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:
August 2, 2019
Publication date:
October 7, 2021
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 increases FXN expression. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Application
Filed:
August 2, 2019
Publication date:
October 7, 2021
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:
Grant
Filed:
March 18, 2021
Date of Patent:
September 7, 2021
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 disease allele associated with muscle disease. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Application
Filed:
August 2, 2019
Publication date:
August 26, 2021
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Jason P. Rhodes
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:
March 18, 2021
Publication date:
July 29, 2021
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 activity of a disease allele associated with muscle disease. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Type:
Application
Filed:
March 18, 2021
Publication date:
July 29, 2021
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Jason P. Rhodes
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:
March 18, 2021
Publication date:
July 22, 2021
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:
March 18, 2021
Publication date:
July 8, 2021
Applicant:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden