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RP L401

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Drug Profile

RP L401

Alternative Names: EFS.HTCIRG1-LV; RP-L401

Latest Information Update: 13 Apr 2022

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At a glance

  • Originator Lund University
  • Developer Lund University; Rocket Pharmaceuticals
  • Class Gene therapies; Haematopoietic stem cells therapies; Stem cell therapies
  • Mechanism of Action Cell replacements; Gene transference
  • Orphan Drug Status

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

    Yes - Osteopetrosis
  • New Molecular Entity No

Highest Development Phases

  • Discontinued Osteopetrosis

Most Recent Events

  • 31 Dec 2021 Discontinued - Phase-I for Osteopetrosis (In adolescents, In children, In infants, In adults) in USA (Parenteral) (Form 10K, December 2021, Rocket Pharmaceuticals)
  • 31 Dec 2021 Discontinued - Phase-I for Osteopetrosis in USA (Parenteral) (Form 10K, December 2021, Rocket Pharmaceuticals)
  • 31 Dec 2021 Discontinued - Preclinical for Osteopetrosis in Sweden (Parenteral) (Form 10K, December 2021, Rocket Pharmaceuticals)

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