[{"orgOrder":0,"company":"LFB Group","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Coagulation Factor VIIa, Recombinant","moa":"Factor VIIa","graph1":"Genetic Disease","graph2":"Phase III","graph3":"LFB Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"LFB Group \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"LFB Group \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Alpha Blue Ocean","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2021","type":"Financing","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Alpha Blue Ocean","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Alpha Blue Ocean"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Neovacs","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Financing","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Neovacs","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Neovacs"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Odiparcil","moa":"Glycosaminoglycans","graph1":"Genetic Disease","graph2":"Phase 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Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Odiparcil","moa":"Glycosaminoglycans","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inventiva Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Inventiva Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Inventiva Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic 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Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Givosiran","moa":"5-aminolevulinate synthase mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"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"}]

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                          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

                          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

                          05

                          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

                          06

                          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

                          07

                          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

                          08

                          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

                          09

                          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

                          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

                          February 03, 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