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    [{"orgOrder":0,"company":"Versantis","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"VS-01","moa":"Ammonia clearance","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Versantis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Versantis \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Versantis \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Disc medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Licensing Agreement","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"F. Hoffmann-La Roche","amount2":0.20000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20000000000000001,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Disc medicine","highestDevelopmentStatusID":"4","companyTruncated":"F. Hoffmann-La Roche \/ Disc medicine"},{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"APR-OD031","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"APR Applied Pharma Research","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"APR Applied Pharma Research \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"APR Applied Pharma Research \/ Undisclosed"},{"orgOrder":0,"company":"Somagenetix","sponsor":"Vi Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2024","type":"Series A Financing","leadProduct":"SGX-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Somagenetix","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Somagenetix \/ Vi Partners","highestDevelopmentStatusID":"4","companyTruncated":"Somagenetix \/ Vi Partners"},{"orgOrder":0,"company":"Addex Therapeutics","sponsor":"Charcot Marie Tooth Association","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Addex Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Addex Therapeutics \/ Charcot Marie Tooth Association","highestDevelopmentStatusID":"4","companyTruncated":"Addex Therapeutics \/ Charcot Marie Tooth Association"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Somagenetix

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                            Bio-Europe Autumn
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                            Somagenetix

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                            Lead Product(s) : SGX-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Vi Partners

                            Deal Size : $11.6 million

                            Deal Type : Series A Financing

                            DEALS_DEV arrow-down

                            Somagenetix Secures CHF 10M to Advance Gene Therapy For CGD

                            Details : The funds will enable Somagenetix to advance its leading gene therapy-candidate, SGX-001, into a clinical trial for the treatment of p47 Chronic Granulomatous Disease (CGD).

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            October 08, 2024

                            Lead Product(s) : SGX-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Vi Partners

                            Deal Size : $11.6 million

                            Deal Type : Series A Financing

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                            02

                            Addex Therapeutics

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                            Bio-Europe Autumn
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                            Addex Therapeutics

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                            Bio-Europe Autumn
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Charcot Marie Tooth Association

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            CMTA and Addex Therapeutics Enter Collaboration to Advance GABA B PAM as Potential Treatment for CMT1A

                            Details : The primary goal of the collaboration is to evaluate the benefit of Addex’s proprietary positive allosteric modulator’s (PAM’s) targeting the gamma-aminobutyric acid subtype B (GABAB) receptor in rodent models of CMT1A.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            September 22, 2021

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Charcot Marie Tooth Association

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            03

                            F. Hoffmann-La Roche

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                            Bio-Europe Autumn
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                            F. Hoffmann-La Roche

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                            Bio-Europe Autumn
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                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Disc medicine

                            Deal Size : $200.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Disc Medicine Expands Hematology Pipeline with Worldwide Licensing Agreement for Bitopertin, a First-in-Class ...

                            Details : Under the terms of the agreement, Disc Medicine will obtain exclusive, global rights to and be responsible for all development, manufacturing, and commercialization of bitopertin and related back up compounds.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Undisclosed

                            May 27, 2021

                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Disc medicine

                            Deal Size : $200.0 million

                            Deal Type : Licensing Agreement

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                            04

                            Novartis Pharmaceuticals Corporation

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                            Bio-Europe Autumn
                            Not Confirmed

                            Novartis Pharmaceuticals Corporation

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                            Bio-Europe Autumn
                            Not Confirmed
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                            Lead Product(s) : Branaplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Novartis receives US FDA Orphan Drug Designation for branaplam (LMI070) in Huntington’s disease (HD)

                            Details : In preclinical models, branaplam has been shown to reduce levels of mutant huntingtin protein. In addition, during the investigation of branaplam in spinal muscular atrophy (SMA), it was also observed to reduce huntingtin messenger RNA (mRNA) in SMA pati...

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            October 21, 2020

                            Lead Product(s) : Branaplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Versantis

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                            Bio-Europe Autumn
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                            Versantis

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                            Bio-Europe Autumn
                            Not Confirmed
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                            Lead Product(s) : VS-01

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Versantis Receives FDA Rare Pediatric Disease Designation for VS-01 for the Treatment of Urea Cycle Disorders

                            Details : VS-01 is an innovative liposomal-based detoxification therapy that acts as a clearance enhancer for a large spectrum of toxic metabolites accumulated during liver and kidney failures.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            October 19, 2020

                            Lead Product(s) : VS-01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            APR Applied Pharma Research

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                            Bio-Europe Autumn
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                            APR Applied Pharma Research

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                            Bio-Europe Autumn
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                            Lead Product(s) : APR-OD031

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Grants Orphan Drug Designation to APR-OD031 for the Treatment of Phenylketonuria (PKU)

                            Details : First mix of amino acids having a pharmacological primary mode of action designed to reduce and control Phenylalanine fluctuations.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 26, 2020

                            Lead Product(s) : APR-OD031

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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