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Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","amount":"$735.0 million","upfrontCash":"$224.0 million","newsHeadline":"Vertex and Entrada Therapeutics Establish Collaboration to Discover and Develop Endosomal Escape Vehicle (EEV) Therapeutics for Myotonic Dystrophy Type 1 (DM1)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Entrada Therapeutics Announces Clinical Hold on IND Application for ENTR-601-44 in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND 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Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"}]

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            Details:

            ENTR-601-44, a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), is the lead product candidate within Entrada’s Duchenne muscular dystrophy franchise.

            Lead Product(s): ENTR-601-44

            Therapeutic Area: Genetic Disease Product Name: ENTR-601-44

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 22, 2023

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            ENTR-601-44, a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), is the lead product candidate within its Duchenne franchise from Entrada’s growing pipeline of EEV-therapeutics.

            Lead Product(s): ENTR-601-44

            Therapeutic Area: Genetic Disease Product Name: ENTR-601-44

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 21, 2023

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            Details:

            ENTR-601-44, a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), is the lead product candidate within its Duchenne franchise from Entrada’s growing pipeline of EEV-therapeutics.

            Lead Product(s): ENTR-601-44

            Therapeutic Area: Genetic Disease Product Name: ENTR-601-44

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 01, 2023

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            Details:

            The agreement includes a four-year global research collaboration whereby Entrada will continue to advance for certain research activities related to ENTR-701 (EEV-conjugated phosphorodiamidate morpholino oligomer), as well as additional DM1-related research activities.

            Lead Product(s): EEV-conjugated Phosphorodiamidate Morpholino Oligomer

            Therapeutic Area: Genetic Disease Product Name: ENTR-701

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Vertex Pharmaceuticals

            Deal Size: $735.0 million Upfront Cash: $224.0 million

            Deal Type: Agreement February 09, 2023

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            Details:

            ENTR-601-45 is a Endosomal Escape Vehicle-conjugated phosphorodiamidate morpholino oligomer, has the potential to restore the mRNA reading frame and allow for the translation of dystrophin protein, it Duchenne muscular dystrophy patients.

            Lead Product(s): ENTR-601-45

            Therapeutic Area: Genetic Disease Product Name: ENTR-601-45

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 09, 2023

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            Details:

            The Company’s lead oligonucleotide programs include ENTR-601-44 targeting Duchenne muscular dystrophy (DMD) and ENTR-701 targeting myotonic dystrophy type 1 (DM1).

            Lead Product(s): ENTR-601-44

            Therapeutic Area: Genetic Disease Product Name: ENTR-601-44

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 19, 2022

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            Details:

            ENTR-701, a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer, is the second novel clinical candidate from Entrada’s growing pipeline of EEV therapeutics.

            Lead Product(s): EEV-conjugated Phosphorodiamidate Morpholino Oligomer

            Therapeutic Area: Genetic Disease Product Name: ENTR-701

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Vertex Pharmaceuticals

            Deal Size: $735.0 million Upfront Cash: $224.0 million

            Deal Type: Collaboration December 08, 2022

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            Details:

            New non-human primate data demonstrate a durability of response through 12 weeks for lead clinical candidate, ENTR-601-44, for the potential treatment of Duchenne muscular dystrophy.

            Lead Product(s): ENTR-601-44

            Therapeutic Area: Genetic Disease Product Name: ENTR-601-44

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 11, 2022

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            Details:

            ENTR-601-44 (Exon 44 Skipping Oligonucleotide) is being developed for patients with DMD that are exon 44 skipping amenable. We plan to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for ENTR-601-44 in 2022.

            Lead Product(s): ENTR-601-44

            Therapeutic Area: Genetic Disease Product Name: ENTR-601-44

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Goldman Sachs & Co. LLC

            Deal Size: $181.5 million Upfront Cash: Undisclosed

            Deal Type: Public Offering November 02, 2021

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            Details:

            The Company plans to use the investment proceeds to advance its diverse pipeline to the clinic. Included in this pipeline are several of Entrada’s oligonucleotide programs for the treatment of multiple neuromuscular diseases, led by Duchenne muscular dystrophy (DMD).

            Lead Product(s): Oligonucleotide antibody

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Wellington Management Company

            Deal Size: $116.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing March 31, 2021

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