Patents Assigned to The Broad Institute
  • Publication number: 20250189529
    Abstract: Provided herein are biomarkers and combinations of biomarkers for use in manufacturing panels for determining whether a patient is at risk for multiple myeloma precursor disease progression. Also provided herein are methods of treatment for subjects identified as being at risk for multiple myeloma precursor disease progression.
    Type: Application
    Filed: December 6, 2024
    Publication date: June 12, 2025
    Applicants: Dana-Farber Cancer Institute, Inc., The Broad Institute, Inc., The General Hospital Corporation
    Inventors: Elizabeth Lightbody, Irene M. Ghobrial, Steven Carr, Denkanikota R. Mani, Michael Gillette
  • Publication number: 20250179453
    Abstract: 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: Application
    Filed: October 3, 2024
    Publication date: June 5, 2025
    Applicants: The Broad Institute, Inc., Massachusetts Institute of Technology
    Inventors: Feng ZHANG, Linyi GAO, Bernd ZETSCHE, Ian SLAYMAKER
  • Publication number: 20250171806
    Abstract: Provided are engineered viral vectors, compositions comprising viral vectors, and methods of producing and using engineered viral vectors. The engineered viral vectors provided herein include capsid proteins derived from densovirus (DNV). Such DNV capsid proteins can assemble in mammalian cells and encapsulate adeno-associated virus (AAV) and/or DNV genomic DNA. The engineered viral vectors may be used as delivery vectors in target gene therapies.
    Type: Application
    Filed: November 6, 2024
    Publication date: May 29, 2025
    Applicants: The Broad Institute, Inc., Massachusetts Institute of Technology
    Inventors: Feng ZHANG, Victoria MADIGAN
  • Publication number: 20250154528
    Abstract: The invention provides adeno-associated viral vectors and methods of using such vectors for cell transduction.
    Type: Application
    Filed: January 31, 2023
    Publication date: May 15, 2025
    Applicant: The Broad Institute, Inc.
    Inventors: Ken Y. CHAN, Benjamin E. DEVERMAN, FatmaElzahraa Abdelmouty EID
  • Patent number: 12297436
    Abstract: The invention provides for systems, methods, and compositions for targeting and editing nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA-targeting systems comprising a DNA-targeting Cpf1 protein, at least one guide molecule, and at least one cytidine deaminase protein or catalytic domain thereof.
    Type: Grant
    Filed: May 18, 2018
    Date of Patent: May 13, 2025
    Assignees: The Broad Institute, Inc., Massachusetts Institute of Technology
    Inventor: Feng Zhang
  • Publication number: 20250136599
    Abstract: Compounds of formula (I), formula (I), processes for their production and their use as pharmaceuticals.
    Type: Application
    Filed: November 15, 2024
    Publication date: May 1, 2025
    Applicants: Bayer Aktiengesellschaft, The Broad Institute, Inc., Dana-Farber Cancer Institute, Inc.
    Inventors: Stephan Siegel, Franziska Siegel, Volker Schulze, Markus Berger, Keith Graham, Detlev Sülzle, Ulf Bömer, Daniel Korr, Jens Schröder, Ursula Mönning, Michael Niehues, Matthew Meyerson, Heidi Greulich, Bethany Kaplan
  • Publication number: 20250127768
    Abstract: The subject matter disclosed herein is generally directed to treating cancers sensitive to phosphate dysregulation with inhibitors of inositol pyrophosphate (PP-InsP) synthesis, in particular, inhibitors of inositol hexakisphosphate kinases IP6Ks.
    Type: Application
    Filed: December 26, 2024
    Publication date: April 24, 2025
    Applicant: The Broad Institute, Inc.
    Inventors: Todd GOLUB, Daniel BONDESON
  • Publication number: 20250129400
    Abstract: The present disclosure provides compositions, reagents, and methods for producing capped, circular RNA molecules, circularized RNA molecules, and in particular, circularized mRNA molecules encoding a polypeptide such as a therapeutic protein.
    Type: Application
    Filed: December 23, 2024
    Publication date: April 24, 2025
    Applicants: The Broad Institute, Inc., Massachusetts Institute of Technology
    Inventors: Xiao WANG, Hongyu CHEN, Abhishek ADITHAM
  • Patent number: 12281338
    Abstract: Some aspects of this disclosure provide strategies, systems, reagents, methods, and kits that are useful for the targeted editing of nucleic acids or the modification of nucleic acids or proteins, including editing a single site within the genome of a cell or subject, e.g., within the human genome. In some embodiments, fusion proteins of nucleic acid programmable DNA binding proteins e.g., GeoCas9 or variants thereof, and effector domains, e.g., deaminase domains, are provided. In some embodiments, methods for targeted nucleic acid editing or protein modification are provided. In some embodiments, reagents and kits for the generation of targeted nucleic acid editing proteins, e.g., fusion proteins of a GeoCas9 and effector domains, are provided.
    Type: Grant
    Filed: October 29, 2019
    Date of Patent: April 22, 2025
    Assignees: The Broad Institute, Inc., President and Fellows of Harvard College
    Inventors: David R. Liu, Yongjoo Kim
  • Patent number: 12281303
    Abstract: Compositions and methods are provided herein for conducting prime editing of a target DNA molecule (e.g., a genome) that enables the incorporation of a nucleotide change and/or targeted mutagenesis. The compositions include fusion proteins comprising nucleic acid programmable DNA binding proteins (napDNAbp) and a polymerase (e.g., reverse transcriptase), which is guided to a specific DNA sequence by a modified guide RNA, named an PEgRNA. The PEgRNA has been altered (relative to a standard guide RNA) to comprise an extended portion that provides a DNA synthesis template sequence which encodes a single strand DNA flap which is synthesized by the polymerase of the fusion protein and which becomes incorporated into the target DNA molecule.
    Type: Grant
    Filed: May 31, 2023
    Date of Patent: April 22, 2025
    Assignees: The Broad Institute, Inc., President and Fellows of Harvard College
    Inventors: David R. Liu, Andrew Vito Anzalone, James William Nelson
  • Publication number: 20250120676
    Abstract: Echocardiography deep learning and cardiovascular outcomes are described. An echocardiogram analysis module may include a deep learning model to generate a video output for an input echocardiogram video, the deep learning model comprising a convolutional neural network and at least one dense layer. The echocardiogram analysis module may further include a cardiac prediction generator to generate a cardiac prediction based on video outputs generated for a plurality of input echocardiogram videos of an echocardiogram study, the cardiac prediction comprising a measurement prediction or a classification prediction.
    Type: Application
    Filed: October 11, 2024
    Publication date: April 17, 2025
    Applicants: The General Hospital Corporation, Beth Israel Deaconess Medical Center, Inc., The Broad Institute, Inc.
    Inventors: Patrick T. Ellinor, II, Emily S. Lau, Jennifer Ho, Mostafa Al-Alusi, Paolo Di Achille, Puneet Batra, Steven Lubitz
  • Publication number: 20250115901
    Abstract: Compositions and methods are provided herein for conducting prime editing of a target DNA molecule (e.g., a genome) that enables the incorporation of a nucleotide change and/or targeted mutagenesis. The compositions include fusion proteins comprising nucleic acid programmable DNA binding proteins (napDNAbp) and a polymerase (e.g., reverse transcriptase), which is guided to a specific DNA sequence by a modified guide RNA, named an PEgRNA. The PEgRNA has been altered (relative to a standard guide RNA) to comprise an extended portion that provides a DNA synthesis template sequence which encodes a single strand DNA flap which is synthesized by the polymerase of the fusion protein and which becomes incorporated into the target DNA molecule.
    Type: Application
    Filed: April 25, 2024
    Publication date: April 10, 2025
    Applicants: The Broad Institute, Inc., President and Fellows of Harvard College
    Inventors: David R. Liu, Andrew Vito Anzalone, James William Nelson
  • Publication number: 20250109177
    Abstract: Aspects of the disclosure relate to compositions and methods for targeted protein degradation. In some embodiments, the disclosure relates to methods of evolving protein degrons to interact with certain small molecule inducers (e.g., VS-777, PT-179, or PK-1016). In some embodiments, the disclosure relates to compositions (e.g., peptides, nucleic acids encoding the protein degrons, etc.) used for targeted protein degradation. In some embodiments, the disclosure relates to methods of degrading a target polypeptide in a cell.
    Type: Application
    Filed: December 12, 2024
    Publication date: April 3, 2025
    Applicants: The Broad Institute, Inc., President and Fellows of Harvard College, The Brigham and Women's Hospital, Inc.
    Inventors: David R. Liu, Amit Choudhary, Jaron August McClure Mercer, Stephan DeCarlo, Praveen Tiwari, Praveen Kokkonda, Veronika Shoba, Arghya Deb, Sreekanth Vedagopuram
  • Publication number: 20250109165
    Abstract: Provided herein are compounds of Formula (I-A), (I-B), or (I-C), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically enriched forms, prodrugs, or mixtures thereof, and compositions thereof. Also provided are methods and kits involving the inventive compounds or compositions for treating and/or preventing diseases and/or conditions (e.g., neurological disease (e.g., Alzheimer's disease, multiple sclerosis, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis), metabolic disorder (e.g., obesity, diabetes, X-linked adrenoleukodystrophy (X-ALD)), proliferative disease (e.g., cancers), hepatic disease (e.g., liver cirrhosis), conditions associated with autophagy (e.g., neurodegenerative disease, infection, cancer, conditions associated with aging, heart disease), conditions associated with aging, conditions associated with modulating the mPTP, cardiovascular conditions (e.g.
    Type: Application
    Filed: December 9, 2024
    Publication date: April 3, 2025
    Applicants: The Broad Institute, Inc., President and Fellows of Harvard College
    Inventors: David R. Liu, Alexander A. Peterson
  • Patent number: 12264367
    Abstract: Described herein are methods and uses thereof for in vivo evaluating functions of multiple genes in parallel by combining in utero genetic perturbation of progenitor cells and single-cell transcriptomic profiling of progeny cells in animals. These methods can be used, among other things, to reveal in vivo gene functions in a cell type-specific manner.
    Type: Grant
    Filed: January 29, 2021
    Date of Patent: April 1, 2025
    Assignees: The Broad Institute, Inc., Massachusetts Institute of Technology, President and Fellows of Harvard College
    Inventors: Xin Jin, Paola Arlotta, Aviv Regev, Feng Zhang, Sean Simmons
  • Publication number: 20250101395
    Abstract: The present disclosure provides Cas protein variants comprising one or more amino acid substitutions relative to wild-type Cas14a1. Fusion proteins comprising the Cas protein variants described herein are also provided by the present disclosure. Further provided herein are methods for modifying a target nucleic acid using the Cas proteins and fusion proteins provided herein. The present disclosure also provides guide RNAs, complexes, polynucleotides, systems, cells, kits, and pharmaceutical compositions.
    Type: Application
    Filed: December 6, 2024
    Publication date: March 27, 2025
    Applicants: The Broad Institute, Inc., President and Fellows of Harvard College
    Inventors: David R. Liu, Aditya Raguram
  • Publication number: 20250101468
    Abstract: 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: Application
    Filed: June 11, 2024
    Publication date: March 27, 2025
    Applicants: The Broad Institute, Inc., Massachusetts Institute of Technology, President and Fellows of Harvard College
    Inventors: Feng ZHANG, Le CONG, Fei RAN
  • Publication number: 20250101500
    Abstract: Described in various embodiments herein are tiled amplification nucleic acid detection systems and uses thereof. In some embodiments, the nucleic acids amplified and detected are cell free DNA (cfDNA).
    Type: Application
    Filed: October 4, 2024
    Publication date: March 27, 2025
    Applicants: The Broad Institute, Inc., Massachusetts Institute of Technology, THE GENERAL HOSPITAL CORPORATION
    Inventors: Deborah HUNG, Gowtham THAKKU, Sharon WONG, James GOMEZ
  • Patent number: 12258323
    Abstract: The present invention provides dihydrooxydiazinone compounds of general formula (I): in which R1, R2, R3, and R4, 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 diseases, as a sole agent or in combination with other active ingredients.
    Type: Grant
    Filed: July 11, 2022
    Date of Patent: March 25, 2025
    Assignees: Bayer Aktiengesellschaft, Bayer Pharma Aktiengesellschaft, The Broad Institute, Inc., Dana-Farber Cancer Institute, Inc.
    Inventors: Manuel Ellermann, Stefan Nikolaus Gradl, Charlotte Christine Kopitz, Martin Lange, Adrian Tersteegen, Philip Lienau, Christa Hegele-Hartung, Detlev Sülzle, Timothy A. Lewis, Heidi Greulich, Xiaoyun Wu, Matthew Meyerson, Alex Burgin
  • Publication number: 20250092397
    Abstract: The disclosure features compositions, systems, and methods for preparation and use of efficient RNA nuclear export of ribozyme-assisted circular RNA molecules (racRNAs). In embodiments, the methods involve characterizing a cell or tissue using racRNAs.
    Type: Application
    Filed: November 22, 2024
    Publication date: March 20, 2025
    Applicants: The Broad Institute, Inc., Massachusetts Institute of Technology
    Inventors: Hailing Shi, Yiming Zhou, Xiao Wang