[{"orgOrder":0,"company":"MC2 Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"DENMARK","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Calcipotriol","moa":"||Vitamin D receptor","graph1":"Dermatology","graph2":"Approved FDF","graph3":"MC2 Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Dermatology","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"MC2 Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"MC2 Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"MC2 Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"DENMARK","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Calcipotriol","moa":"||Vitamin D receptor","graph1":"Dermatology","graph2":"Approved FDF","graph3":"MC2 Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Dermatology","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"MC2 Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"MC2 Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"GeneTx Biotherapeutics","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 III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ GeneTx Biotherapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ GeneTx Biotherapeutics"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2023","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","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":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","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":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","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":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","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":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","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":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ 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 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 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 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 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":"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 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":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2022","type":"Acquisition","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":"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"},{"orgOrder":0,"company":"Acasti Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Steroid","year":"2022","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Acasti Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Spray","sponsorNew":"Acasti Pharma \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Acasti Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Acasti Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Steroid","year":"2022","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Acasti Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Spray","sponsorNew":"Acasti Pharma \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Acasti Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Surface Oncology","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Neurology","graph2":"Phase II","graph3":"Surface Oncology","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Ophthalmic Solution","sponsorNew":"Surface Oncology \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Surface Oncology \/ Inapplicable"},{"orgOrder":0,"company":"Cadila Pharmaceuticals","sponsor":"Lipidor AB","pharmaFlowCategory":"D","therapeuticArea":"Dermatology","country":"INDIA","productType":"Other Small Molecule","year":"2020","type":"Agreement","leadProduct":"Calcipotriol","moa":"||Vitamin D receptor","graph1":"Dermatology","graph2":"Phase II","graph3":"Cadila Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Dermatology","amount2New":0,"dosageForm":"Topical Spray","sponsorNew":"Cadila Pharmaceuticals \/ Lipidor AB","highestDevelopmentStatusID":"8","companyTruncated":"Cadila Pharmaceuticals \/ Lipidor AB"},{"orgOrder":0,"company":"Lipidor AB","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"SWEDEN","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Calcipotriol","moa":"||Vitamin D receptor","graph1":"Dermatology","graph2":"Phase III","graph3":"Lipidor AB","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Dermatology","amount2New":0,"dosageForm":"Topical Spray","sponsorNew":"Lipidor AB \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Lipidor AB \/ Inapplicable"},{"orgOrder":0,"company":"Lipidor AB","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"SWEDEN","productType":"Other Small 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Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Lipidor AB \/ Inapplicable"}]

Find Clinical Drug Pipeline Developments & Deals for Betamethasone Butyrate Propionate

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                          01

                          World Vaccine Conference
                          Not Confirmed
                          World Vaccine Conference
                          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 : Steroid

                          Upfront Cash : Inapplicable

                          December 19, 2024

                          Lead Product(s) : Betamethasone

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          World Vaccine Conference
                          Not Confirmed
                          World Vaccine Conference
                          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 : Steroid

                          Upfront Cash : Inapplicable

                          July 17, 2024

                          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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                          03

                          World Vaccine Conference
                          Not Confirmed
                          World Vaccine Conference
                          Not Confirmed

                          Details : GTX-102 is an investigational antisense oligonucleotide which is designed to target and inhibit expression of UBE3A-AS. It is being phase 1/2 clinical trials for the treatment of Angelman Syndrome.

                          Product Name : GTX-102

                          Product Type : Steroid

                          Upfront Cash : Inapplicable

                          May 02, 2024

                          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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                          04

                          World Vaccine Conference
                          Not Confirmed
                          World Vaccine Conference
                          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.

                          Product Name : GTX-102

                          Product Type : Steroid

                          Upfront Cash : Inapplicable

                          April 15, 2024

                          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

                          World Vaccine Conference
                          Not Confirmed
                          World Vaccine Conference
                          Not Confirmed

                          Details : GTX-102 is an investigational antisense oligonucleotide targeting UBE3A antisense transcript (UBE3A-AS), currently under Phase 1/2 clinical development for pediatric patients with Angelman Syndrome.

                          Product Name : GTX-102

                          Product Type : Steroid

                          Upfront Cash : Inapplicable

                          March 01, 2024

                          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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                          06

                          World Vaccine Conference
                          Not Confirmed
                          World Vaccine Conference
                          Not Confirmed

                          Details : GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and designed to target and inhibit expression of UBE3A-AS and is associated with improvements in some of the neurological symptoms.

                          Product Name : GTX-102

                          Product Type : Steroid

                          Upfront Cash : Inapplicable

                          May 17, 2023

                          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

                          Acasti Pharma

                          Country arrow
                          World Vaccine Conference
                          Not Confirmed

                          Acasti Pharma

                          Country arrow
                          World Vaccine Conference
                          Not Confirmed

                          Details : GTX-102 (betamethasone) is a novel, proprietary, concentrated oral-mucosal metered spray of betamethasone intended to improve the neurological symptoms of Ataxia Telangiectasia (A-T).

                          Product Name : GTX-102

                          Product Type : Steroid

                          Upfront Cash : Inapplicable

                          December 28, 2022

                          Lead Product(s) : Betamethasone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Acasti Pharma

                          Country arrow
                          World Vaccine Conference
                          Not Confirmed

                          Acasti Pharma

                          Country arrow
                          World Vaccine Conference
                          Not Confirmed

                          Details : GTX-102 is a novel, proprietary, concentrated oral-mucosal metered spray of betamethasone intended to improve the neurological symptoms of Ataxia Telangiectasia (AT) in a pediatric population for which there are currently no FDA-approved therapies.

                          Product Name : GTX-102

                          Product Type : Steroid

                          Upfront Cash : Inapplicable

                          September 13, 2022

                          Lead Product(s) : Betamethasone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          World Vaccine Conference
                          Not Confirmed
                          World Vaccine Conference
                          Not Confirmed

                          Details : GTX-102 an antisense oligonucleotide delivered via intrathecal administration and designed to target and inhibit expression of UBE3A-AS. Nonclinical studies show that GTX-102 reduces levels of UBE3A-AS and reactivates expression of paternal UBE3A allele ...

                          Product Name : GTX-102

                          Product Type : Steroid

                          Upfront Cash : Inapplicable

                          July 18, 2022

                          Lead Product(s) : Betamethasone

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : GeneTx Biotherapeutics

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          World Vaccine Conference
                          Not Confirmed
                          World Vaccine Conference
                          Not Confirmed

                          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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