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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Check out Praxis Precision Medicines on @MedStartr Today A clinical-stage biopharmaceutical company that translates genetic insights into the development of precision medicines for central nervous system (CNS) disorders. #NeuronalExcitationInhibitionImbalance #ParkinsonSDisease #DevelopmentalAndEpilepticEncephalopathiesDEEs #Cephalgia #MonogeneticAutism #CNSDisorders #GeneticEpilepsies #PCDH19RelatedEpilepsy #SCN2ARelatedEpilepsy #AngelmanSyndrome #EssentialTremor #MentalBehavioralHealth #EpilepsySeizureDisorders #CentralNervousSystemDisorders #DrugDiscoveryDevelopment #MedMo #MedStartrVentures

https://www.medstartr.ai/s/h6uQAdnGSWg

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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: Only accessible to MedStartr community members. Join now
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Website: praxismedicines.com

Disease Areas

Neuronal Excitation-Inhibition Imbalance Parkinson's Disease Developmental and Epileptic Encephalopathies (DEEs)Cephalgia Monogenetic Autism CNS Disorders Genetic Epilepsies PCDH19-related Epilepsy SCN2A-related Epilepsy Angelman Syndrome Essential Tremor Mental Health Epilepsy & Seizure Disorders Central Nervous System Disorders

Technologies

Drug DevelopmentGeneticsSmall MoleculeAntisense Oligonucleotide (ASO)Genetic NeuroscienceCNS DisordersDrug DiscoveryTherapeuticsPrecision Medicine Tech

Clinical & IP

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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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MedStartr Engagement Score

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