Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Application
Filed:
October 16, 2020
Publication date:
May 9, 2024
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. QATANANI, Timothy WEEDEN, Cody A. DESJARDINS
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Grant
Filed:
October 26, 2023
Date of Patent:
April 30, 2024
Assignee:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins, Brendan Quinn, John Najim
Abstract: The present application relates to oligonucleotides (e.g., antisense oligonucleotides such as gapmers) designed to target DMPK RNAs and targeting complexes for delivering the oligonucleotides to cells (e.g., muscle cells) and uses thereof, particularly uses relating to treatment of disease. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DMPK.
Type:
Application
Filed:
December 30, 2021
Publication date:
April 11, 2024
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Timothy Weeden, Cody A. Waltham, Stefano Zanotti, Kim Tang, Mohammed T. Qatanani, Brendan Quinn, John Najim
Abstract: Aspects of the disclosure relate to oligonucleotides (e.g., RNAi oligonucleotides such as siRNAs) designed to target DUX4 RNAs and targeting complexes for delivering the oligonucleotides to cells (e.g., muscle cells) and uses thereof, particularly uses relating to treatment of disease (e.g., FSHD).
Type:
Application
Filed:
December 30, 2021
Publication date:
April 4, 2024
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Duncan Brown, Nelson Hsia, Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins, Brendan Quinn, John Najim
Abstract: Provided herein are oligonucleotide-antibody complexes, methods of preparing the complexes, and methods of using the complexes (e.g., treating muscle diseases). In particular, provided are compounds of Formula (I) and various methods for the preparation of the compounds. Also provided are pharmaceutical compositions comprising compounds of Formula (I) and methods of treating muscle disease in a subject by administering a compound or composition described herein.
Type:
Application
Filed:
December 3, 2021
Publication date:
March 28, 2024
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Scott Hilderbrand, John Najim, Qifeng Qiu, Benjamin Vieira, Timothy Weeden, Sean Spring
Abstract: Aspects of the disclosure relate to complexes and other aspects relate to formulations (e.g., aqueous, lyophilized forms) comprising such complexes comprising an oligonucleotide (e.g., useful for targeting DMPK) covalently linked to an antibody (e.g., anti-TfR1 antibody).
Type:
Grant
Filed:
April 19, 2023
Date of Patent:
March 19, 2024
Assignee:
Dyne Therapeutics, Inc.
Inventors:
Scott Hilderbrand, Timothy Weeden, John Najim, Stefano Zanotti, Romesh R. Subramanian, Mohammed T. Qatanani, Cody A. Desjardins, Kim Tang, Brendan Quinn
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.
Type:
Application
Filed:
October 24, 2023
Publication date:
February 29, 2024
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Application
Filed:
September 15, 2023
Publication date:
February 29, 2024
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Application
Filed:
September 15, 2023
Publication date:
February 29, 2024
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Application
Filed:
October 26, 2023
Publication date:
February 29, 2024
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins, Brendan Quinn, John Najim
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Grant
Filed:
March 18, 2021
Date of Patent:
February 27, 2024
Assignee:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins
Abstract: Aspects of the disclosure relate to complexes and other aspects relate to formulations (e.g., aqueous, lyophilized forms) comprising such complexes (e.g., wherein each complex is of the exemplary formula shown below) comprising a phosphorodiamidate morpholino oligomer (e.g., useful for targeting DMD) covalently linked to an antibody (e.g., anti-TfR1 antibody). In some embodiments, the complexes are formulated with histidine (e.g., L-histidine) and sucrose at a specified pH (e.g., about 5.0 to 7.0). Also provided are uses of these formulations for treating a subject having a mutated DMD allele associated with Duchenne Muscular Dystrophy.
Type:
Application
Filed:
August 24, 2023
Publication date:
January 18, 2024
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Timothy Weeden, Scott Hilderbrand, Sean Spring, Peiyi Shen, Cody A. Desjardins, Romesh R. Subramanian, Mohammed T. Qatanani, Brendan Quinn, John Najim
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Application
Filed:
July 9, 2021
Publication date:
January 18, 2024
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins, Brendan Quinn, John Najim
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Grant
Filed:
June 6, 2023
Date of Patent:
December 19, 2023
Assignee:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins, Brendan Quinn, John Najim
Abstract: Aspects of the disclosure relate to antibodies that bind to transferrin receptor (e.g., transferrin receptor 1) and complexes comprising the antibody covalently linked to a molecular payload. Methods of using the antibodies are also provided.
Type:
Grant
Filed:
March 10, 2023
Date of Patent:
December 12, 2023
Assignee:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins, Brendan Quinn, John Najim
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.
Type:
Grant
Filed:
March 10, 2023
Date of Patent:
December 5, 2023
Assignee:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits activity of a pro-atrophy gene. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Application
Filed:
July 9, 2021
Publication date:
November 2, 2023
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins, Brendan Quinn, John Najim
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.
Type:
Application
Filed:
July 10, 2023
Publication date:
November 2, 2023
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins, Brendan Quinn, John Najim
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Grant
Filed:
March 16, 2023
Date of Patent:
October 24, 2023
Assignee:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden
Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Grant
Filed:
March 16, 2023
Date of Patent:
October 24, 2023
Assignee:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden