X
[{"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"}]
Find Clinical Drug Pipeline Developments & Deals for Branaplam
Filters
Companies By Therapeutic Area
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.
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
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.
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
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.
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