Abstract: The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA-targeting systems comprising a novel DNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA. Methods for making and using and uses of such systems, methods, and compositions and products from such methods and uses are also disclosed and claimed.
Type:
Grant
Filed:
April 30, 2019
Date of Patent:
October 10, 2023
Assignees:
THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
Inventors:
Feng Zhang, Bernd Zetsche, Jonathan S. Gootenberg, Omar O. Abudayyeh, Ian Slaymaker
Abstract: The present disclosure provides an automated method of producing genetically modified immune cells, including chimeric antigen receptor T (CAR T) cells, utilizing a fully-enclosed cell engineering system.
Type:
Grant
Filed:
May 24, 2022
Date of Patent:
October 10, 2023
Assignees:
LONZA WALKERSVILLE, INC., LONZA COLOGNE GMBH, OCTANE BIOTECH INC.
Inventors:
Yaling Shi, Erika McAfee, Samatha Bandapalle, Ann Siehoff, Timo Gleissner, Joseph O'Connor, Eytan Abraham, Kelly Purpura, Nuala Trainor, Timothy Smith
Abstract: The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA-targeting systems comprising a novel DNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA. Methods for making and using and uses of such systems, methods, and compositions and products from such methods and uses are also disclosed and claimed.
Type:
Grant
Filed:
December 17, 2021
Date of Patent:
October 3, 2023
Assignees:
THE BROAD INSTITUTE INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
Inventors:
Feng Zhang, Bernd Zetsche, Johnathan S. Gootenberg, Omar O. Abudayyeh, Ian Slaymaker
Abstract: The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a novel RNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.
Type:
Grant
Filed:
June 24, 2022
Date of Patent:
October 3, 2023
Assignees:
The Broad Institute, Inc., Massachusetts Institute of Technology, President and Fellows of Harvard College, Rutgers, the State University of New Jersey, Skolkovo Institute of Science and Technology, The United States of America, as represented by the Secretary, Department of Health and Human Services
Inventors:
Konstantin Severinov, Feng Zhang, Yuri I. Wolf, Sergey Shmakov, Ekaterina Semenova, Leonid Minakhin, Kira S. Makarova, Eugene Koonin, Silvana Konermann, Julia Joung, Jonathan S. Gootenberg, Omar O. Abudayyeh, Eric S. Lander
Abstract: Provided herein are isolated DNA vectors comprising a heterologous gene, wherein the DNA vector is devoid of bacterial plasmid DNA and/or bacterial signatures, which can abrogate persistence in vivo. The invention also features pharmaceutical compositions (non-immunogenic pharmaceutical compositions) including the DNA vectors of the invention, which can be used for induction of long-term, episomal expression of a heterologous gene in a subject. The invention involves methods of treating a subject by administering the DNA vectors of the invention, including methods of treating disorders associated with a defect in a target gene.
Abstract: Provided herein are safe harbor loci and methods for identifying and using safe harbor loci to engineer cells to express transgenes. The safe harbor loci exhibit increased transgene knock-in efficiency and allow for increased, stable expression of transgenes in engineered cells. Guide ribonucleic acids (gRNAs) may be used for insertion of transgenes in the safe harbor loci.
Abstract: The present disclosure provides methods and compositions for the treatment of diseases and/or disorders in a subject, including, but not limited to neurological disorders such as giant axonal neuropathy. The methods described herein include direct administration of a gene therapy (e.g. an rAAV viral vector) to a subject via injection into a vagus nerve (e.g. the left vagus nerve) of the subject.
Type:
Grant
Filed:
April 14, 2021
Date of Patent:
September 12, 2023
Assignee:
THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
Abstract: The present invention relates to a genetic tool comprising at least two different nucleic acids allowing the transformation, by homologous recombination, of a bacterium of the genus Clostridium, typically of a solventogenic bacterium.
Abstract: Disclosed herein, are systems and methods for analyzing complex data signals using artificial intelligence and/or deconvolution algorithms to determine output pertaining to the state or status of one or more parameters. Data sets may include signals from various sources that can confound or distort the signals of interest. Accordingly, disclosed herein are deconvolution algorithms that enable the determination of the status of sources that correspond to the signals of interest.
Type:
Grant
Filed:
November 5, 2020
Date of Patent:
August 29, 2023
Assignee:
COFACTOR GENOMICS, INC.
Inventors:
Ian Schillebeeckx, Jon R. Armstrong, Jeffrey Hiken
Abstract: The present disclosure provides instruments, modules and methods for improved detection of edited cells following nucleic acid-guided nuclease genome editing. The disclosure provides improved automated instruments that perform methods—including high throughput methods—for screening cells that have been subjected to editing and identifying cells that have been properly edited.
Type:
Grant
Filed:
December 17, 2021
Date of Patent:
August 29, 2023
Assignee:
INSCRIPTA, INC
Inventors:
Andrew Garst, Richard Fox, Phillip Belgrader, Don Masquelier
Abstract: The present invention relates to an in vitro method of culturing a segmented filamentous bacterium strain, comprising co-culturing said segmented filamentous bacterium strain with a eukaryotic host cell, wherein the culture is performed at an O2 level inferior to 5% in a rich tissue culture liquid medium containing bacterial medium components including iron. The present invention also relates to methods for genetically modifying a segmented filamentous bacterium strain comprising a step a culturing the strain in vitro.
Type:
Grant
Filed:
August 22, 2019
Date of Patent:
August 15, 2023
Assignees:
INSTITUT PASTEUR, FONDATION IMAGINE, ASSISTANCE PUBLIQUE—HOPITAUX DE PARIS, UNIVERSITE PARIS CITE, INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
Inventors:
Gerard Eberl, David Bikard, Pamela Schnupf, Nadine Cerf Bensussan, Valerie Gaboriau-Routhiau, Philippe Sansonetti
Abstract: Compositions and methods for gene editing are provided. The methods employ an oligo-based annealing mechanism that is rooted in the process of DNA replication rather than homologous recombination (HR). Oligo incorporation efficiencies are comparable and often exceed those of CRISPR/cas9 editing without the need for double strand breaks (DSBs). By relying on the multiplex annealing of oligos rather than DSBs the process is highly scalable across a genomic region of interest and can generate many scarless modifications of a chromosome simultaneously. Combinatorial genomic diversity can be generated across a population of cells in a single transformation event; genomic landscapes can be traversed through successive iterations of the process, and genome-wide changes can be massively parallelized and amplified through systematic strain mating.
Abstract: Described herein is a method of preventing or treating a disease in a mammalian subject, comprising administering to the subject who is in need thereof an effective dosage of a pharmaceutical composition comprising a virus like particle (VLP) comprising: an alphavirus replicon comprising a recombinant polynucleotide, wherein the polynucleotide comprises a sequence encoding both subunits of a human class II major histocompatibility antigen, a retroviral gag protein, and a fusogenic envelope protein, wherein the VLP does not contain an alphavirus structural protein gene.
Type:
Grant
Filed:
December 14, 2020
Date of Patent:
August 8, 2023
Assignee:
The USA, as represented by the Secretary, Dept. of Health and Human Services
Inventors:
Deb K. Chatterjee, Stanislaw J. Kaczmarczyk
Abstract: The present disclosure provides methods and systems for identification of genomic regions for therapeutic targeting. A method for identifying one or more genomic regions for therapeutic targeting, which may facilitate re-programming of a cell from one phenotypic state to another, may comprise: providing single-cell RNA-seq data for a plurality of diseased cells and a plurality of normal cells of a cell type; mapping the single-cell RNA-seq data for the plurality of diseased cells and the plurality of normal cells into a latent space corresponding to a plurality of phenotypic states of the cell type; identifying, based at least in part on a topology of the latent space, the one or more genomic regions for therapeutic targeting; and electronically outputting the one or more genomic regions for therapeutic targeting.
Abstract: Described herein are methods and vectors for rational, multiplexed manipulation of chromosomes within open reading frames (e.g., in protein libraries) or any segment of a chromosome in a cell or population of cells, in which various CRISPR systems are used.
Type:
Grant
Filed:
August 23, 2019
Date of Patent:
July 18, 2023
Assignee:
THE REGENTS OF THE UNIVERSITY OF COLORADO
Abstract: Provided herein are compositions and methods for modifying a predetermined nucleic acid sequence. A programmable nucleoprotein molecular complex containing a polypeptide moiety and a specificity conferring nucleic acid (SCNA) which assembles in-vivo, in a target cell, and is capable of interacting with the predetermined target nucleic acid sequence is provided. The programmable nucleoprotein molecular complex is capable of specifically modifying and/or editing a target site within the target nucleic acid sequence and/or modifying the function of the target nucleic acid sequence.
Type:
Grant
Filed:
March 3, 2017
Date of Patent:
July 4, 2023
Assignee:
TARGETGENE BIOTECHNOLOGIES LTD.
Inventors:
Yoel Moshe Shiboleth, Dan Michael Weinthal
Abstract: The present invention relates to a technique for genomic library screening and provides a method for separating, capturing, analyzing, and retrieving cells and cell products by using a microstructure that can be preferentially applied to the field of antibody engineering for the development of new therapeutic antibodies and can be extensively applied to multiple genetic/phenotypic analysis of various biochemical molecules, for example, in the field of protein engineering and metabolic engineering.
Type:
Grant
Filed:
May 14, 2018
Date of Patent:
July 4, 2023
Assignee:
SEOUL NATIONAL UNIVERSITY R&DB FOUNDATION
Inventors:
Sung Hoon Kwon, Jun Hoi Kim, Seo Hee Chang, Ok Ju Kim