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Research programme: small interfering RNA therapeutics - Crucible Therapeutics

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Drug Profile

Research programme: small interfering RNA therapeutics - Crucible Therapeutics

Latest Information Update: 04 Nov 2025

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At a glance

  • Originator Crucible Therapeutics; University of Sheffield
  • Developer Crucible Therapeutics
  • Class Small interfering RNA
  • Mechanism of Action RNA interference; SRSF1 protein expression inhibitors
  • 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; Motor neuron disease

Most Recent Events

No Data Available

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