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Marnetegragene autotemcel - Rocket Pharmaceuticals

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Drug Profile

Marnetegragene autotemcel - Rocket Pharmaceuticals

Alternative Names: KRESLADI; LAD-1-gene-therapy-Rocket-Pharmaceuticals; Leukocyte-adhesion-deficiency-1-gene-therapy-Rocket-Pharmaceuticals; LV RP-L201; marne-cel; RP-L201

Latest Information Update: 12 Mar 2025

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At a glance

  • Originator CIEMAT
  • Developer Rocket Pharmaceuticals
  • Class Gene therapies; Haematopoietic stem cells therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    Yes - Leukocyte-Adhesion Deficiency Syndrome
  • New Molecular Entity No

Highest Development Phases

  • Preregistration Leukocyte-Adhesion Deficiency Syndrome

Most Recent Events

  • 27 Feb 2025 Rocket Pharmaceuticals announces intention to submit the complete BLA to the US FDA to resolve Complete Response Letter in 2025
  • 28 Jun 2024 The US FDA issues a Complete Response Letter (CRL) in response to Biologics License Application for Marnetegragene autotemcel
  • 06 May 2024 Rocket Pharmaceuticals anticipates to launch marnetegragene autotemcel for Leukocyte adhesion deficiency syndrome

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