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Research programme: CNS disorders RNA interference-based therapeutics - Alnylam Pharmaceuticals

Drug Profile

Research programme: CNS disorders RNA interference-based therapeutics - Alnylam Pharmaceuticals

Alternative Names: ALN-APP

Latest Information Update: 13 Dec 2018

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At a glance

  • Originator Sirna Therapeutics
  • Developer Alnylam Pharmaceuticals; Sirna Therapeutics
  • Class RNA; Small interfering RNA
  • Mechanism of Action RNA interference
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Cerebral amyloid angiopathy; Neurodegenerative disorders
  • Discontinued Spinocerebellar degeneration

Most Recent Events

  • 06 Dec 2018 Preclinical trials in Cerebral amyloid angiopathy in USA (Parenteral)
  • 09 May 2018 Preclinical trials in Neurodegenerative disorders in USA (Intrathecal)
  • 08 May 2018 Alnylam Pharmaceuticals plans to file an IND application for Central nervous system-targeted candidate in between late-2019 to early 2020
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