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RP L301

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Drug Profile

RP L301

Alternative Names: LV RP-L301; RP-L301

Latest Information Update: 20 Oct 2025

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At a glance

  • Originator Centro de Investigacion Biomedica en Red de Enfermedades Raras; CIEMAT; Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
  • Developer Rocket Pharmaceuticals
  • Class Antianaemics; Gene therapies; Pyruvates
  • Mechanism of Action Gene transference; Pyruvate kinase replacements
  • Orphan Drug Status

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

    Yes - Pyruvate kinase deficiency of red cells
  • New Molecular Entity No

Highest Development Phases

  • Phase II Pyruvate kinase deficiency of red cells

Most Recent Events

  • 20 Oct 2025 Rocket Pharmaceuticals completes Phase-I clinical trials in Pyruvate kinase deficiency of red cells (In children, In adults, In adolescents) in USA and Spain (IV) (EudraCT2019-001656-19) (NCT04105166)
  • 28 Oct 2024 No recent reports of development identified for phase-I development in Pyruvate kinase deficiency of red cells(In adolescents, In children, In adults) in Spain (IV, Infusion)
  • 28 Oct 2024 No recent reports of development identified for phase-I development in Pyruvate kinase deficiency of red cells(In adolescents, In children, In adults) in USA (IV, Infusion)

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