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Inapplicable"},{"orgOrder":0,"company":"Bristol Myers Squibb","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Luspatercept","moa":"Transforming growth factor beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bristol Myers Squibb","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bristol Myers Squibb \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Bristol Myers Squibb \/ Inapplicable"},{"orgOrder":0,"company":"ReNeuron","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase 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Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"HM15136","moa":"GLP-1R\/GIPR\/GCGR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Hanmi Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Hanmi Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Hanmi Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Metro International Biotech","sponsor":"Children's Hospital of Philadelphia","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"MIB-626","moa":"NAD+","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Metro International Biotech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Metro International Biotech \/ Children's Hospital of Philadelphia","highestDevelopmentStatusID":"8","companyTruncated":"Metro International Biotech \/ Children's Hospital of Philadelphia"},{"orgOrder":0,"company":"Akershus University Hospital","sponsor":"The Bergesen Foundation | South-Eastern Norway Regional Health Authority | Sykehuset Innlandet HF | Oslo University Hospital | St. Olavs Hospital | Haukeland University Hospital | University Hospital of North Norway | University of Bergen","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Nicotinamide Riboside","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Akershus University Hospital","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Akershus University Hospital \/ The Bergesen Foundation | South-Eastern Norway Regional Health Authority | Sykehuset Innlandet HF | Oslo University Hospital | St. Olavs Hospital | Haukeland University Hospital | University Hospital of North Norway | University of Bergen","highestDevelopmentStatusID":"8","companyTruncated":"Akershus University Hospital \/ The Bergesen Foundation | South-Eastern Norway Regional Health Authority | Sykehuset Innlandet HF | Oslo University Hospital | St. Olavs Hospital | Haukeland University Hospital | University Hospital of North Norway | University of Bergen"},{"orgOrder":0,"company":"Matrix Biomed","sponsor":"University of Texas Health Science Center at Houston","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"MBM-01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase 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Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"David Williams \/ National Heart, Lung, and Blood Institute | California Institute for Regenerative Medicine (CIRM) | Bluebird Bio | Blood and Marrow Transplant Clinical Trials Network","highestDevelopmentStatusID":"8","companyTruncated":"David Williams \/ National Heart, Lung, and Blood Institute | California Institute for Regenerative Medicine (CIRM) | Bluebird Bio | Blood and Marrow Transplant Clinical Trials Network"},{"orgOrder":0,"company":"Columbia University","sponsor":"Attralus","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabeled Compounds","year":"2022","type":"Inapplicable","leadProduct":"124-I Evuzamitide","moa":"MUC1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Columbia University","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Columbia University \/ Attralus","highestDevelopmentStatusID":"8","companyTruncated":"Columbia University \/ Attralus"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SAR447537","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SAR447537","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2024","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":"Thiogenesis Therapeutics, Inc.","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"TTI-0102","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Thiogenesis Therapeutics, Inc.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Thiogenesis Therapeutics, Inc. \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Thiogenesis Therapeutics, Inc. \/ Inapplicable"},{"orgOrder":0,"company":"ChromaDex, Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2021","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":"Aridis Pharmaceuticals","sponsor":"Cystic Fibrosis 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":"Nasal 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":"Nasal 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 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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":"Nasal 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 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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":"Nasal 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":"Nasal 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":"Nasal 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":"Nasal 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":"Nasal 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 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Disease","amount2New":0,"dosageForm":"Nasal 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":"Nasal 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"}]

    Find Drugs for Genetic Disease in Phase II Clinical Development

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                            01

                            Sanofi

                            France
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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

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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

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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

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                            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
                            ChemOutsourcing
                            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) : 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
                            ChemOutsourcing
                            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) : 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
                            ChemOutsourcing
                            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

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                            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
                            ChemOutsourcing
                            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

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                            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
                            ChemOutsourcing
                            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
                            ChemOutsourcing
                            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

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                            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
                            ChemOutsourcing
                            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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