Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

Avalotcagene ontaparvovec - Dimension Therapeutics

Next Previous
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: 03 Mar 2025

Price :
$50 *
Note:
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

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

  • 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)
  • 06 Jan 2023 Ultragenyx plans a phase III (eNH3ance study) for Inborn urea cycle disorders in the first quarter of 2023

You need to be a logged in or subscribed to view this content

Request demo (opens in a new window)
If your organization or you do not have a subscription, try one of the following: If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top