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ER 2001

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Drug Profile

ER 2001

Alternative Names: ER-2001

Latest Information Update: 13 Sep 2023

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At a glance

  • Originator ExoRNA Bioscience
  • Class Small interfering RNA
  • Mechanism of Action Huntingtin protein expression inhibitors; RNA interference
  • 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

  • Phase 0 Huntington's disease

Most Recent Events

  • 13 Sep 2023 ExoRNA Bioscience has patents pending for siRNA for inhibiting HTT gene expression in the US
  • 04 Apr 2023 Phase-0 for Huntington's disease in China (IV) (NCT06024265)

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