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Mozafancogene autotemcel - Rocket Pharmaceuticals

Drug Profile

Mozafancogene autotemcel - Rocket Pharmaceuticals

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

Latest Information Update: 08 Nov 2023

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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 A protein expression stimulants; 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

  • Phase II Fanconi's anaemia

Most Recent Events

  • 08 Nov 2023 Rocket Pharmaceuticals plans for regulatory filings in the US and Europe in 1H of 2024
  • 19 May 2023 Efficacy and adverse events data from phase-II trial in Fanconi's anaemia presented presented at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT-2023)
  • 27 Feb 2023 Rocket pharmaceuticals announces intention to submit BLA for Fanconi's anaemia in the fourth quarter of 2023
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