Praxis Precision Medicines
Precision CNS from genetic insight
Cambridge, MA, US
A clinical-stage biopharmaceutical company that translates genetic insights into the development of precision medicines for central nervous system (CNS) disorders.
How We Help
Problem Statement
To develop effective therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance.
Core Technology
Small molecule and antisense oligonucleotide (ASO) platforms
Key Differentiators
Translates genetic insights from rare epilepsies into precision medicines for broader neurological and psychiatric conditions.; Focuses on drug targets identified through human genetics.; Utilizes two proprietary platforms: Cerebrum™ (small molecule) and Solidus™ (antisense oligonucleotide).
Clinical Focus
Neurology; Psychiatry
Company Info
- Business Model
- Licensing
- Total Funding
- 100,000,000
- Website
- praxismedicines.com
Categories
Disease Areas
Technologies
Clinical & IP
Clinical Validation
Ulixacaltamide: FDA Breakthrough Therapy Designation (Dec 2025), positive Phase 3 Essential3 studies. Relutrigine: FDA Breakthrough Therapy Designation (July 2025), early stopping in EMBOLD trial due to overwhelming efficacy in SCN2A/SCN8A-DEEs. Vormatrigine: 56.3% median seizure reduction in RADIANT Phase 2.
MedStartr Index Score
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