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?

Mozafancogene autotemcel - Rocket Pharmaceuticals

Next Previous
Drug Profile

Mozafancogene autotemcel - Rocket Pharmaceuticals

Alternative Names: LV-RP-L102; RP L102; RP-L02

Latest Information Update: 19 May 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 CIEMAT
  • Developer CIEMAT; Rocket Pharmaceuticals
  • Class Antianaemics; Gene therapies; Stem cell therapies
  • Mechanism of Action Fanconi anaemia complementation group 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 - Fanconi's anaemia
  • New Molecular Entity No

Highest Development Phases

  • Preregistration Fanconi's anaemia

Most Recent Events

  • 08 May 2025 Rocket Pharmaceuticals expects to submit the final module of its filing in late 2025 or early 2026
  • 07 Nov 2024 Preregistration for Fanconi's anaemia (In adolescents, In children, In the elderly, In infants, In adults) in USA (IV)
  • 30 Apr 2024 European Medicines Agency accepts the Marketing Authorization Application (MAA) for Mozafancogene autotemcel for Fanconi's anaemia

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