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Avalotcagene ontaparvovec - Dimension Therapeutics

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Drug Profile

Avalotcagene ontaparvovec - Dimension Therapeutics

Alternative Names: DTX 301; Ornithine-transcarbamylase-gene-therapy-Dimension-Therapeutics; OTC-gene-therapy-Dimension-Therapeutics; scAAV8OTC

Latest Information Update: 26 Mar 2026

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At a glance

  • Originator Dimension Therapeutics
  • Developer Dimension Therapeutics; Ultragenyx Pharmaceutical
  • Class Gene therapies
  • Mechanism of Action Gene transference; Ornithine carbamoyltransferase replacements
  • Orphan Drug Status

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

    Yes - Inborn urea cycle disorders
  • New Molecular Entity No

Highest Development Phases

  • Phase III Inborn urea cycle disorders

Most Recent Events

  • 12 Mar 2026 Pharmacokinetics, efficacy and adverse events data form phase-III clinical trials in Inborn urea cycle disorders released by Ultragenyx
  • 31 Dec 2024 Ultragenyx Pharmaceutical has patent protection for avalotcagene ontaparvovec in the USA and other foreign countries, sub-licensed from REGENX, before December 2024
  • 15 Feb 2023 Phase-III clinical trials in Inborn urea cycle disorders (In adolescents, In adults, In the elderly) in Spain, Canada, USA (IV) (NCT05345171)

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