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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: 30 Mar 2026

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At a glance

  • Originator CIEMAT
  • Developer Rocket Pharmaceuticals
  • Class Gene therapies; Haematopoietic stem cells therapies
  • Mechanism of Action Gene transference; ITGB2 protein replacements
  • Orphan Drug Status

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

    Yes - Leukocyte adhesion deficiency type 1
  • New Molecular Entity No

Highest Development Phases

  • Registered Leukocyte adhesion deficiency type 1

Most Recent Events

  • 26 Mar 2026 Registered for Leukocyte adhesion deficiency type-1 (In infants, In children) in USA (IV) - First global approval
  • 26 Mar 2026 The US FDA grants a Rare Pediatric Disease Priority Review Voucher (PRV) for rare pediatric diseases
  • 26 Mar 2026 Updated adverse event data from the phase I/II trial for Leukocyte adhesion deficiency type-1 released by Rocket Pharmaceuticals

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