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    [{"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":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"Crovalimab","moa":"Complement C5","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"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":"survival motor neurin SMN1 and SMN2 splicing modulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","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":"2021","type":"Inapplicable","leadProduct":"Branaplam","moa":"survival motor neurin SMN1 and SMN2 splicing modulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","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":"survival motor neurin SMN1 and SMN2 splicing modulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"Satralizumab","moa":"IL6Ralpha\/GP130","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ 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":"Cad-1883","moa":"Calcium-activated potassium channel KCa2 (KCNN)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","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":"Alpelisib","moa":"PI3-kinase p110-alpha subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"CSL Vifor","sponsor":"Fortrea","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Vamifeport","moa":"Ferroportin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CSL Vifor","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"CSL Vifor \/ Fortrea","highestDevelopmentStatusID":"8","companyTruncated":"CSL Vifor \/ Fortrea"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"DFV890","moa":"NLRP3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Basmisanil","moa":"GABA(A) receptor alpha-5 (GABRA5)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ 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":"survival motor neurin SMN1 and SMN2 splicing modulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"Crovalimab","moa":"Complement C5","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"Ypsomed","sponsor":"Astria Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody","year":"2024","type":"Partnership","leadProduct":"Navenibart","moa":"KLKB1","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":"Alpelisib","moa":"PI3-kinase p110-alpha subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Alpelisib","moa":"PI3-kinase p110-alpha subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Novartis Pharmaceuticals Corporation

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                            Novartis Pharmaceuticals Corporation

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                            Lead Product(s) : Alpelisib

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Alpelisib (BYL719) Safety & Efficacy in Pediatric & Adult PROS Patients

                            Details : Alpelisib is a Other Small Molecule drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of PIK3CA-mutated Overgrowth Spectrum.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 30, 2025

                            Lead Product(s) : Alpelisib

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Ypsomed

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                            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 : Undisclosed

                            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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                            03

                            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

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                            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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                            04

                            F. Hoffmann-La Roche

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                            Lead Product(s) : Satralizumab

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            June 10, 2024

                            Lead Product(s) : Satralizumab

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            F. Hoffmann-La Roche

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                            F. Hoffmann-La Roche

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                            Lead Product(s) : Delandistrogene Moxeparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Sarepta Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Un...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            November 13, 2023

                            Lead Product(s) : Delandistrogene Moxeparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Sarepta Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Santhera Pharmaceuticals

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                            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 : Other Small Molecule

                            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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                            07

                            Santhera Pharmaceuticals

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                            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 : Other Small Molecule

                            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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                            08

                            Santhera Pharmaceuticals

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                            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 : Other Small Molecule

                            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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                            09

                            Santhera Pharmaceuticals

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                            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 : Other Small Molecule

                            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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                            10

                            Santhera Pharmaceuticals

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                            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 : Other Small Molecule

                            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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