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Mozafancogene autotemcel - Rocket Pharmaceuticals

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Drug Profile

Mozafancogene autotemcel - Rocket Pharmaceuticals

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

Latest Information Update: 12 Nov 2024

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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

  • 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
  • 02 Apr 2024 Rocket Pharmaceuticals plans to file biologics license application (BLA) for FA to the US FDA in the first half of 2024

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