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    [{"orgOrder":0,"company":"TikunTX","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"BPN-36964","moa":"ELP1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"TikunTX","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"TikunTX \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"TikunTX \/ Inapplicable"}]

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                            Lead Product(s) : BPN-36964

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Tikun Gets FDA Orphan, Rare Pediatric Designation for Dysautonomia

                            Details : BPN-36964 is a gene replacement therapy and a small molecule splicing modulator, being investgated for systemic treatment of familial dysautonomia.

                            Product Name : BPN-36964

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 23, 2025

                            Lead Product(s) : BPN-36964

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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