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Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Funding","leadProduct":"Gallium Citrate","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aridis Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Aerosol","sponsorNew":"Aridis Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"8","companyTruncated":"Aridis Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Aridis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Gallium Citrate","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aridis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"Aridis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Aridis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Aridis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Gallium Citrate","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aridis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"Aridis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Aridis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Aridis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Gallium Citrate","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aridis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"Aridis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Aridis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Aridis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Gallium Citrate","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aridis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"Aridis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Aridis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"STSP-0601","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Inapplicable"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Novotech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Novotech","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Novotech"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Funding","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Bayer AG","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Series D Financing","leadProduct":"REC-994","moa":"Superoxide Radical","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Recursion Pharma","amount2":0.23999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23999999999999999,"dosageForm":"Tablet","sponsorNew":"Recursion Pharma \/ Bayer AG","highestDevelopmentStatusID":"8","companyTruncated":"Recursion Pharma \/ Bayer AG"},{"orgOrder":0,"company":"SpliSense","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"SPL84","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SpliSense","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"SpliSense \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"SpliSense \/ Inapplicable"},{"orgOrder":0,"company":"Healx","sponsor":"R42 Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2024","type":"Series C Financing","leadProduct":"HLX-1502","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Healx","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Healx \/ R42 Group","highestDevelopmentStatusID":"8","companyTruncated":"Healx \/ R42 Group"},{"orgOrder":0,"company":"Tyra Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"TYRA-300","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tyra Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tyra Biosciences \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Tyra Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Veristat | Metrum Research Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"CTI-1601","moa":"Soluble guanylate cyclase (GCS)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Larimar Therapeutics \/ Veristat | Metrum Research Group","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Veristat | Metrum Research Group"},{"orgOrder":0,"company":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"STSP-0601","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Inapplicable"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ChromaDex, Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2024","type":"Inapplicable","leadProduct":"Nicotinamide Riboside Chloride","moa":"NAD","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ChromaDex, Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ChromaDex, Inc \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"ChromaDex, Inc \/ Inapplicable"},{"orgOrder":0,"company":"Connoya Biomedical Technology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"CM336","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Connoya Biomedical Technology","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Connoya Biomedical Technology \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Connoya Biomedical Technology \/ Undisclosed"},{"orgOrder":0,"company":"IASO Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Equecabtagene Autoleucel","moa":"BCMA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"IASO Bio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IASO Bio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"IASO Bio \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Suzanne Lentzsch, MD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Teclistamab","moa":"Tumor necrosis factor receptor superfamily member 17 | T cell surface glycoprotein CD3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Suzanne Lentzsch, MD","highestDevelopmentStatusID":"8","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Suzanne Lentzsch, MD"},{"orgOrder":0,"company":"Pharming","sponsor":"Aixial Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Pharming \/ Aixial Group","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Aixial Group"},{"orgOrder":0,"company":"Inova Health Care Services","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Deferoxamine Mesylate","moa":"Iron","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inova Health Care Services","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inova Health Care Services \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inova Health Care Services \/ Undisclosed"},{"orgOrder":0,"company":"Orphalan","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Trientine Hydrochloride","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphalan","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orphalan \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Orphalan \/ Undisclosed"},{"orgOrder":0,"company":"TJ Biopharma Co., Ltd.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Plonmarlimab","moa":"Granulocyte-macrophage colony-stimulating factor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"TJ Biopharma Co., Ltd.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"TJ Biopharma Co., Ltd. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"TJ Biopharma Co., Ltd. \/ Undisclosed"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"SION-719","moa":"CFTR NBD1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sionna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sionna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sionna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Alpelisib","moa":"PI3-kinase p110-alpha subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Angitia Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"AGA2115","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Angitia Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Angitia Biopharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Angitia Biopharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Jerry Vockley","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Jerry Vockley","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jerry Vockley \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Jerry Vockley \/ Ultragenyx Pharmaceutical"}]

    Find Drugs for Genetic Disease in Phase II Clinical Development

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                            01

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alfa

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sp...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            April 29, 2025

                            Lead Product(s) : Olipudase Alfa

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            AbbVie Inc

                            U.S.A
                            BioProcess International
                            Not Confirmed
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                            • EDQM
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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) : ABBV-CLS-628

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Calico Life Sciences

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess Adverse Events and Effectiveness of IntraVenous Infusions of ABBV-CLS-628 in Adult Participa...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            March 30, 2025

                            Lead Product(s) : ABBV-CLS-628

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Calico Life Sciences

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            AbbVie Inc

                            U.S.A
                            BioProcess International
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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) : Galicaftor,Navocaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Sionna Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Sionna Expands CF Pipeline with AbbVie License Agreement

                            Details : Under the terms of the agreement, Sionna will assume all development for ABBV-2222 (galicaftor), which is being evaluated in combination with navocaftor for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            July 16, 2024

                            Lead Product(s) : Galicaftor,Navocaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Sionna Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            04

                            Sanofi

                            France
                            CPhI WW Frankfurt
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                            • fda
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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : SAR447537

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            June 09, 2023

                            Lead Product(s) : SAR447537

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : SAR447537

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            May 12, 2023

                            Lead Product(s) : SAR447537

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            AbbVie Inc

                            U.S.A
                            BioProcess International
                            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) : Galicaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate Adverse Events and Change in Disease Activity With Oral Capsules of Galicaftor/Navocaftor/AB...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 21, 2021

                            Lead Product(s) : Galicaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            AbbVie Inc

                            U.S.A
                            BioProcess International
                            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) : Navocaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Phase 2 Study of ABBV-3067 Alone and in Combination With ABBV-2222

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 31, 2019

                            Lead Product(s) : Navocaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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) : Tesevatinib

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of the Efficacy and Safety of Tesevatinib in Subjects With ADPKD

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            June 29, 2017

                            Lead Product(s) : Tesevatinib

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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) : Avalglucosidase Alfa

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patie...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            January 12, 2017

                            Lead Product(s) : Avalglucosidase Alfa

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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) : Lademirsen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of Lademirsen (SAR339375) in Patients With Alport Syndrome

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            August 04, 2016

                            Lead Product(s) : Lademirsen

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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