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RP A501

Drug Profile

RP A501

Alternative Names: AAV9.LAMP2B; RP-A501

Latest Information Update: 08 Nov 2023

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At a glance

  • Originator Rocket Pharmaceuticals
  • Class Gene therapies
  • Mechanism of Action Gene transference; Lysosomal-associated membrane protein 2 replacements
  • Orphan Drug Status

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

    Yes - Glycogen storage disease type II
  • New Molecular Entity No

Highest Development Phases

  • Phase II Glycogen storage disease type II

Most Recent Events

  • 06 Nov 2023 Rocket Pharmaceuticals reached an alignment with the US FDA on the global phase II pivotal trial of RP A501 for Glycogen storage disease
  • 06 Nov 2023 Rocket Pharmaceuticals submits Clinical Trial Application (CTA)/Investigational Medicinal Product Dossier (IMPD) to EMA and MHRA for Glycogen storage disease
  • 06 Nov 2023 Rocket Pharmaceuticals plans a phase II trial in Glycogen storage disease in Europe and United Kingdom
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