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Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"iECURE","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"ECUR-506","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"iECURE \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"iECURE \/ Not Applicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"OCU400","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Ocugen \/ Not Applicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"GTX-102","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"OCU400","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Ocugen \/ Not Applicable"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"INZ-701","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Cornell University","sponsor":"Lexeo Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Licensing Agreement","leadProduct":"LX2006","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Cornell University","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cornell University \/ Lexeo Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Cornell University \/ Lexeo Therapeutics"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AAV2\/5-hPDE6B","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Coave Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Exegenesis Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"EXG001-307","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Exegenesis Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Exegenesis Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Exegenesis Bio \/ Not Applicable"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"ARO-DM1","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arrowhead Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"WVE-006","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"ATSN-201","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AAVB-081","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"AAVantgarde Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Not Applicable"},{"orgOrder":0,"company":"iECURE","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"ECUR-506","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"iECURE \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"iECURE \/ Not Applicable"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.33000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.37,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"MYR-101","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Myrtelle \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Not Applicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AAV5-hRORA","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ocugen \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Ocugen \/ Not Applicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AAV5-hRORA","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ocugen \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Ocugen \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AMT-130","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AMT-130","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Grace Science","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"GS-100","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Grace Science \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"Marnetegragene Autotemcel","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"NGN-401","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"NTLA-2002","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Intellia Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Intellia Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"RGX-202","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Public Offering","leadProduct":"TSHA-102","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Public Offering","leadProduct":"TSHA-102","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"DNL126","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Denali Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Denali Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"DYNE-101","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Dyne Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"ION582","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"mRNA-3705","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Moderna Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Seroba","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Series B Financing","leadProduct":"VO659","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"","sponsorNew":"Vico Therapeutics \/ Seroba","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Seroba"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"WVE-003","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Wave Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Not Applicable"}]

    Find Drugs for Genetic Disease in Phase I/ Phase II Clinical Development

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                            01

                            Sanofi

                            France
                            Fi Europe 2024
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Translate Bio Provides MRT5005 Program Updates

                            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.

                            Brand Name : Undisclosed

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            March 26, 2020

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            02

                            Sanofi

                            France
                            Fi Europe 2024
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Translate Bio Receives FDA Fast Track Designation for MRT5005 for the Treatment of Cystic Fibrosis

                            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.

                            Brand Name : Undisclosed

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            February 26, 2020

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            03

                            Ocugen

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                            Fi Europe 2024
                            Not Confirmed

                            Ocugen

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                            Fi Europe 2024
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : AAV5-hRORA

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            DSMB Approves Phase 2 Start of OCU410ST Trial for Stargardt Disease

                            Details : OCU410ST (AAV5-hRORA) is a modifier gene therapy candidate, which utilizes an AAV delivery platform for the retinal delivery of the RORA gene. It is being evaluated for Stargardt Disease.

                            Brand Name : OCU410ST

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            October 22, 2024

                            Lead Product(s) : AAV5-hRORA

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            04

                            ARTHEx Biotech

                            Spain arrow
                            Fi Europe 2024
                            Not Confirmed

                            ARTHEx Biotech

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                            Fi Europe 2024
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : ATX-01

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            ARTHEx Doses First Patient in ArthemiR™ Trial for Myotonic Dystrophy

                            Details : ATX-01, an antimiR designed to target microRNA 23b (miR-23b), is the first microRNA therapeutic which is being evaluated for the treatment of Myotonic Dystrophy Type 1.

                            Brand Name : ATX-01

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            October 18, 2024

                            Lead Product(s) : ATX-01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            05

                            Wave Life Sciences

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Wave Life Sciences

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed
                            • fda
                            • EDQM
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                            Lead Product(s) : WVE-006

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Wave Life Sciences Achieves First RNA Editing in Humans in Alpha-1 Antitrypsin Deficiency

                            Details : WVE-006, a first-in-class GalNAc-conjugated RNA editing oligonucleotide. It is currently undergoing evaluation in early-stage clinical trials for treating alpha-1 antitrypsin deficiency.

                            Brand Name : WVE-006

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            October 16, 2024

                            Lead Product(s) : WVE-006

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            06

                            Myrtelle

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Myrtelle

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                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : MYR-101,Levetiracetam,Prednisone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Myrtelle Reports NAA Reduction in Phase 1/2 Gene Therapy Trial For Canavan Disease

                            Details : MYR-101 (rAAV-Olig001-ASPA) is a gene therapy candidate, which is intended to restore ASPA function and brain development in patients with Canavan Disease.

                            Brand Name : MYR-101

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            October 10, 2024

                            Lead Product(s) : MYR-101,Levetiracetam,Prednisone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            Astria Therapeutics

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Astria Therapeutics

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : Navenibart

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Astria Receives FDA Orphan Drug Designation for Navenibart in Hereditary Angioedema

                            Details : STAR-0215 is a monoclonal antibody dug candidate that acts as inhibitor of plasma kallikrein, which is being evaluated for the treatment of hereditary angioedema.

                            Brand Name : STAR-0215

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            September 30, 2024

                            Lead Product(s) : Navenibart

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            08

                            ReCode Therapeutics

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            ReCode Therapeutics

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                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : RCT2100

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            ReCode Doses First Patient in Phase 1b Study Of RCT2100 For Cystic Fibrosis

                            Details : RCT2100 is an investigational inhaled mRNA therapy, which is currently being evaluated for the treatment of patients with cystic fibrosis.

                            Brand Name : RCT2100

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            September 26, 2024

                            Lead Product(s) : RCT2100

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            09

                            Elixirgen Therapeutics

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Elixirgen Therapeutics

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                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : EXG-34217

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Elixirgen Receives Rare Pediatric Designation for EXG-34217 in Dyskeratosis Congenita

                            Details : EXG-001, a non-integrating, non-transmissible, temperature-sensitive Sendai virus vector encoding human ZSCAN4, being evaluated for dyskeratosis congenita and related telomere biology disorders.

                            Brand Name : EXG-34217

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            September 25, 2024

                            Lead Product(s) : EXG-34217

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            10

                            NS Pharma

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                            Fi Europe 2024
                            Not Confirmed

                            NS Pharma

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                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : NS-050/NCNP-03

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            FDA Grants Rare Pediatric Disease Designation For NS-050/NCNP-03 in DMD

                            Details : NS-050/NCNP-03 is an antisense oligonucleotide and is being developed for the treatment of Duchenne muscular dystrophy (Duchenne).

                            Brand Name : NS-050/NCNP-03

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            September 10, 2024

                            Lead Product(s) : NS-050/NCNP-03

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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