[{"orgOrder":0,"company":"Virica Biotech","sponsor":"Innovation, Science and Economic Development Canada","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"Re-engineered AAV-LPL","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Virica Biotech","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Virica Biotech \/ Innovation, Science and Economic Development Canada","highestDevelopmentStatusID":"1","companyTruncated":"Virica Biotech \/ Innovation, Science and Economic Development Canada"},{"orgOrder":0,"company":"Genevant Sciences","sponsor":"2Seventy Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Cell and Gene therapy","year":"2022","type":"Licensing Agreement","leadProduct":"MegaTAL mRNA","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genevant Sciences","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genevant Sciences \/ 2Seventy Bio","highestDevelopmentStatusID":"1","companyTruncated":"Genevant Sciences \/ 2Seventy Bio"},{"orgOrder":0,"company":"CYTOO","sponsor":"Astellas Gene Therapies","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"CYTOO","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CYTOO \/ Astellas Gene Therapies","highestDevelopmentStatusID":"1","companyTruncated":"CYTOO \/ Astellas Gene Therapies"},{"orgOrder":0,"company":"Sirion Biotech","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sirion Biotech","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sirion Biotech \/ Sanofi","highestDevelopmentStatusID":"1","companyTruncated":"Sirion Biotech \/ Sanofi"},{"orgOrder":0,"company":"Genespire","sponsor":"Sofinnova Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genespire","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous","sponsorNew":"Genespire \/ Sofinnova Partners","highestDevelopmentStatusID":"1","companyTruncated":"Genespire \/ Sofinnova Partners"},{"orgOrder":0,"company":"Genespire","sponsor":"Sofinnova Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2020","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genespire","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genespire \/ Sofinnova Partners","highestDevelopmentStatusID":"1","companyTruncated":"Genespire \/ Sofinnova Partners"},{"orgOrder":0,"company":"SR-Tiget","sponsor":"Genespire","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"Microrna-Regulated Lentiviral Vectors","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"SR-Tiget","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SR-Tiget \/ Genespire","highestDevelopmentStatusID":"1","companyTruncated":"SR-Tiget \/ Genespire"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Mitsubishi Ufj Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"GtCCR4","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Daiichi Sankyo \/ Mitsubishi Ufj Capital","highestDevelopmentStatusID":"1","companyTruncated":"Daiichi Sankyo \/ Mitsubishi Ufj Capital"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Ysios Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"ABCA4 gene","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"SpliceBio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SpliceBio \/ Ysios Capital","highestDevelopmentStatusID":"1","companyTruncated":"SpliceBio \/ Ysios Capital"},{"orgOrder":0,"company":"Anjarium Biosciences","sponsor":"Abingworth","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Anjarium Biosciences","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Anjarium Biosciences \/ Abingworth","highestDevelopmentStatusID":"1","companyTruncated":"Anjarium Biosciences \/ Abingworth"},{"orgOrder":0,"company":"AavantiBio","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AavantiBio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ Sarepta Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"AavantiBio \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Affinia Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Affinia Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"Affinia Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Personalis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Personalis","highestDevelopmentStatusID":"1","companyTruncated":"Sarepta Therapeutics \/ Personalis"},{"orgOrder":0,"company":"hC Bioscience","sponsor":"ARCH Venture Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"hC Bioscience","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"hC Bioscience \/ ARCH Venture Partners","highestDevelopmentStatusID":"1","companyTruncated":"hC Bioscience \/ ARCH Venture Partners"},{"orgOrder":0,"company":"PepGen","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PepGen \/ RA Capital Management","highestDevelopmentStatusID":"1","companyTruncated":"PepGen \/ RA Capital Management"},{"orgOrder":0,"company":"Duchenne research","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Funding","leadProduct":"Undisclosed","moa":"DMD gene","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Duchenne research","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Duchenne research \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Duchenne research \/ Undisclosed"},{"orgOrder":0,"company":"AgeX Therapeutics","sponsor":"University of California, Irvine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"Neural Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AgeX Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AgeX Therapeutics \/ University of California, Irvine","highestDevelopmentStatusID":"1","companyTruncated":"AgeX Therapeutics \/ University of California, Irvine"},{"orgOrder":0,"company":"Replay Bio","sponsor":"KKR","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Replay Bio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Replay Bio \/ KKR","highestDevelopmentStatusID":"1","companyTruncated":"Replay Bio \/ KKR"},{"orgOrder":0,"company":"2Seventy Bio","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"MegaTAL mRNA","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"2Seventy Bio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"2Seventy Bio \/ Novo Nordisk","highestDevelopmentStatusID":"1","companyTruncated":"2Seventy Bio \/ Novo Nordisk"},{"orgOrder":0,"company":"Mammoth Biosciences","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Mammoth Biosciences","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mammoth Biosciences \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"Mammoth Biosciences \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arcturus Therapeutics \/ Guggenheim Securities","highestDevelopmentStatusID":"1","companyTruncated":"Arcturus Therapeutics \/ Guggenheim Securities"},{"orgOrder":0,"company":"AavantiBio","sponsor":"University of Florida","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AavantiBio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ University of Florida","highestDevelopmentStatusID":"1","companyTruncated":"AavantiBio \/ University of Florida"},{"orgOrder":0,"company":"Emendo Biotherapeutics","sponsor":"AnGes","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Series B Financing","leadProduct":"OMNI Nuclease","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Emendo Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Emendo Biotherapeutics \/ AnGes","highestDevelopmentStatusID":"1","companyTruncated":"Emendo Biotherapeutics \/ AnGes"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"CureDuchenne Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2020","type":"Financing","leadProduct":"Undisclosed","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dyne Therapeutics \/ CureDuchenne Ventures","highestDevelopmentStatusID":"1","companyTruncated":"Dyne Therapeutics \/ CureDuchenne Ventures"},{"orgOrder":0,"company":"University of Massachusetts Medical School","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Massachusetts Medical School","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Massachusetts Medical School \/ CANbridge Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"University of Massachusetts Medical School \/ CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Codexis","sponsor":"Crosswalk Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Codexis","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Codexis \/ Crosswalk Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Codexis \/ Crosswalk Therapeutics"},{"orgOrder":0,"company":"Cornell University","sponsor":"reVision Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Licensing Agreement","leadProduct":"REV-0100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Cornell University","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cornell University \/ reVision Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Cornell University \/ reVision Therapeutics"},{"orgOrder":0,"company":"Hunterian Medicine","sponsor":"Inscripta","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"MAD7 Nuclease","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Hunterian Medicine","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hunterian Medicine \/ Inscripta","highestDevelopmentStatusID":"1","companyTruncated":"Hunterian Medicine \/ Inscripta"},{"orgOrder":0,"company":"Agtc","sponsor":"TeamedOn","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"rAAV2tYF-CB-hRS1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ TeamedOn","highestDevelopmentStatusID":"1","companyTruncated":"Agtc \/ TeamedOn"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Curamys","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"MaxCyte","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MaxCyte \/ Curamys","highestDevelopmentStatusID":"1","companyTruncated":"MaxCyte \/ Curamys"},{"orgOrder":0,"company":"Entos Pharmaceuticals","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Entos Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Entos Pharmaceuticals \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Entos Pharmaceuticals \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Vigene Bioscience","sponsor":"ASC Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vigene Bioscience","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vigene Bioscience \/ ASC Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Vigene Bioscience \/ ASC Therapeutics"},{"orgOrder":0,"company":"Frontera Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"FT-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Frontera Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intraocular Injection","sponsorNew":"Frontera Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"Frontera Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"MitoChem Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"MC16","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"MitoChem Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MitoChem Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"MitoChem Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"rAAVrh8-HexA\/HexB","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathalamic Infusion","sponsorNew":"Sio Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"Sio Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"National Institutes of Health","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"National Institutes of Health","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Institutes of Health \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"National Institutes of Health \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-102","moa":"Gene","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"Orchard Therapeutics \/ Inapplicable"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Through the acquisition, Crosswalk will gain Codexis' investigational AAV based gene therapy, that is being developed to target fabry and pompe diseases.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          January 07, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Crosswalk Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Acquisition

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                          02

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : The Collaboration to combine Korro’s RNA editing technology with Genevant’s industry-leading LNP technology to develop a differentiated therapeutic option for patients with Alpha-1 Antitrypsin Deficiency (AATD).

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Undisclosed

                          July 03, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Genevant Sciences

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          03

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : FT-002 is an intraocular injection of recombinant AAV virus carrying the gene that expresses active functional proteins and repairs damaged retinal cell structure and function. It treat patients with X-linked retinitis pigmentosa caused by RPGR gene muta...

                          Product Name : FT-002

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 17, 2023

                          Lead Product(s) : FT-002

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : The collaboration is focused on an in vivo gene editing treatment for hemophilia A by utilizing 2seventy bio’s megaTAL technology that has the potential to provide a highly specific and efficient way to silence, edit, or insert genetic components.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : $5.0 million

                          January 05, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Novo Nordisk

                          Deal Size : $40.0 million

                          Deal Type : Collaboration

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                          05

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Using patient-derived retina organoids, the discovered that a type of LCA caused by mutations in the AAV-based NPHP5 Gene Thearapy (also called IQCB1) gene leads to severe defects in the primary cilium, a structure found in nearly all cells of the body.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          August 09, 2022

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Replay’s synHSV™ technology, a high payload capacity HSV vector capable of delivering up to 30 times the payload of AAV, is utilized by Replay’s four gene therapy product companies, bringing big DNA treatments to diseases affecting the skin, eye, b...

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          July 25, 2022

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : KKR

                          Deal Size : $55.0 million

                          Deal Type : Financing

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                          07

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details :

                          Product Name : MegaTAL mRNA

                          Product Type : Cell and Gene therapy

                          Upfront Cash : $5.0 million

                          June 01, 2022

                          Lead Product(s) : MegaTAL mRNA

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : 2Seventy Bio

                          Deal Size : $40.0 million

                          Deal Type : Licensing Agreement

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                          08

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : The collaboration utilizes 2seventy bio’s megaTAL technology that has the potential to provide a highly specific and efficient way to silence, edit, or insert genetic components. Hemophilia A is a genetic bleeding disorder resulting from defective Fact...

                          Product Name : MegaTAL mRNA

                          Product Type : Cell and Gene therapy

                          Upfront Cash : $5.0 million

                          June 01, 2022

                          Lead Product(s) : MegaTAL mRNA

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Novo Nordisk

                          Deal Size : $40.0 million

                          Deal Type : Collaboration

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                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Under the terms of the agreement, Curamys obtains non-exclusive clinical and commercial rights to use MaxCyte’s Flow Electroporation® technology and ExPERT™ platform.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          May 12, 2022

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Curamys

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

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                          10

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : CYTOO pioneered MyoScreen™, an in vitro R&D platform that enables the evaluation of therapies in patient-derived primary skeletal muscle cells under physiological conditions.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          May 04, 2022

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Astellas Gene Therapies

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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