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Avidity Biosciences
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AOC 1001 (Del-desiran) consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. It is being evaluated for the treatment of myotonic dystrophy type 1.


Lead Product(s): Del-desiran

Therapeutic Area: Genetic Disease Product Name: AOC 1001

Highest Development Status: Phase IIProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 04, 2024

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Avidity intends to use the net proceeds from the private placement to fund research and development of its clinical-stage product candidate AOC 1001, designed to reduce the levels of DMPK mRNA for the treatment of myotonic dystrophy type 1.


Lead Product(s): AOC 1001

Therapeutic Area: Genetic Disease Product Name: AOC 1001

Highest Development Status: Phase IIProduct Type: Large molecule

Partner/Sponsor/Collaborator: Adage Capital Partners LP

Deal Size: $400.0 million Upfront Cash: Undisclosed

Deal Type: Private Placement February 29, 2024

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AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers to skeletal muscle and heart tissue. It is being evaluated for treatment of duchenne muscular dystrophy in people with mutations amenable to exon 44 skipping.


Lead Product(s): AOC 1044

Therapeutic Area: Genetic Disease Product Name: AOC 1044

Highest Development Status: Phase IIProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 20, 2024

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The collaboration aims to focus on discovery, development and commercialization of up to five cardiovascular targets by leveraging Avidity's proprietary AOC platform technology, to deliver a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates.


Lead Product(s): RNA Therapeutic

Therapeutic Area: Cardiology/Vascular Diseases Product Name: Undisclosed

Highest Development Status: Discovery PlatformProduct Type: Large molecule

Partner/Sponsor/Collaborator: Bristol Myers Squibb

Deal Size: $2,275.0 million Upfront Cash: $100.0 million

Deal Type: Collaboration November 28, 2023

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AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. It is being investigated for myotonic dystrophy type 1.


Lead Product(s): AOC 1001

Therapeutic Area: Genetic Disease Product Name: AOC 1001

Highest Development Status: Phase IIProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 07, 2023

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AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production in people living with Duchenne muscular dystrophy.


Lead Product(s): AOC 1044

Therapeutic Area: Genetic Disease Product Name: AOC 1044

Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable August 15, 2023

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Details:

AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. It is being investigated for myotonic dystrophy type 1.


Lead Product(s): AOC 1001

Therapeutic Area: Genetic Disease Product Name: AOC 1001

Highest Development Status: Phase IIProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable May 17, 2023

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Details:

AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. It is being investigated for myotonic dystrophy type 1.


Lead Product(s): AOC 1001

Therapeutic Area: Genetic Disease Product Name: AOC 1001

Highest Development Status: Phase IIProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 27, 2023

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Details:

AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production in patients with DMD, with mutations amenable to exon 44 skipping.


Lead Product(s): AOC 1044

Therapeutic Area: Genetic Disease Product Name: AOC 1044

Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 24, 2023

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AOC 1020 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DUX4 mRNA to reduce the expression of DUX4 mRNA and DUX4 protein in muscles in patients with FSHD.


Lead Product(s): AOC 1020

Therapeutic Area: Musculoskeletal Product Name: AOC 1020

Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 14, 2023

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