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CMP 002

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Drug Profile

CMP 002

Alternative Names: CMP 002; CMP-SYNGAP-01

Latest Information Update: 21 Sep 2026

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At a glance

  • Originator CAMP4 Therapeutics
  • Class Antisense oligonucleotides
  • Mechanism of Action Gene silencing; SYNGAP1 protein expression stimulants
  • Orphan Drug Status

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

    Yes - CNS disorders
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical CNS disorders

Most Recent Events

  • 10 Sep 2026 Medicines and Healthcare Products Regulatory Agency approves IND application for phase I/II study in SYNGAP1-related disorder
  • 10 Sep 2026 CAMP4 Therapeutics plans to initiate a first-in-human phase I/II clinical trial of CMP 002 in Australia, Argentina and United Kingdom in Q4 of 2026
  • 14 Aug 2026 CAMP4 Therapeutics receives regulatory clearance from Argentina’s ANMAT to initiate the first-in-human phase I/II clinical trial of CMP 002 in patients with SYNGAP1-related disorder

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