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    [{"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":"GABA-B receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Addex Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Addex Therapeutics \/ Charcot Marie Tooth Association","highestDevelopmentStatusID":"4","companyTruncated":"Addex Therapeutics \/ Charcot Marie Tooth Association"},{"orgOrder":0,"company":"Somagenetix","sponsor":"Vi Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2024","type":"Series A Financing","leadProduct":"SGX-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Somagenetix","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Somagenetix \/ Vi Partners","highestDevelopmentStatusID":"4","companyTruncated":"Somagenetix \/ Vi Partners"},{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","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 \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"APR Applied Pharma Research \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Disc medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Bitopertin","moa":"GlyT1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"F. Hoffmann-La Roche \/ Disc medicine","highestDevelopmentStatusID":"4","companyTruncated":"F. Hoffmann-La Roche \/ Disc medicine"},{"orgOrder":0,"company":"Versantis","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","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":"Intraperitoneal Injection","sponsorNew":"Versantis \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Versantis \/ Inapplicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            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

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                            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 : SGX-001

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            August 10, 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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                            Addex Therapeutics

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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

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                            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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                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Disc medicine

                            Deal Size : $200.0 million

                            Deal Type : Licensing Agreement

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                            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 : RO4917838

                            Product Type : Other Small Molecule

                            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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                            Lead Product(s) : Branaplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            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 : LMI070

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            October 21, 2020

                            Lead Product(s) : Branaplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Versantis

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                            Versantis

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                            Lead Product(s) : VS-01

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            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 : VS-01

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            October 19, 2020

                            Lead Product(s) : VS-01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            APR Applied Pharma Research

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                            Lead Product(s) : APR-OD031

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            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 : APR-OD031

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 26, 2020

                            Lead Product(s) : APR-OD031

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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