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Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"PF-06741086","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Pfizer Inc \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Inapplicable"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inozyme Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Inozyme Pharma \/ Inapplicable"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"KalVista Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"KalVista Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Aryogen Pharmed","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRAN","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Eptacog Alfa","moa":"Coagulation factor VII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Aryogen Pharmed","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aryogen Pharmed \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Aryogen Pharmed \/ Inapplicable"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Group \/ Chiesi Group","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Chiesi Group"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Velmanase Alfa","moa":"High mannose oligosaccharides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Group \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Inapplicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Troriluzole","moa":"EAAT2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biohaven Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"TAK-881","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Virchow Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Immunoglobulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Virchow Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Virchow Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Virchow Group \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Zodasiran","moa":"ANGPTL3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Beijing Suncadia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"SHR-1918","moa":"ANGPTL3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Beijing Suncadia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beijing Suncadia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Beijing Suncadia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Atumelnant","moa":"MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Asklepion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Citrulline","moa":"G-protein coupled receptor GPCR33 (GPRC6A)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Asklepion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Asklepion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Asklepion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Rilzabrutinib","moa":"Tyrosine-protein kinase BTK","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Clinical Research Consultants, Inc.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Vitamins\/Minerals\/Inorganic 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 Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Vonicog Alfa","moa":"von Willebrand factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Vega Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic 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 Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Licensing Agreement","leadProduct":"Navenibart","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Kaken Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Kaken Pharmaceuticals"},{"orgOrder":0,"company":"Visirna","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Visirna","amount2":0.40000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic 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"}]

Find Drugs for Genetic Disease in Phase III Clinical Development

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Rilzabrutinib

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Rilzabrutinib is a Other Small Molecule drug candidate, which is currently being evaluated in Phase III clinical studies for the treatment of Anemia, Sickle Cell.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          May 16, 2025

                          Lead Product(s) : Rilzabrutinib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          April 04, 2023

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophili...

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          January 25, 2023

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          04

                          Details : Vertex will have the rights to conduct research using ImmunoGen's ADC technology to discover novel targeted conditioning agents for use with gene editing. CTX001 (exagamglogene autotemcel) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edite...

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : $15.0 million

                          January 03, 2023

                          Lead Product(s) : Exagamglogene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Vertex Pharmaceuticals

                          Deal Size : $352.0 million

                          Deal Type : Licensing Agreement

                          Immunogen

                          05

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          December 22, 2022

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          06

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with hemophilia A.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          November 30, 2022

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          07

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Swedish Orphan Biovitrum AB

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Investigational once-weekly BIVV001 (efanesoctocog alfa) prophylaxis met the primary efficacy endpoint providing clinically meaningful bleed protection for people with severe hemophilia A.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          October 07, 2022

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Swedish Orphan Biovitrum AB

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          08

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          October 07, 2022

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          09

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Swedish Orphan Biovitrum AB

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : The study met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A receiving weekly prophylaxis with BIVV001 (efanesoctocog alfa) over a period of 52 weeks.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          September 03, 2022

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Swedish Orphan Biovitrum AB

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          10

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 15, 2022

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner