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AAV miR C9orf 72

Drug Profile

AAV miR C9orf 72

Alternative Names: AAV-miR-C9orf-72

Latest Information Update: 22 Apr 2024

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At a glance

  • Originator AviadoBio
  • Class Antidementias; Gene therapies; RNA
  • Mechanism of Action C9orf72 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.

  • New Molecular Entity No

Highest Development Phases

  • Research Amyotrophic lateral sclerosis; Frontotemporal dementia

Most Recent Events

  • 18 Apr 2024 Early research in Frontotemporal dementia in United Kingdom (Parenteral) (AviadoBio pipeline, April 2024)
  • 02 Jan 2024 Early research in Amyotrophic lateral sclerosis in United Kingdom (Parenteral)
  • 23 Feb 2023 AviadoBio in-licenses technology from Neurgain Technologies for novel gene therapy spinal delivery technology
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