Drug Profile

Research programme: citrullinemia type 1 therapeutics - iECURE

Latest Information Update: 28 Jun 2026

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 University of Pennsylvania
Developer iECURE; University of Pennsylvania
Class Gene therapies
Mechanism of Action Argininosuccinate synthase replacements; Gene modulators

Orphan Drug Status

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

No
New Molecular Entity No

28 Jun 2026 No recent reports of development identified for research development in Citrullinemia in USA (Parenteral)
24 May 2022 Early research in Citrullinemia in USA (Parenteral) as of May 2022 (iECURE pipeline, May 2022)

If your organization or you do not have a subscription, try one of the following:

Contacting your organization’s admin about adding this content to your AdisInsight subscription
Buying a PDF version of any individual profile
Request a free trial (opens in a new window)

If your organization has a subscription, there are several access options, even while working remotely:

Working within your organization’s network
Login with username/password or try to access via your institution
Persisted access using your organization’s identifier stored in your user browser for 90 days

For assistance, contact us at asktheexpert.adisinsight@springer.com