[{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"The Dion Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Partnership","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ The Dion Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ The Dion Foundation"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"VTX-801","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Vivet Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"SAR444836","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"VTX-801","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vivet Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Vivet Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"VTX-801","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vivet Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"7","companyTruncated":"Vivet Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"SAR446268","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Bpifrance","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Bpifrance","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Bpifrance"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Coave Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Laboratoires Thea","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Injection","sponsorNew":"Coave Therapeutics \/ Laboratoires Thea","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Laboratoires Thea"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"GS030","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GenSight Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GenSight Biologics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GenSight Biologics \/ Inapplicable"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"GS030","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GenSight Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GenSight Biologics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GenSight Biologics \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"MRT5005","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"MRT5005","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"SparingVision","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Inapplicable"},{"orgOrder":0,"company":"SparingVision","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Inapplicable"},{"orgOrder":0,"company":"SparingVision","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Inapplicable"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Coave Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"SparingVision","sponsor":"4BIO Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Financing","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Injection","sponsorNew":"SparingVision \/ 4BIO Capital","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ 4BIO Capital"},{"orgOrder":0,"company":"SparingVision","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Inapplicable"},{"orgOrder":0,"company":"SparingVision","sponsor":"Jeito Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Series B Financing","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Jeito Capital","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Jeito Capital"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase 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Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Inapplicable"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : SAR446268

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          February 25, 2025

                          Lead Product(s) : SAR446268

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : SAR444836

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          August 02, 2023

                          Lead Product(s) : SAR444836

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Lead Product(s) : MRT5005

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Response to COVID-19 pandemic anticipated to cause interruptions in certain clinical trial operations; updated interim data readout timing to be provided at later date.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          March 26, 2020

                          Lead Product(s) : MRT5005

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          04

                          Lead Product(s) : MRT5005

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          February 26, 2020

                          Lead Product(s) : MRT5005

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          05

                          Lead Product(s) : MRT5201

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          December 06, 2018

                          Lead Product(s) : MRT5201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          06

                          Lead Product(s) : MRT5005

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          December 15, 2017

                          Lead Product(s) : MRT5005

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          07

                          Lead Product(s) : Recombinant Antihemophilic Factor

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          July 02, 2017

                          Lead Product(s) : Recombinant Antihemophilic Factor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          08

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          September 18, 2015

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          09

                          Lead Product(s) : Tesevatinib

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 21, 2012

                          Lead Product(s) : Tesevatinib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          10

                          Lead Product(s) : SAR422459

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          June 07, 2011

                          Lead Product(s) : SAR422459

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner