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.
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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
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Only accessible to MedStartr community members.
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- 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.
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