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    [{"orgOrder":0,"company":"Cure Rare","sponsor":"Charles River Laboratories, Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"CRD-TMH-001","moa":"Dystrophin protein","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Cure Rare","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cure Rare \/ Charles River Laboratories, Inc","highestDevelopmentStatusID":"5","companyTruncated":"Cure Rare \/ Charles River Laboratories, Inc"},{"orgOrder":0,"company":"Cure Rare","sponsor":"University of Massachusetts, Worcester","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"CRD-TMH-001","moa":"Dystrophin protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Cure Rare","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Cure Rare \/ University of Massachusetts, Worcester","highestDevelopmentStatusID":"6","companyTruncated":"Cure Rare \/ University of Massachusetts, Worcester"},{"orgOrder":0,"company":"Cure Rare","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"CRD-003","moa":"FKRP","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cure Rare","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cure Rare \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Cure Rare \/ Undisclosed"},{"orgOrder":0,"company":"Cure Rare","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"CRD-003","moa":"FKRP","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cure Rare","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cure Rare \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Cure Rare \/ Undisclosed"}]

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                            01

                            Cure Rare

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                            OTS 2025
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                            Cure Rare

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                            OTS 2025
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                            Lead Product(s) : CRD-003

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Cure Rare Disease Gets FDA Orphan Drug Tag for Gene Therapy in LGMD2i/R9

                            Details : CRD-003, is gene therapy candidate, being evaluated for the treatment of Limb-Girdle Muscular Dystrophy Type R9 caused by biallelic mutations in the FKRP gene.

                            Product Name : CRD-003

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            September 24, 2025

                            Lead Product(s) : CRD-003

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Cure Rare

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                            OTS 2025
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                            OTS 2025
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                            Lead Product(s) : CRD-003

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Cure Rare Disease Gets FDA Feedback on Limb-Girdle 2i/R9 Gene Therapy Program

                            Details : The LGMDR9 program, known as CRD-003, utilizes a novel, liver de-targeting, muscle tropic capsid. It is being evaluated for the treatment of Limb-Girdle Muscular Dystrophy type 2I/R9.

                            Product Name : CRD-003

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            June 23, 2025

                            Lead Product(s) : CRD-003

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Cure Rare

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                            Lead Product(s) : CRD-TMH-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : University of Massachusetts, Worcester

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Treatment of a Single Patient With CRD-TMH-001

                            Details : CRD-TMH-001 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Muscular Dystrophy, Duchenne.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            August 24, 2022

                            Lead Product(s) : CRD-TMH-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : University of Massachusetts, Worcester

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

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                            Lead Product(s) : CRD-TMH-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Charles River Laboratories, Inc

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Cure Rare Disease Receives FDA Approval to Administer First-in-Human CRISPR Therapeutic

                            Details : The drug, named CRD-TMH-001, treats muscle promoter and exon 1 mutations on the dystrophin gene, to stop the progression of Duchenne muscular dystrophy in a single-patient dosing.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            August 11, 2022

                            Lead Product(s) : CRD-TMH-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Charles River Laboratories, Inc

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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