Lead Product(s): Tolcapone
Therapeutic Area: Genetic Disease Product Name: CRX-1008
Highest Development Status: Phase I Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 15, 2020
The results confirm that CRX-1008 is a potent TTR stabilizer in both plasma and CSF using established ex vivo assays. CRX-1008 normalized TTR concentration in plasma with an overall mean increase in plasma TTR tetramer concentration of 55%.