciliated cell with a recombinant serotype 2 AAV vector containing a corrective transgene.
Type:
Grant
Filed:
May 30, 2008
Date of Patent:
June 30, 2020
Assignee:
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
Abstract: This disclosure provides an improved system for culturing human embryonic stem cells. The cells are cultured in suspension so as to maximize the production capacity of the culture environment. The new culture system of this invention allows for bulk proliferation of hES cells in a more cost-effective manner, which facilitates commercial production of important products for use in human therapy.
Type:
Grant
Filed:
February 12, 2018
Date of Patent:
June 9, 2020
Assignee:
Asterias Biotherapeutics, Inc.
Inventors:
Ramkumar Mandalam, Yan Li, Isabelle Nadeau-Demers
Abstract: A method for qualitatively assessing products used in in vitro fertilization is provided. Also disclosed is an improved quality control assay for use in clinical Assisted Reproductive Technologies (ART).
Type:
Grant
Filed:
April 9, 2014
Date of Patent:
June 2, 2020
Assignee:
FUJIFILM Irvine Scientific, Inc.
Inventors:
Hsiao-Tzu Ni, Samira Es-Slami, Rebecca Susan Gilbert
Abstract: The present invention relates to transgenic mammals that express canine-based immunoglobulins, including transgenic rodents that express canine-based immunoglobulins for the development of canine therapeutic antibodies.
Abstract: The invention provides non-human cells and mammals having a genome encoding chimeric antibodies and methods of producing transgenic cells and mammals. Certain aspects of the invention include chimeric antibodies, humanized antibodies, pharmaceutical compositions and kits. Certain aspects of the invention also relate to diagnostic and treatment methods using the antibodies of the invention.
Abstract: The present disclosure relates to a method for controlling the differentiation of embryonic stem cells into adipocytes or kidney precursor cells by regulating SIRT1 (silent mating type information regulation 2 homolog; sirtuin 1) expression, the method, which controls the differentiation of embryonic stem cells into adipocytes by regulating SIRT1 expression, being capable of controlling the inhibition or promotion of adipocyte differentiation in accordance with the timing of a SIRT1 expression inhibitor treatment. Furthermore, kidney precursor cell differentiation can be regulated by a SIRT1 expression inhibitor or promoter treatment. Accordingly, a SIRT1 inhibitor can be selected to be used as an inhibitor or a therapeutic agent for obesity, diabetes accompanying obesity, and renal and metabolic diseases.
Type:
Grant
Filed:
January 22, 2016
Date of Patent:
May 26, 2020
Assignees:
INDUSTRIAL COOPERATION FOUNDATION CHONBUK NATIONAL UNIVERSITY, Chonbuk National University Hospital
Abstract: The present invention relates to the fields of life sciences and medicine. Specifically, the invention relates to cancer therapies of humans. More specifically, the present invention relates to oncolytic adenoviral vectors alone or together with therapeutic compositions for therapeutic uses and therapeutic methods for cancer. In one aspect the present invention relates to separate administration of adoptive cell therapeutic composition and oncolytic adenoviral vectors. Furthermore, the present invention relates to a pharmaceutical kit and a pharmaceutical composition, both utilizing oncolytic adenoviral vectors.
Type:
Grant
Filed:
April 16, 2014
Date of Patent:
May 12, 2020
Assignee:
TILT BIOTHERAPEUTICS OY
Inventors:
Akseli Hemminki, Markus Vaha-Koskela, Siri Tahtinen, Vincenzo Cerullo
Abstract: Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian vascular system, and complications from Type I diabetes. Also disclosed are methods for systemic and tissue-localized delivery of therapeutic rAAV-based gene expression cassettes to vascular endothelial cells, tissues, and organs, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of vasculitis, and complications arising from Type I diabetes, such as macular edema, nephropathy, diabetic retinopathy, and the like.
Type:
Grant
Filed:
February 16, 2016
Date of Patent:
May 12, 2020
Assignee:
University of Florida Research Foundation, Incorporated
Inventors:
William W. Hauswirth, Sanford L. Boye, Daniel M. Lipinski, Michael E. Boulton, Shannon E. Boye
Abstract: A modified effector cell includes a non-reversibly produced vector-encoded anti-GD2-BB-? chimeric receptor for use in disialoganglioside GD2-expressing neoplasia, which is inserted in the cell, to obtain an effector cell that stably produces the anti-GD2-BB-? chimeric receptor, the chimeric receptor having two distinct mutually fused portions, i.e. an intra-cytoplasmic portion and an extra-cytoplasmic portion.
Type:
Grant
Filed:
October 25, 2012
Date of Patent:
May 12, 2020
Assignee:
St. Jude Children's Research Hospital, Inc.
Inventors:
Massimo Dominici, Sara Caldrer, Maria Carlotta Spano, Paolo Paolucci, Marco Bestagno, Dario Campana
Abstract: A method of generating protein-induced pluripotent stem cells by delivering bacterially expressed reprogramming proteins into nuclei of starting somatic cells using the QQ-protein transduction technique, repeating several cell reprogramming cycles for creating reprogrammed protein-induced pluripotent stem cells, moving the reprogrammed cells into a feeder-free medium for expansion, and expanding and passaging the reprogrammed cells in a whole dish for generating homogeneous piPS cells. Also provided are the piPS cells formed using this method and uses thereof.
Abstract: The invention provides for methods of differentiating pancreatic endocrine cells into pancreatic beta cells expressing PDX1, NKX6.1, MAFA, UCN3 and SLC2A. These pancreatic beta cells may be obtained by step-wise differentiation of pluripotent stem cells. The pancreatic beta cells exhibit glucose-dependent mitochondrial respiration and glucose-stimulated insulin secretion similar to islet cells.
Abstract: The invention relates to a novel non-obese diabetic (NOD) mouse and its application. Particularly, the invention relates to a NOD mouse specifically expressing anti-polyethylene glycol membrane antibody reporter (anti-PEG reporter) in the NOD mouse.
Abstract: A transgenic cat with a phenotype characterized by the substantial absence of the major cat allergen, Fel d I. The phenotype is conferred in the transgenic cat by disrupting the coding sequence of the target gene with a specialized construct. The phenotype of the transgenic cat is transmissible to its offspring.
Type:
Grant
Filed:
October 11, 2016
Date of Patent:
April 21, 2020
Inventors:
David B. Avner, James Kehler, Sven Bocklandt
Abstract: The invention provides non-human cells and mammals having a genome encoding chimeric antibodies and methods of producing transgenic cells and mammals. Certain aspects of the invention include chimeric antibodies, humanized antibodies, pharmaceutical compositions and kits. Certain aspects of the invention also relate to diagnostic and treatment methods using the antibodies of the invention.
Abstract: The invention provides non-human cells and mammals having a genome encoding chimeric antibodies and methods of producing transgenic cells and mammals. Certain aspects of the invention include chimeric antibodies, humanized antibodies, pharmaceutical compositions and kits. Certain aspects of the invention also relate to diagnostic and treatment methods using the antibodies of the invention.
Abstract: The invention provides non-human cells and mammals having a genome encoding chimeric antibodies and methods of producing transgenic cells and mammals. Certain aspects of the invention include chimeric antibodies, humanized antibodies, pharmaceutical compositions and kits. Certain aspects of the invention also relate to diagnostic and treatment methods using the antibodies of the invention.
Abstract: The present invention is of methods of establishing and propagating human embryonic stem cell lines using feeder cells-free, xeno-free culture systems and stem cells which are capable of being maintained in an undifferentiated, pluripotent and proliferative state in culture which is free of xeno contaminants and feeder cells.
Type:
Grant
Filed:
September 3, 2018
Date of Patent:
March 17, 2020
Assignee:
Technion Research & Development Foundation Limited
Abstract: The present invention relates to an artificial cartilage containing mesenchymal stem cell (MSC)-like dedifferentiated cells obtained by passage culturing costal chondrocytes, and a preparation process thereof.
Type:
Grant
Filed:
June 12, 2017
Date of Patent:
March 10, 2020
Assignees:
BIO SOLUTION CO., LTD., KOREA INSTITUTE OF RADIOLOGICAL & MEDICAL SCIENCES, UNIVERSITY-INDUSTRY COOPERATION GROUP OF KYUNGHEE UNIVERSITY
Inventors:
Young Sook Son, Jung Sun Lee, Eun Kyung Lee, Jin Yeon Lee