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PGN-EDO51 blocks the inclusion of exon 51, restoring functional, dystrophin production. Today it received U.S. FDA orphan drug and rare pediatric disease designations for the treatment of duchenne muscular dystrophy.


Lead Product(s): PGN-EDO51

Therapeutic Area: Genetic Disease Product Name: PGN-EDO51

Highest Development Status: Phase IProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 13, 2024

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PGN-EDO51, PepGen’s lead clinical candidate for the treatment of DMD, utilizes the Enhanced Delivery Oligonucleotide (EDO) technology to deliver a therapeutic oligonucleotide. It is under phase 2 clinical development for the treatment of Duchenne Muscular Dystrophy.


Lead Product(s): PGN-EDO51

Therapeutic Area: Genetic Disease Product Name: PGN-EDO51

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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PGN-EDODM1 is an investigational candidate designed to deliver a peptide-conjugated antisense oligonucleotide (ASO) to restore cellular function. It is under development for the treatment of Myotonic Dystrophy Type 1.


Lead Product(s): PGN-EDODM1

Therapeutic Area: Genetic Disease Product Name: PGN-EDODM1

Highest Development Status: Phase IProduct Type: Peptide

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 net proceeds will be used for the development of PGN-EDO51, which is designed to skip exon 51 of the dystrophin transcript for the treatment of Duchenne muscular dystrophy.


Lead Product(s): PGN-EDO51

Therapeutic Area: Genetic Disease Product Name: PGN-EDO51

Highest Development Status: Phase IIProduct Type: Large molecule

Partner/Sponsor/Collaborator: RA Capital Management

Deal Size: $80.1 million Upfront Cash: Undisclosed

Deal Type: Public Offering February 07, 2024

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PGN-EDO51, PepGen’s lead clinical candidate for the treatment of DMD, utilizes the Enhanced Delivery Oligonucleotide (EDO) technology to deliver a therapeutic oligonucleotide. It is under phase 2 clinical development for the treatment of Duchenne Muscular Dystrophy.


Lead Product(s): PGN-EDO51

Therapeutic Area: Genetic Disease Product Name: PGN-EDO51

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 January 08, 2024

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PGN-EDODM1 is an investigational candidate designed to deliver a peptide-conjugated antisense oligonucleotide (ASO) to restore cellular function. It is under phase 1 clinical development for the treatment of Myotonic Dystrophy Type 1.


Lead Product(s): PGN-EDODM1

Therapeutic Area: Genetic Disease Product Name: PGN-EDODM1

Highest Development Status: Phase IProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable December 18, 2023

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PGN-EDODM1 is an investigational candidate designed to deliver a peptide-conjugated antisense oligonucleotide (ASO) to restore cellular function, it is being investigated for the treatment of myotonic dystrophy type 1.


Lead Product(s): PGN-EDODM1

Therapeutic Area: Genetic Disease Product Name: PGN-EDODM1

Highest Development Status: IND EnablingProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 12, 2023

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PGN-EDODM1, delivers a peptide conjugated antisense oligonucleotide (ASO) to restore cellular function, it is being investigated for the treatment of myotonic dystrophy type 1.


Lead Product(s): PGN-EDODM1

Therapeutic Area: Genetic Disease Product Name: PGN-EDODM1

Highest Development Status: IND EnablingProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 06, 2023

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PGN-EDODM1, delivers a peptide conjugated antisense oligonucleotide (ASO) to restore cellular function, it is being investigated for the treatment of myotonic dystrophy type 1.


Lead Product(s): PGN-EDODM1

Therapeutic Area: Genetic Disease Product Name: PGN-EDODM1

Highest Development Status: PreclinicalProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 13, 2023

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PGN-EDODM1, delivers a peptide conjugated antisense oligonucleotide (ASO) to restore cellular function, it is being investigated for the treatment of myotonic dystrophy type 1.


Lead Product(s): PGN-EDODM1

Therapeutic Area: Genetic Disease Product Name: PGN-EDODM1

Highest Development Status: PreclinicalProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable May 30, 2023

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