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[{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Designation to APR-OD031 for the Treatment of Phenylketonuria (PKU)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"SWITZERLAND","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Undisclosed","graph2":"APR Applied Pharma Research"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis receives US FDA Orphan Drug Designation for branaplam (LMI070) in Huntington\u2019s disease (HD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Versantis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Versantis Receives FDA Rare Pediatric Disease Designation for VS-01 for the Treatment of Urea Cycle Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Versantis"},{"orgOrder":0,"company":"Actelion Pharmaceuticals Ltd","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Beyond Batten Disease Foundation Announces Agreement with Actelion Pharmaceuticals Ltd to Provide Drug Product for BBDF-101","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"March 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Actelion Pharmaceuticals Ltd"},{"orgOrder":0,"company":"Versantis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Versantis Receives U.S. FDA Orphan Drug Designation for VS-01","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Versantis"},{"orgOrder":0,"company":"Addex Therapeutics","sponsor":"The Charcot\u2013Marie\u2013Tooth Association","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"CMTA and Addex Therapeutics Enter Collaboration to Advance GABA B PAM as Potential Treatment for CMT1A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Undisclosed","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Addex Therapeutics"}]

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            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.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: The Charcot–Marie–Tooth Association

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration September 22, 2021

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            Versantis’ lead program, VS-01, is in clinical development as a first-line therapy for the timely reversal of ACLF. VS-01 has received ODD from the EMA and U.S. FDA.

            Lead Product(s): VS-01

            Therapeutic Area: Genetic Disease Product Name: VS-01

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 07, 2021

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            Janssen will provide drug supply for the clinical trials and Extended Access Program, and right of reference to data within the Zavesca New Drug Application (NDA) to support the development of BBDF-101.

            Lead Product(s): Miglustat,Trehalose API

            Therapeutic Area: Genetic Disease Product Name: BBDF 101

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Beyond Batten Disease Foundation

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration March 02, 2021

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            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 patients.

            Lead Product(s): Branaplam

            Therapeutic Area: Genetic Disease Product Name: LMI070

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 21, 2020

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            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.

            Lead Product(s): VS-01

            Therapeutic Area: Genetic Disease Product Name: VS-01

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 19, 2020

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            Details:

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

            Lead Product(s): APR-OD031

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 26, 2020

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