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Details:
The goal of the collaboration is to generate new insights focused on genetic modifiers and the discovery of novel therapeutic targets for rare diseases and other unmet medical needs.
Lead Product(s): Undisclosed
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Undisclosed
Partner/Sponsor/Collaborator: BridgeBio Pharma
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration January 30, 2024
Details:
PRAX-222 is an ASO designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early-seizure-onset SCN2A-DEE to treat seizures and other symptoms in patients with gain-of-function SCN2A mutations.
Lead Product(s): PRAX-222
Therapeutic Area: Genetic Disease Product Name: PRAX-222
Highest Development Status: IND EnablingProduct Type: Small molecule
Recipient: Praxis Bioresearch
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 20, 2022
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