Parand Darou Pharma  Parand Darou Pharma

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[{"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":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis Receives FDA Fast Track Designation for Branaplam (LMI070) For the Treatment of Huntington\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Branaplam : VIBRANT-HD Study Update","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"}]

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            Branaplam (LMI070), is being developed as a potential first in class orally administered disease modifying therapy for HD. Branaplam has the potential to address this unmet medical need.

            Lead Product(s): Branaplam

            Therapeutic Area: Genetic Disease Product Name: LMI070

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 25, 2022

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            Branaplam (LMI070), is an mRNA splicing modulator that targets the underlying pathophysiology in HD by modifying HTT mRNA throughout the brain and the body, resulting in lower levels of HTT protein.

            Lead Product(s): Branaplam

            Therapeutic Area: Genetic Disease Product Name: LMI070

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 16, 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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