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    [{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"Ypsomed","sponsor":"Astria Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody","year":"2024","type":"Partnership","leadProduct":"Navenibart","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ypsomed","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ypsomed \/ Astria Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Ypsomed \/ Astria Therapeutics"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Branaplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Branaplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Branaplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"Lonza Group","sponsor":"Capricor Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Lonza Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Lonza Group \/ Capricor Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Lonza Group \/ Capricor Therapeutics"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"Santhera Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Steroid","year":"2020","type":"Licensing Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Idorsia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"Idorsia Pharmaceuticals \/ Santhera Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Idorsia Pharmaceuticals \/ Santhera Pharmaceuticals"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Ypsomed

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                            Lead Product(s) : Navenibart

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Astria Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            Astria Chooses YpsoMate Device For STAR-0215 Angioedema Treatment

                            Details : Ypsomed will be responsible for the development of an autoinjector for STAR-0215, a monoclonal antibody inhibitor of plasma kallikrein in development for the treatment of hereditary angioedema.

                            Product Name : STAR-0215

                            Product Type : Antibody

                            Upfront Cash : Undisclosed

                            December 08, 2024

                            Lead Product(s) : Navenibart

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Astria Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            02

                            F. Hoffmann-La Roche

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                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Chugai Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Evrysdi Two-Year Data in SMA: Most Children Can Sit, Stand, and Walk Independently

                            Details : Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier, which is being evaluated for the treatment of infants with pre-symptomatic spinal muscular atrophy.

                            Product Name : Evrysdi

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            October 14, 2024

                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Chugai Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Santhera Pharmaceuticals

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                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Santhera Receives Positive CHMP Opinion Recommending Approval of AGAMREE® (vamorolone) for the Treatment of D...

                            Details : Agamree (vamorolone) is a novel drug candidate with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity, being developed for the treatment Duchenne muscular dystrophy.

                            Product Name : Agamree

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            October 13, 2023

                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Santhera Pharmaceuticals

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                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Santhera and ReveraGen Announce FDA Acceptance of New Drug Application for Vamorolone in Duchenne Muscular Dys...

                            Details : VBP15 (vamorolone) is an investigational drug candidate, which binds to the same receptor as corticosteroids but modifying its downstream activity and as such is considered a dissociative anti-inflammatory drug. Vamorolone is being developed for DMD.

                            Product Name : Agamree

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            September 01, 2023

                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Santhera Pharmaceuticals

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                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Catalyst Pharmaceuticals

                            Deal Size : $231.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Santhera Announces Closing of Exclusive North America License Agreement with Catalyst Pharmaceuticals for Vamo...

                            Details : Under the agreement, Catalyst gains commercialization rights in North America for VBP15 (vamorolone), an investigational drug candidate with a mode of action based on binding to the same receptor as glucocorticoids, in Duchenne muscular dystrophy (DMD).

                            Product Name : Agamree

                            Product Type : Steroid

                            Upfront Cash : $75.0 million

                            July 19, 2023

                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Catalyst Pharmaceuticals

                            Deal Size : $231.0 million

                            Deal Type : Licensing Agreement

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                            06

                            Santhera Pharmaceuticals

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                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Catalyst Pharmaceuticals

                            Deal Size : $231.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Santhera Grants Exclusive North America License for Vamorolone to Catalyst Pharmaceuticals in Deal Valued at u...

                            Details : Santhera will grant Catalyst exclusive commercialization rights to VBP15, an orally active dissociative steroidal anti-inflammatory agent, in North America and Santhera will continue to focus on European commercialization of vamorolone in duchenne muscul...

                            Product Name : Agamree

                            Product Type : Steroid

                            Upfront Cash : $90.0 million

                            June 20, 2023

                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Catalyst Pharmaceuticals

                            Deal Size : $231.0 million

                            Deal Type : Licensing Agreement

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                            07

                            Santhera Pharmaceuticals

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                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Vamorolone NDA Mid-Cycle Review Meeting by FDA Completed

                            Details : VBP15 (vamorolone) is an investigational drug candidate, which binds to the same receptor as corticosteroids but modifying its downstream activity and as such is considered a dissociative anti-inflammatory drug. Vamorolone is being developed for DMD.

                            Product Name : Agamree

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            April 25, 2023

                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Spexis

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                            Spexis

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                            Lead Product(s) : Colistimethate Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : SPRIM Global Investments

                            Deal Size : Undisclosed

                            Deal Type : Financing

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                            Spexis Announces Closing of Capital Commitment of USD 4.5 Million From SPRIM Global Investments (sgi) to Enabl...

                            Details : The net proceeds from the capital commitment will enable the initiation of the Phase 3 COPILOT study, the first part of the registrational clinical studies planned for ColiFin, an inhaled therapeutic for the management of chronic infections in cystic fib...

                            Product Name : ColiFin

                            Product Type : Antibiotic

                            Upfront Cash : Undisclosed

                            April 18, 2023

                            Lead Product(s) : Colistimethate Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : SPRIM Global Investments

                            Deal Size : Undisclosed

                            Deal Type : Financing

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                            09

                            Santhera Pharmaceuticals

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                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Highbridge Capital Management

                            Deal Size : $23.5 million

                            Deal Type : Private Placement

                            DEALS_DEV arrow-down

                            Santhera Secures Additional Funding Through a Private Placement of Shares and Upsizing of Existing Financing A...

                            Details : The net funding will be used to support the Company through FDA decision on VBP15 (vamorolone), a dissociative steroid with novel mode of action, in Duchenne muscular dystrophy (DMD).

                            Product Name : Agamree

                            Product Type : Steroid

                            Upfront Cash : Undisclosed

                            February 28, 2023

                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Highbridge Capital Management

                            Deal Size : $23.5 million

                            Deal Type : Private Placement

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                            10

                            Santhera Pharmaceuticals

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                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Santhera Submits Marketing Authorization Application to the UK MHRA for Vamorolone in Duchenne Muscular Dystro...

                            Details : VBP15 (vamorolone) it is an antagonist of the mineralocorticoid receptor, inactivating it. Studies in DMD patients have shown that mineralocorticoid receptor antagonists aid in preserving heart function in DMD.

                            Product Name : Agamree

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            February 03, 2023

                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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