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Tominersen - Ionis Pharmaceuticals

Drug Profile

Tominersen - Ionis Pharmaceuticals

Alternative Names: HTT ASO; IONIS-HTTRx; ISIS-443139; ISIS-HTT Rx; RG 6042; RO-7234292

Latest Information Update: 05 Nov 2023

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At a glance

  • Originator Isis Pharmaceuticals
  • Developer Ionis Pharmaceuticals; Roche
  • Class Antisense oligonucleotides; Neuroprotectants
  • Mechanism of Action HD protein inhibitors; RNA interference
  • Orphan Drug Status

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

    Yes - Huntington's disease
  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Huntington's disease

Most Recent Events

  • 21 Jun 2023 Roche plans to file regulatory application for Huntington’s disease, in or after 2026 (Roche pipeline, June 2023)
  • 19 Jan 2023 Roche initiates phase II GENERATION HD2 for Huntington's-disease in USA (NCT05686551)
  • 09 Nov 2022 Roche plans to begin enrolment in phase II GENERATION HD2 trial for Huntington's disease in early 2023
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