Switzerland

Novartis Pharmaceuticals Corporation

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Novartis receives US FDA Orphan Drug Designation for branaplam (LMI070) in Huntington’s disease (HD)

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.

Versantis

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Versantis Receives FDA Rare Pediatric Disease Designation for VS-01 for the Treatment of Urea Cycle Disorders

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.