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Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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            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

            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 patients.

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            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

            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.

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            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

            Details:

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