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    [{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Branaplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Branaplam","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Branaplam","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"}]

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                            Novartis Pharmaceuticals Corporation

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                            Lead Product(s) : Branaplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

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                            Branaplam : VIBRANT-HD Study Update

                            Details : 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.

                            Brand Name : LMI070

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            August 25, 2022

                            Lead Product(s) : Branaplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

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                            Lead Product(s) : Branaplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

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                            Novartis Receives FDA Fast Track Designation for Branaplam (LMI070) For the Treatment of Huntington’s Diseas...

                            Details : 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.

                            Brand Name : LMI070

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            December 16, 2021

                            Lead Product(s) : Branaplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

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                            Lead Product(s) : Branaplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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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...

                            Brand Name : LMI070

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 21, 2020

                            Lead Product(s) : Branaplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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