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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 IIProduct 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 IIProduct 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: PreclinicalProduct 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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