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[{"orgOrder":0,"company":"Healx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Healx Receives Ind and Orphan Drug Designation for Fragile X Clinical Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"October 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Healx"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces U.S. FDA Clearance of IND Application for OTL-203 in MPS-IH","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"}]

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            Details:

            OTL-203 is an investigational hematopoietic stem cell gene therapy being developed for the treatment of MPS-IH. It uses a modified virus to insert a functional copy of the IDUA gene into a patient’s cells.

            Lead Product(s): OTL-203

            Therapeutic Area: Genetic Disease Product Name: OTL-203

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 05, 2023

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            HLX-0201 was then validated in preclinical models of fragile X, where it was found to modify several behaviours similar to those that are exhibited in patients with the condition.

            Lead Product(s): HLX-0201

            Therapeutic Area: Genetic Disease Product Name: HLX-0201

            Highest Development Status: IND Enabling Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 20, 2021

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