[{"orgOrder":0,"company":"Poxel","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Poxel Announces PXL770 Granted Orphan Drug Designation from the U.S. FDA for Autosomal-Dominant Polycystic Kidney Disease (ADPKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Poxel"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Bpifrance","pharmaFlowCategory":"D","amount":"$8.6 million","upfrontCash":"Undisclosed","newsHeadline":"ATA-200, Atamyo Therapeutics\u2019 Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C\/R5, Reaches Key Milestones with the Filing of a Clinical Trial Application in Europe and a Non-Dilutive Financing from France 2030 Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atamyo Therapeutics"}]
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The financing will be used to support company's clinical trials and manufacturing development programs, including ATA-200 gene therapy targeting γ-sarcoglycan (SGCG) related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5).
PXL770 is a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator. AMPK is a central regulator of multiple metabolic pathways leading to the control of lipid metabolism, glucose homeostasis and inflammation.