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Morgan"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"DTX401","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ultragenyx Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"Rebisufligene Etisparvovec","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ultragenyx Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Setrusumab","moa":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"9","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"GTX-102","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Ultragenyx Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"Ultragenyx Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"UX701","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Ultragenyx Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"Ultragenyx Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Setrusumab","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Ultragenyx Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"Ultragenyx Pharmaceutical \/ Not Applicable"}]

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

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

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

                          Brand Name : UX143

                          Molecule 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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                          02

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          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.

                          Brand Name : UX701

                          Molecule 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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                          03

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          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.

                          Brand Name : GTX-102

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          July 17, 2024

                          Lead Product(s) : GTX-102

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          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.

                          Brand Name : UX143

                          Molecule 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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                          05

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          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).

                          Brand Name : UX111

                          Molecule 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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                          06

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

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

                          Brand Name : UX143

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          June 11, 2024

                          Lead Product(s) : Setrusumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Mereo BioPharma Group

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          blank

                          07

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : DTX401 is an investigational AAV8 gene therapy designed to deliver G6Pase-α in patients with Glycogen Storage Disease Type Ia.

                          Brand Name : DTX401

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          May 30, 2024

                          Lead Product(s) : DTX401,Prednisolone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : UX143 (setrusumab) is a fully human monoclonal antibody that inhibits sclerostin, a critical protein in bone-signaling pathways affecting bone-forming cell maturation in osteogenesis imperfecta.

                          Brand Name : UX143

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          April 30, 2024

                          Lead Product(s) : Setrusumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated in the treating of Patients with Angelman Syndrome.

                          Brand Name : GTX-102

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          April 15, 2024

                          Lead Product(s) : GTX-102

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          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).

                          Brand Name : UX111

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          February 06, 2024

                          Lead Product(s) : UX111

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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