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?

EXG 34217

Next Previous
Drug Profile

EXG 34217

Alternative Names: EXG34217

Latest Information Update: 08 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 Elixirgen
  • Class Cell therapies; 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.

    Yes - Bone marrow disorders
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Bone marrow disorders
  • Preclinical Dyskeratosis congenita; Fanconi's anaemia
  • Research Myelodysplastic syndromes
  • No development reported Aplastic anaemia

Most Recent Events

  • 25 Feb 2025 Pharmacodynamics and adverse events data from a phase I/II trial in Bone marrow failure patients with telomere biology disorders released by Elixirgen Therapeutics
  • 20 Feb 2025 EXG 34217 receives Orphan Drug status for Bone marrow disorders in USA
  • 13 Feb 2025 EXG 34217 receives Regenerative Medicine Advanced Therapy (RMAT) status for Bone marrow disorders in USA

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