Patents Assigned to THE BROAD INSTITUTE, INC.
-
Publication number: 20210062246Abstract: The specification relates generally to methods of detecting, diagnosing, and/for identifying pathogens, c.g., infectious disease pathogens and determining their drug sensitivity and appropriate methods of treatment. This invention also relates generally to methods of monitoring pathogen infection in individual subjects as well as larger populations of subjects.Type: ApplicationFiled: May 18, 2020Publication date: March 4, 2021Applicants: THE BROAD INSTITUTE, INC., THE GENERAL HOSPITAL CORPORATIONInventors: James Gomez, Deborah Hung, Amy Barczak
-
Patent number: 10930367Abstract: Disclosed are thermodynamic and multiplication methods concerning CRISPR-Cas systems, and apparatus therefor.Type: GrantFiled: June 12, 2015Date of Patent: February 23, 2021Assignees: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY, PRESIDENT AND FELLOWS OF HARVARD COLLEGEInventors: Feng Zhang, Yinqing Li, David Arthur Scott, Joshua Asher Weinstein, Patrick Hsu
-
Publication number: 20210047617Abstract: The present invention features assays for co-culturing primary cells while maintaining key biological activities specific to the primary cells. The invention is based, at least in part, on the discovery that compositions and methods for primary cells in a high-throughput co-culture platform, image analysis for distinguishing cells in co-cultures and assays that are suitable for screening of agents in epithelial cells, such as hepatocytes.Type: ApplicationFiled: August 27, 2020Publication date: February 18, 2021Applicant: The Broad Institute, Inc.Inventors: Sangeeta Bhatia, Jing Shan, Anne Carpenter Van Dyk, David Logan, Nathan Ross
-
Publication number: 20210040546Abstract: 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: ApplicationFiled: September 28, 2020Publication date: February 11, 2021Applicants: The Broad Institute, Inc., Massachusetts Institute of Technology, President and Fellows of Harvard CollegeInventors: Feng ZHANG, Bernd ZETSCHE, Jonathan S. GOOTENBERG, Omar O. ABUDAYYEH, Ian SLAYMAKER
-
Publication number: 20210024655Abstract: Novel programmable targeting sequences and applications thereof. The targeting sequences can be engineered for binding to proteins, polypeptides, and other macromolecules.Type: ApplicationFiled: July 23, 2020Publication date: January 28, 2021Applicants: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGYInventors: Han Altae-Tran, Linyi Gao, Feng Zhang
-
Publication number: 20210009986Abstract: The disclosure provides for systems, methods, and compositions for targeting and editing nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a RNA-targeting Cas13 protein, at least one guide molecule, and at least one adenosine deaminase protein or catalytic domain thereof.Type: ApplicationFiled: May 13, 2020Publication date: January 14, 2021Applicants: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY, PRESIDENT AND FELLOWS OF HARVARD COLLEGEInventors: Feng Zhang, Jonathan Gootenberg, David Benjamin Turitz Cox, Omar Abudayyeh, Soumya Kannan
-
Publication number: 20210009972Abstract: The invention provides for systems, methods, and compositions for targeting and editing nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a RNA-targeting Cas13 protein, at least one guide molecule, and at least one adenosine deaminase protein or catalytic domain thereof.Type: ApplicationFiled: October 4, 2018Publication date: January 14, 2021Applicants: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY, PRESIDENT AND FELLOWS OF HARVARD COLLEGEInventors: Feng Zhang, Jonathan Gootenberg, Omar Abudayyeh
-
Patent number: 10876100Abstract: Disclosed and claimed are mutation(s) or modification(s) of the CRISPR enzyme, for example a Cas enzyme such as a Cas9, which obtain an improvement, for instance a reduction, as to off-target effects of a CRISPR-Cas or CRISPR-enzyme or CRISPR-Cas9 system or complex containing or including such a mutated or modified Cas or CRISPR enzyme or Cas9. Methods for making and using and uses of such mutated or modified Cas or CRISPR enzyme or Cas9 and systems or complexes containing the same and products from such methods and uses are also disclosed and claimed.Type: GrantFiled: December 16, 2017Date of Patent: December 29, 2020Assignees: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGYInventors: Feng Zhang, Linyi Gao, Bernd Zetsche, Ian Slaymaker
-
Publication number: 20200399626Abstract: The disclosure provides methods and compositions for treating blood diseases/disorders, such as sickle cell disease, hemochromatosis, hemophilia, and beta-thalassemia. For example the disclosure provides therapeutic guide RNAs that target the promotor of HBG1/2 to generate point mutations that increase expression of fetal hemoglobin. As another example, the disclosure provides therapeutic guide RNAs that target mutations in HBB, Factor VIII, and HFE to treat sickle cell disease, beta-thalassemia, hemophilia and hemochromatosis. The disclosure also provides fusion proteins comprising a Cas9 (e.g., a Cas9 nickase) domain and adenosine deaminases that deaminate adenosine in DNA. In some embodiments, the fusion proteins are in complex with nucleic acids, such as guide RNAs (gRNAs), which target the fusion proteins to a DNA sequence (e.g., an HBG1 or HBG2 protmoter sequence, or an HFE, GBB, or F8 gene sequence).Type: ApplicationFiled: October 16, 2018Publication date: December 24, 2020Applicants: The Broad Institute, Inc., President and Fellows of Flarvard College, Beam Therapeutics, Inc.Inventors: David R. Liu, Nicole Gaudelli, Michael S. Packer, Gregory Newby
-
Publication number: 20200399276Abstract: Provided herein are compounds of the formula (I): as well as pharmaceutically acceptable salts thereof, wherein the substituents are as those disclosed in the specification. These compounds, and the pharmaceutical compositions containing them, are useful for the treatment of malaria.Type: ApplicationFiled: June 29, 2020Publication date: December 24, 2020Applicant: THE BROAD INSTITUTE, INC.Inventors: EAMON COMER, NOBUTAKA KATO, CHRISTINA SCHERER, JESSICA BASTIEN, JEREMY DUVALL, TIMOTHY LEWIS, MORGANE SAYES, MATTHEW LEIGHTY, JUN PU, JENNIFER BEAUDOIN, BERTRAND BRAIBANT, BENITO MUNOZ
-
Patent number: 10870885Abstract: This invention relates generally to compositions and methods for identifying the regulatory network that modulates, controls or otherwise influences dendritic cell (DC) response(s), for example, dendritic cell maturation, dendritic cell antiviral response(s) and/or dendritic cell inflammatory response(s), as well compositions and methods for exploiting the regulatory network that modulates, controls or otherwise influences dendritic cell response(s) in a variety of therapeutic and/or diagnostic indications.Type: GrantFiled: September 4, 2015Date of Patent: December 22, 2020Assignees: The Broad Institute, Inc., Massachusetts Institute of Technology, President and Fellows of Harvard CollegeInventors: Aviv Regev, Alexander K. Shalek, Rahul Satija, Hongkun Park
-
Publication number: 20200392473Abstract: Embodiments herein include engineered CRISPR-Cas effector proteins that comprise at least one modification compared to an unmodified CRISPR-Cas effector protein that enhances binding of the CRISPR complex to the binding site and/or alters editing preference as compared to wild type. In certain embodiments, the CRISPR-Cas effector protein is a Type V-B effector protein, e.g., C2c1. Embodiments also include viral vectors for delivery of CRISPR-Cas effector proteins, including C2c1. In certain embodiments, the vectors allow packaging of the CRISPR-Cas effector protein within a single vector. The disclosure also includes delivery vectors, constructs, and methods of delivering larger genes for systemic delivery.Type: ApplicationFiled: December 21, 2018Publication date: December 17, 2020Applicants: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGYInventors: Feng Zhang, Bernd Zetsche, Jonathan Strecker
-
Publication number: 20200392482Abstract: A diversity-generating system comprising: a polynucleotide comprising: a variable region within a sequence encoding a protein or polypeptide, wherein the system is configured to generate a library of diversified molecules with an efficiency of 80% as measured by number of the diversified molecules in total number of molecules. The system may further comprise a template region; and a reverse transcriptase or functional domain thereof.Type: ApplicationFiled: June 17, 2020Publication date: December 17, 2020Applicants: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGYInventors: Feng Zhang, Jonathan Leo Schmid-Burgk
-
Publication number: 20200392541Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues of organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provide dare methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.Type: ApplicationFiled: February 25, 2020Publication date: December 17, 2020Applicants: The Broad Institute, Inc., Massachusetts Institute of Technology, President and Fellows of Harvard CollegeInventors: Feng ZHANG, Le Cong, Fei Ran
-
Publication number: 20200389425Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.Type: ApplicationFiled: August 25, 2020Publication date: December 10, 2020Applicants: The Broad Institute, Inc., Massachusetts Institute of Technology, The Rockefeller UniversityInventors: Sangeeta Bhatia, Charles Rice, Feng Zhang, David Benjamin Turitz Cox, Vyas Ramanan, Robert Schwartz, Amir Shlomai
-
Publication number: 20200377472Abstract: The present invention provides triazolone compounds of general formula (I): in which R1, R2, R3, R4 and R5 are as defined herein, methods of preparing said compounds, intermediate compounds useful for preparing said compounds, pharmaceutical compositions and combinations comprising said compounds and the use of said compounds for manufacturing pharmaceutical compositions for the treatment or prophylaxis of diseases, in particular of hyperproliferative disorders, as a sole agent or in combination with other active ingredients.Type: ApplicationFiled: August 18, 2020Publication date: December 3, 2020Applicants: BAYER AKTIENGESELLSCHAFT, BAYER PHARMA AKTIENGELLSCHAFT, THE BROAD INSTITUTE, INC., PRESIDENT AND FELLOWS OF HARVARD COLLEGE, THE GENERAL HOSPITAL CORPORATIONInventors: Stefan Nikolaus GRADL, Duy NGUYEN, Knut EIS, Judith GÜNTHER, Timo STELLFELD, Andreas JANZER, Sven CHRISTIAN, Thomas MÜLLER, Sherif El SHEIKH, Han Jie ZHOU, Changjia ZHAO, David B. SYKES, Steven James FERRARA, Kery LIU, Michael KRÖBER, Claudia MERZ, Michael NIEHUES, Martina SCHÄFER, Katja ZIMMERMANN, Carl Friedrich NISING
-
Publication number: 20200377912Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in prokaryotic and eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity.Type: ApplicationFiled: April 9, 2020Publication date: December 3, 2020Applicants: The Broad Institute, Inc., Massachusetts Institute of Technology, President and Fellows of Harvard CollegeInventors: Feng ZHANG, Patrick HSU, Chie-Yu LIN, Fei RAN
-
Patent number: 10851051Abstract: In one aspect, the present invention provides a method of inhibiting proliferation of a multiple myeloma cell, the method comprising contacting the cell with BRD9647, thereby inhibiting proliferation of the cell. In another aspect, the present invention provides a method of treating multiple myeloma in a pre-selected subject, the method comprising administering an effective amount of BRD9647 to the subject, wherein the subject is pre-selected by detecting a mutation in an AZIN1 polynucleotide or polypeptide relative to a reference in a biological sample obtained from the subject. In another aspect, the present invention provides a method of modulating benzoylation of an agent in a cell, the method comprising contacting the cell with BRD9647, thereby modulating benzoylation of an agent in the cell.Type: GrantFiled: August 12, 2016Date of Patent: December 1, 2020Assignees: THE BROAD INSTITUTE, INC., THE GENERAL HOSPITAL CORPORATION, PRESIDENT AND FELLOWS OF HARVARD COLLEGEInventors: Shrikanta Chattopadhyay, Stuart L. Schreiber, Zarko Boskovic
-
Patent number: 10851357Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences especially for use as to nucleotide repeat disorders. Provided are delivery systems and tissues or organ which are targeted as sites for delivery especially for use as to nucleotide repeat disorders. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex or system especially for use as to nucleotide repeat disorders, as well as methods for the design and of such. Also provided are methods of directing CRISPR complex or system formation in eukaryotic cells especially for use as to nucleotide repeat disorders including with consideration of specificity for target recognition and avoidance of toxicity and editing or modifying a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.Type: GrantFiled: June 10, 2016Date of Patent: December 1, 2020Assignees: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY, UNIVERSITY OF IOWA RESEARCH FOUNDATIONInventors: Beverly Davidson, Chie-yu Lin, Edgardo Rodriguez, Feng Zhang
-
Publication number: 20200370133Abstract: The present invention features methods for characterizing mutational profiles in patients with bladder cancer.Type: ApplicationFiled: August 7, 2020Publication date: November 26, 2020Applicants: The Broad Institute, Inc., The General Hospital Corporation, Baylor College of Medicine, The Brigham and Women?s Hospital, Inc., Northwestern University, The Johns Hopkins University, United States Government as represented by the U.S. Department of Veterans AffairsInventors: Jaegil Kim, Gad Getz, Seth Paul Lerner, David Kwiatkowski, Joshua Meeks, Joaquim Bellmunt, David McConkey