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APRTX 001

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Drug Profile

APRTX 001

Alternative Names: APRTX-001

Latest Information Update: 26 Dec 2025

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At a glance

  • Originator Aperture Therapeutics
  • Class Antidementias; Antisense oligonucleotides
  • Mechanism of Action CD33 protein expression modulators; Gene silencing
  • Orphan Drug Status

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

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Amyotrophic lateral sclerosis; Frontotemporal dementia

Most Recent Events

  • 02 Dec 2025 Aperture Therapeutics has patent pending for "Compositions for modulating cd33 expression" in the multiple countries worldwide
  • 19 Nov 2025 Preclinical trials in Amyotrophic lateral sclerosis in USA (Parenteral)
  • 19 Nov 2025 Preclinical trials in Frontotemporal dementia in USA (Parenteral)

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