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Salts","year":"2025","type":"Inapplicable","leadProduct":"Riboflavin","moa":"Riboflavin kinase; Riboflavin synthase; Flavin reductase (NADPH)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Clinical Research Consultants, Inc.","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Clinical Research Consultants, Inc. \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Clinical Research Consultants, Inc. \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic 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Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"VGA039","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vega Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vega Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vega Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Eidos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Acoramidis Hydrochloride","moa":"Transthyretin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eidos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eidos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Eidos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"Del-desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2025","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Icon Plc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2025","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Icon Plc","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Icon Plc"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"AOC-1020","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Kaken Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic 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Disease","amount2New":0.40000000000000002,"dosageForm":"Injection","sponsorNew":"Visirna \/ Sanofi","highestDevelopmentStatusID":"10","companyTruncated":"Visirna \/ Sanofi"},{"orgOrder":0,"company":"Seaside Therapeutics","sponsor":"Allos Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Acquisition","leadProduct":"Baclofen","moa":"GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Seaside Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Seaside Therapeutics \/ Allos Pharma","highestDevelopmentStatusID":"10","companyTruncated":"Seaside Therapeutics \/ Allos Pharma"}]
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