Drug Profile

CRD 003

Latest Information Update: 26 Sep 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 Cure Rare Disease
Class Gene therapies
Mechanism of Action FKRP protein replacements; Gene transference

Orphan Drug Status

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

Yes - Limb girdle muscular dystrophies
New Molecular Entity No

Highest Development Phases

Preclinical Limb girdle muscular dystrophies

Most Recent Events

24 Sep 2025 CRD 003 receives Orphan Drug status for Limb girdle muscular dystrophies in USA
23 Jun 2025 Preclinical trials in Limb girdle muscular dystrophies in USA (IV) prior to June 2025
23 Jun 2025 Cure Rare Disease completes of a pre-IND meeting with the US FDA regarding CRD 003

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

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