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    [{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and 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Disorders","country":"U.S.A","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph 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Pharmaceutical"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2022","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical"}]

    Find Clinical Drug Pipeline Developments & Deals by GeneTx Biotherapeutics

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                            01

                            GeneTx Biotherapeutics

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                            GeneTx Biotherapeutics

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                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

                            DEALS_DEV arrow-down

                            Ultragenyx Exercises Option to Acquire GeneTx

                            Details : GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx. GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration.

                            Product Name : GTX-102

                            Product Type : Steroid

                            Upfront Cash : $75.0 million

                            July 18, 2022

                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

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                            02

                            GeneTx Biotherapeutics

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                            GeneTx Biotherapeutics

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                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            GeneTx and Ultragenyx Provide Preliminary Update on Phase 1/2 Clinical Study of GTX-102 in Canada and U.K. Pat...

                            Details : Phase 1/2 Clinical Study of GTX-102 that inhibit expression of UBE3A-AS, showed no treatment-related serious adverse events to lower extremity weakness observed in the patients, and initial assessments have shown early signs of clinical activity.

                            Product Name : GTX-102

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            May 01, 2022

                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            GeneTx Biotherapeutics

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                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            GeneTx and Ultragenyx Announce FDA has Removed Clinical Hold on Phase 1/2 Clinical Study of GTX-102 for the Tr...

                            Details : GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and designed to target and inhibit expression of UBE3A-AS used in pediatric patients with Angelman syndrome.

                            Product Name : GTX-102

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            September 27, 2021

                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            GeneTx Biotherapeutics

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                            GeneTx Biotherapeutics

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                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            GeneTx and Ultragenyx Receive Approval from U.K. Regulatory Agency to Begin Clinical Study of GTX-102 for the ...

                            Details : The phase 1/2 study evaluates the safety, tolerability, and plasma and cerebrospinal fluid concentrations of GTX-102 in pediatric patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.

                            Product Name : GTX-102

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            June 22, 2021

                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            GeneTx Biotherapeutics

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                            GeneTx Biotherapeutics

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                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            GeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improv...

                            Details : At the highest doses, all five patients experienced a serious adverse event (SAE) of lower extremity weakness believed to be related to local inflammation due to GTX-102. Following these events, the companies paused enrollment and dosing.

                            Product Name : GTX-102

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            October 26, 2020

                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            GeneTx Biotherapeutics

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                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            GeneTx and Ultragenyx Announce GTX-102 Granted Fast Track Designation by U.S. FDA for Treatment of Angelman Sy...

                            Details : GTX-102 is an investigational antisense oligonucleotide currently being evaluated in a Phase 1/2 study for the treatment of Angelman syndrome.

                            Product Name : GTX-102

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            April 05, 2020

                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            GeneTx Biotherapeutics

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                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with Ang...

                            Details : First investigational study testing an antisense oligonucleotide as a potential therapy to treat AS.

                            Product Name : GTX-102

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            March 16, 2020

                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            GeneTx Biotherapeutics

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                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            GeneTx Announces IRB Approval to Begin Clinical Study of GTX-102 for the Treatment of Angelman Syndrome

                            Details : The Phase 1/2 open-label, multiple-dose, dose-escalating study will evaluate the safety, tolerability, and plasma and cerebrospinal fluid (CSF) concentrations of GTX-102.

                            Product Name : GTX-102

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            February 14, 2020

                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            GeneTx Biotherapeutics

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                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with ...

                            Details : GeneTx’s Phase 1/2 study, named KIK-AS, is expected to enroll 20 patients at multiple trial sites, beginning in the first half of 2020.

                            Product Name : GTX-102

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            January 15, 2020

                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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