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CMPCPS 001

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Drug Profile

CMPCPS 001

Alternative Names: CMP-CPS-001

Latest Information Update: 10 Jun 2025

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At a glance

  • Originator CAMP4 Therapeutics
  • Class Antisense oligonucleotides
  • Mechanism of Action Carbamoyl phosphate synthase (ammonia) expression stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Inborn urea cycle disorders
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I Inborn urea cycle disorders

Most Recent Events

  • 16 May 2025 Adverse events and pharmacokinetic data from a preclinical data in Inborn urea cycle disorders released by CAMP4 Therapeutics
  • 13 May 2025 CAMP4 Therapeutics submits Clinical Trial Application for a phase Ib trial in Inborn urea cycle disorders in Europe
  • 27 Mar 2025 CAMP4 Therapeutics expects CTA clearance in Europe in 2025

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