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    [{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"DT-216","moa":"FXN transcription","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Design Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Design Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"DT-216","moa":"FXN transcription","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Design 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                            01

                            Design Therapeutics

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                            Lead Product(s) : DT-168

                            Therapeutic Area : Ophthalmology

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            DT-168 in Keratoplasty Patients with Fuchs Endothelial Corneal Dystrophy

                            Details : DT-168 is a Other Small Molecule drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Fuchs' Endothelial Dystrophy.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            June 17, 2025

                            Lead Product(s) : DT-168

                            Therapeutic Area : Ophthalmology

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Design Therapeutics

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                            Lead Product(s) : DT-168

                            Therapeutic Area : Ophthalmology

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Design Shows Positive Phase 1 DT-168 Data for Fuchs Corneal Dystrophy Phase 2

                            Details : DT-168 is a novel GeneTAC® small molecule, is designed to selectively target and reduce the expression of the mutant TCF4 gene that causes Fuchs endothelial corneal dystrophy.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 01, 2025

                            Lead Product(s) : DT-168

                            Therapeutic Area : Ophthalmology

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Design Therapeutics

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                            Lead Product(s) : DT-216P2

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            March 13, 2025

                            Lead Product(s) : DT-216P2

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Design Therapeutics

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                            Lead Product(s) : DT-216P2

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            January 14, 2025

                            Lead Product(s) : DT-216P2

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Design Therapeutics

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                            Lead Product(s) : DT-216

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Design Therapeutics Reports Initial Results from Phase 1 Multiple-Ascending Dose Study of DT-216 for the Treat...

                            Details : DT-216 is a GeneTAC™ small molecule designed to specifically target the GAA repeat expansion mutation, unblock the transcriptional machinery, and restore the production of functional, natural FXN mRNA.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            August 14, 2023

                            Lead Product(s) : DT-216

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Design Therapeutics

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                            Lead Product(s) : DT-216

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            October 10, 2022

                            Lead Product(s) : DT-216

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Ophthalmology

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Design Therapeutics Announces Preclinical Data Highlighting Novel GeneTAC™ Therapy for the Treatment of Fuch...

                            Details : GeneTAC™ molecules are designed to target specific genomic sequences and modulate transcription without requiring invasive procedures or gene editing, potentially offering a novel option for earlier medical treatment.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            September 03, 2022

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Ophthalmology

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Design Therapeutics

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                            Lead Product(s) : DT-216

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Design Therapeutics Reports Positive Data from Single-Ascending Dose Trial of DT-216 for the Treatment of Frie...

                            Details : The results show that DT-216 was generally well-tolerated and able to overcome the frataxin (FXN) transcription impairment that causes FA, with a greater than two-fold increase in FXN mRNA in the cohort with the highest response.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            July 12, 2022

                            Lead Product(s) : DT-216

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Design Therapeutics

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                            Lead Product(s) : DT-216

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Design Therapeutics Completes Dosing in First Patient Cohort of Phase 1 Trial of DT-216 GeneTAC Molecule for t...

                            Details : DT-216 is a novel GeneTAC™ gene targeted chimera small molecule designed to specifically target the GAA repeat expansion mutation, the underlying cause of FA, and restore frataxin (FXN) gene expression.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 30, 2022

                            Lead Product(s) : DT-216

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Design Therapeutics

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                            Lead Product(s) : DT-216

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 17, 2022

                            Lead Product(s) : DT-216

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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