[{"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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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.
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.