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Abingworth"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Ysios Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"SpliceBio","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"SpliceBio \/ Ysios Capital","highestDevelopmentStatusID":"1","companyTruncated":"SpliceBio \/ Ysios Capital"},{"orgOrder":0,"company":"Brigham and Women's Hospital","sponsor":"Baylor College of Medicine | Boston Children's Hospital | Broad Institute of MIT and Harvard | Dartmouth-Hitchcock Medical Center | Harvard Pilgrim Health Care | Howard University | HudsonAlpha Institute for Biotechnology | Massachusetts General Hospital ","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Brigham and Women's Hospital","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Brigham and Women's Hospital \/ Baylor College of Medicine | Boston Children's Hospital | Broad Institute of MIT and Harvard | Dartmouth-Hitchcock Medical Center | Harvard Pilgrim Health Care | Howard University | HudsonAlpha Institute for Biotechnology | Massachusetts General Hospital ","highestDevelopmentStatusID":"1","companyTruncated":"Brigham and Women's Hospital \/ Baylor College of Medicine | Boston Children's Hospital | Broad Institute of MIT and Harvard | Dartmouth-Hitchcock Medical Center | Harvard Pilgrim Health Care | Howard University | HudsonAlpha Institute for Biotechnology | Massachusetts General Hospital "},{"orgOrder":0,"company":"University of Massachusetts Medical School","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Massachusetts Medical School","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Massachusetts Medical School \/ CANbridge Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"University of Massachusetts Medical School \/ CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Genelex Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genelex Corporation","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genelex Corporation \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Genelex Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arcturus Therapeutics \/ Guggenheim Securities","highestDevelopmentStatusID":"1","companyTruncated":"Arcturus Therapeutics \/ Guggenheim Securities"},{"orgOrder":0,"company":"National Institutes of Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"National Institutes of Health","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Institutes of Health \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"National Institutes of Health \/ Undisclosed"},{"orgOrder":0,"company":"Codexis","sponsor":"Crosswalk Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Codexis","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Codexis \/ Crosswalk Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Codexis \/ Crosswalk Therapeutics"},{"orgOrder":0,"company":"AavantiBio","sponsor":"University of Florida","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AavantiBio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ University of Florida","highestDevelopmentStatusID":"1","companyTruncated":"AavantiBio \/ University of Florida"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Personalis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Personalis","highestDevelopmentStatusID":"1","companyTruncated":"Sarepta Therapeutics \/ Personalis"},{"orgOrder":0,"company":"Duchenne research","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Duchenne research","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Duchenne research \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Duchenne research \/ Undisclosed"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Curamys","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"MaxCyte","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MaxCyte \/ Curamys","highestDevelopmentStatusID":"1","companyTruncated":"MaxCyte \/ Curamys"},{"orgOrder":0,"company":"PepGen","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"PepGen","amount2":0.050000000000000003,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"PepGen \/ RA Capital Management","highestDevelopmentStatusID":"1","companyTruncated":"PepGen \/ RA Capital Management"},{"orgOrder":0,"company":"Dewpoint Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dewpoint Therapeutics","amount2":0.23999999999999999,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Dewpoint Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"1","companyTruncated":"Dewpoint Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"AavantiBio","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AavantiBio","amount2":0.11,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ Sarepta Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"AavantiBio \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Entos Pharmaceuticals","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Entos Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Entos Pharmaceuticals \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Entos Pharmaceuticals \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Mammoth Biosciences","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Mammoth Biosciences","amount2":0.68999999999999995,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.68999999999999995,"dosageForm":"Undisclosed","sponsorNew":"Mammoth Biosciences \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"Mammoth Biosciences \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"hC Bioscience","sponsor":"Arch Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"hC Bioscience","amount2":0.02,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Undisclosed","sponsorNew":"hC Bioscience \/ Arch Ventures","highestDevelopmentStatusID":"1","companyTruncated":"hC Bioscience \/ Arch Ventures"},{"orgOrder":0,"company":"Vigene Bioscience","sponsor":"ASC Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vigene Bioscience","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vigene Bioscience \/ ASC Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Vigene Bioscience \/ ASC Therapeutics"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Affinia Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Affinia Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"Affinia Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Cureduchenne Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dyne Therapeutics \/ Cureduchenne Ventures","highestDevelopmentStatusID":"1","companyTruncated":"Dyne Therapeutics \/ Cureduchenne Ventures"},{"orgOrder":0,"company":"Replay Bio","sponsor":"KKR","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Replay Bio","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Replay Bio \/ KKR","highestDevelopmentStatusID":"1","companyTruncated":"Replay Bio \/ KKR"},{"orgOrder":0,"company":"Dr Jorg Taubel","sponsor":"Richmond Pharmacology | National Amyloidosis Centre","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dr Jorg Taubel","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dr Jorg Taubel \/ Richmond Pharmacology | National Amyloidosis Centre","highestDevelopmentStatusID":"1","companyTruncated":"Dr Jorg Taubel \/ Richmond Pharmacology | National Amyloidosis Centre"},{"orgOrder":0,"company":"Adventia Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Dietary Supplement","year":"2025","type":"Inapplicable","leadProduct":"Eicosapentaenoic Acid|Docosahexaenoic Acid","moa":"Lipid|PPAR-gamma","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Adventia Pharma","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oil","sponsorNew":"Adventia Pharma \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Adventia Pharma \/ Undisclosed"}]

    Find Novel Drugs for Genetic Disease under Development

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                            01

                            AbbVie Inc

                            U.S.A
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Fosigotifator

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Expanded Access to Fosigotifator

                            Details : Fosigotifator is a Other Small Molecule drug candidate, which is currently being evaluated in clinical studies for the treatment of Leukoencephalopathies.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 19, 2024

                            Lead Product(s) : Fosigotifator

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Adventia Pharma

                            Country arrow
                            OTS 2025
                            Not Confirmed

                            Adventia Pharma

                            Country arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Eicosapentaenoic Acid|Docosahexaenoic Acid

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Effect of a Combined Exercise and Anti-Inflammatory Nutritional Intervention in Frail Older Adults

                            Details : Eicosapentaenoic Acid|Docosahexaenoic Acid is a Dietary Supplement drug candidate, which is currently being evaluated in Undisclosed clinical studies for the treatment of Frail Elderly Syndrome.

                            Product Name : Undisclosed

                            Product Type : Dietary Supplement

                            Upfront Cash : Inapplicable

                            September 19, 2025

                            Lead Product(s) : Eicosapentaenoic Acid|Docosahexaenoic Acid

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Alvotech

                            Iceland arrow
                            OTS 2025
                            Not Confirmed

                            Alvotech

                            Iceland arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Canakinumab

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : Advanz Pharma

                            Deal Size : Undisclosed

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Alvotech and Advanz Expand Partnership To Commercialize Three Biosimilars in Europe

                            Details : Advanz secures exclusive rights from Alvotech to commercialize three biosimilar candidates, Ilaris (canakinumab), Kesimpta (ofatumumab) and an undiclosed biosimilar in Europe.

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Undisclosed

                            May 28, 2025

                            Lead Product(s) : Canakinumab

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : Advanz Pharma

                            Deal Size : Undisclosed

                            Deal Type : Agreement

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                            04

                            Takeda Pharmaceutical

                            Japan arrow
                            OTS 2025
                            Not Confirmed

                            Takeda Pharmaceutical

                            Japan arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : TAK-881

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Expanded Access to TAK-881 for Children and Teenagers With Primary Immunodeficiency Diseases in the USA

                            Details : TAK-881 is a drug candidate, which is currently being evaluated in clinical studies for the treatment of Immunologic Deficiency Syndromes.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            May 02, 2025

                            Lead Product(s) : TAK-881

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Nutricia Inc

                            Country arrow
                            OTS 2025
                            Not Confirmed

                            Nutricia Inc

                            Country arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : GMP Intervention Product

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            GMP Powdered Substitutes in PKU and TYR

                            Details : GMP Intervention Product is a Dietary Supplement drug candidate, which is currently being evaluated in clinical studies for the treatment of Phenylketonurias.

                            Product Name : Undisclosed

                            Product Type : Dietary Supplement

                            Upfront Cash : Inapplicable

                            April 23, 2025

                            Lead Product(s) : GMP Intervention Product

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Applied Science & Performance Institute

                            Country arrow
                            OTS 2025
                            Not Confirmed

                            Applied Science & Performance Institute

                            Country arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : CAS 501-94-0

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            The Effects of Tyrosol and Creatine on Endurance, Strength, and Fatigue Resistance in Healthy Adults

                            Details : Tyrosol is a Plant Extract/Herbal drug candidate, which is currently being evaluated in clinical studies for the treatment of Creatine Deficiency Syndromes.

                            Product Name : Undisclosed

                            Product Type : Plant Extract/Herbal

                            Upfront Cash : Inapplicable

                            March 19, 2025

                            Lead Product(s) : CAS 501-94-0

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Scholar Rock

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Scholar Rock

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Apitegromab

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            EAP of Apitegromab for Patients With Spinal Muscular Atrophy

                            Details : Apitegromab is a Antibody drug candidate, which is currently being evaluated in clinical studies for the treatment of Muscular Atrophy, Spinal.

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Inapplicable

                            March 14, 2025

                            Lead Product(s) : Apitegromab

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Arrowhead Pharmaceuticals

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Arrowhead Pharmaceuticals

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Plozasiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Treatment Protocol of Plozasiran in Adults With FCS

                            Details : Plozasiran is a Oligonucleotide drug candidate, which is currently being evaluated in clinical studies for the treatment of Hyperlipoproteinemia Type I.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            January 28, 2025

                            Lead Product(s) : Plozasiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Genecombio Ltd.

                            Country arrow
                            OTS 2025
                            Not Confirmed

                            Genecombio Ltd.

                            Country arrow
                            OTS 2025
                            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) : GCB-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : The Children's Hospital of Zhejiang University School of Medicine

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy

                            Details : GCB-001 is a drug candidate, which is currently being evaluated in clinical studies for the treatment of Spinal Muscular Atrophies of Childhood.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            January 13, 2025

                            Lead Product(s) : GCB-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : The Children's Hospital of Zhejiang University School of Medicine

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Centre Hospitalier de Saint-Denis

                            Country arrow
                            OTS 2025
                            Not Confirmed

                            Centre Hospitalier de Saint-Denis

                            Country arrow
                            OTS 2025
                            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) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : INSERM U955

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Pilot Study "AHSP as a Biomarker of Sickle Cell Disease in a Population of Adults and Children"

                            Details : This drug candidate is currently being evaluated in clinical studies for the treatment of Anemia, Sickle Cell.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            December 16, 2024

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : INSERM U955

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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