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\/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"GTX-102","moa":"UBE3A-AS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"GTX-102","moa":"UBE3A-AS","graph1":"Genetic Disease","graph2":"Phase 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Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"GTX-102","moa":"UBE3A-AS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"UX053","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"GeneTx Biotherapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Acquisition","leadProduct":"GTX-102","moa":"UBE3A-AS","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"GeneTx Biotherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"GTX-102","moa":"UBE3A-AS","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"GeneTx Biotherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"GTX-102","moa":"UBE3A-AS","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"GeneTx Biotherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"GTX-102","moa":"UBE3A-AS","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Ultragenyx Pharmaceutical"}]

Find Clinical Drug Pipeline Developments & Deals by Ultragenyx Pharmaceutical

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          BioAsia
                          Not Confirmed
                          BioAsia
                          Not Confirmed

                          Details : UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated for the treatment of sanfilippo syndrome type A (MPS IIIA) in children.

                          Product Name : UX111

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 18, 2025

                          Lead Product(s) : Rebisufligene Etisparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          02

                          BioAsia
                          Not Confirmed
                          BioAsia
                          Not Confirmed

                          Details : UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated for the treatment of sanfilippo syndrome type A (MPS IIIA) in children.

                          Product Name : UX111

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 05, 2025

                          Lead Product(s) : Rebisufligene Etisparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          BioAsia
                          Not Confirmed
                          BioAsia
                          Not Confirmed

                          Details : Evkeeza (evinacumab) is the first ANGPTL3 inhibitor. It is approved for the treatment of children 6 month and older with Homozygous Familial Hypercholesterolemia (HoFH).

                          Product Name : Evkeeza

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          January 06, 2025

                          Lead Product(s) : Evinacumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          BioAsia
                          Not Confirmed
                          BioAsia
                          Not Confirmed

                          Details : UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated in phase 3 clinical trials for the treatment of sanfilippo syndrome type A (MPS IIIA).

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 19, 2024

                          Lead Product(s) : Rebisufligene Etisparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          BioAsia
                          Not Confirmed
                          BioAsia
                          Not Confirmed

                          Details : GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated for the treatment of angelman syndrome.

                          Product Name : GTX-102

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          December 19, 2024

                          Lead Product(s) : GTX-102

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          BioAsia
                          Not Confirmed
                          BioAsia
                          Not Confirmed

                          Details : UX143 (setrusumab), is a sclerostin inhibitor antibody drug candidate, which is currently being evaluated for the treatment of osteogenesis imperfecta.

                          Product Name : UX143

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          October 07, 2024

                          Lead Product(s) : Setrusumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          BioAsia
                          Not Confirmed
                          BioAsia
                          Not Confirmed

                          Details : UX701 is an investigational AAV9 gene therapy designed to deliver stable expression of the ATP7B copper transporter. It is being evaluated in clinical trials for the treatment of Wilson Disease.

                          Product Name : UX701

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          October 03, 2024

                          Lead Product(s) : UX701

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          BioAsia
                          Not Confirmed
                          BioAsia
                          Not Confirmed

                          Details : GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated for the treatment of angelman syndrome.

                          Product Name : GTX-102

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          July 17, 2024

                          Lead Product(s) : GTX-102

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          BioAsia
                          Not Confirmed
                          BioAsia
                          Not Confirmed

                          Details : The net proceeds of the offering to continue to advance the late-stage clinical program UX143 (setrusumab), which is being evaluated for the treatment of osteogenesis imperfecta.

                          Product Name : UX143

                          Product Type : Large molecule

                          Upfront Cash : Undisclosed

                          June 13, 2024

                          Lead Product(s) : Setrusumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : J.P. Morgan

                          Deal Size : $350.0 million

                          Deal Type : Public Offering

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                          10

                          BioAsia
                          Not Confirmed
                          BioAsia
                          Not Confirmed

                          Details : UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated in phase 3 clinical trials for the treatment of sanfilippo syndrome type A (MPS IIIA).

                          Product Name : UX111

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          June 12, 2024

                          Lead Product(s) : Rebisufligene Etisparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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