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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":"2023","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 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 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":"Ophthalmology","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"CTG","graph1":"Ophthalmology","graph2":"Preclinical","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Design Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Design Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Renaissance Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Design Therapeutics \/ Renaissance Capital","highestDevelopmentStatusID":"2","companyTruncated":"Design Therapeutics \/ Renaissance Capital"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"DT-168","moa":"TCF4 gene expression","graph1":"Ophthalmology","graph2":"Phase I","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Ophthalmic Solution","sponsorNew":"Design Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Design Therapeutics \/ Inapplicable"}]

    Find Clinical Drug Pipeline Developments & Deals by Design Therapeutics

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                            01

                            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 : DT-168

                            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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                            02

                            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 : DT-216

                            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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                            03

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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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                            04

                            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 : DT-216

                            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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                            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 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 : DT-216

                            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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                            06

                            Design Therapeutics

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Discovery

                            Sponsor : Renaissance Capital

                            Deal Size : $228.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Preclinical Genetic Disease Biotech Design Therapeutics Sets Terms for $228 Million IPO

                            Details : Design Therapeutics is developing novel small-molecule therapies, called gene targeted chimeras (GeneTACs), that are designed to be disease-modifying and target the underlying cause of inherited nucleotide repeat expansion diseases.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            March 22, 2021

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Discovery

                            Sponsor : Renaissance Capital

                            Deal Size : $228.0 million

                            Deal Type : Public Offering

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