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?

Research programme: Adeno-associated virus -based gene therapeutics - Alexion, AstraZeneca Rare Disease

Next Previous
Drug Profile

Research programme: Adeno-associated virus -based gene therapeutics - Alexion, AstraZeneca Rare Disease

Latest Information Update: 10 Dec 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 Alexion AstraZeneca Rare Disease
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • 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

  • Research Unspecified

Most Recent Events

  • 09 Jul 2025 JCR Pharmaceuticals entered into a license agreement with Alexion, AstraZeneca Rare Disease, to develop genomic medicines
  • 09 Jul 2025 Early research in Unspecified in USA (Parenteral), Prior to July 2025

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