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AMT 162

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Drug Profile

AMT 162

Alternative Names: AMT-162; APB-102

Latest Information Update: 06 Feb 2025

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At a glance

  • Originator University of Massachusetts Medical School
  • Developer uniQure
  • Class Gene therapies
  • Mechanism of Action Gene silencing; Superoxide dismutase 1 expression inhibitors
  • Orphan Drug Status

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

    Yes - Amyotrophic lateral sclerosis
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Amyotrophic lateral sclerosis

Most Recent Events

  • 30 Jan 2025 uniQure receives favorable recommendation from Independent Data Monitoring Committee for phase I/II EPISOD1 clinical trial for Amyotrophic lateral sclerosis
  • 01 Aug 2024 Phase-I/II clinical trials in Amyotrophic lateral sclerosis in USA (Intrathecal) (NCT06100276)
  • 28 Feb 2024 UniQure has an exclusive license to two patent families directed to gene therapy treatment of ALS, including AMT 162

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