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CRD 002

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Drug Profile

CRD 002

Alternative Names: Antisense oligonucleotide therapy - Cure Rare Disease; CRD-002

Latest Information Update: 27 May 2025

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At a glance

  • Originator Cure Rare Disease
  • Class Antisense oligonucleotides; Neuroprotectants; Oligonucleotides
  • Mechanism of Action Gene silencing
  • Orphan Drug Status

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

    Yes - Spinocerebellar ataxias
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Machado-Joseph Disease

Most Recent Events

  • 14 May 2025 CRD 002 receives Orphan Drug status for Spinocerebellar Ataxia Disease in USA
  • 14 May 2025 Cure Rare Disease announces intention to submit IND to US FDA for Machado-Joseph disease in 2026
  • 06 Feb 2025 Preclinical trials in Machado-Joseph Disease in USA (Parenteral) before February 2025

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