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RP A501

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Drug Profile

RP A501

Alternative Names: AAV9.LAMP2B; RP-A501

Latest Information Update: 25 Nov 2024

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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

  • 18 Nov 2024 Efficacy and adverse events data from a phase-I clinical trials in Glycogen storage disease type II released by Rocket Pharmaceuticals
  • 17 Sep 2024 Rocket Pharmaceuticals plans to pursue regulatory filings in the US and outside US for Glycogen storage disease type II
  • 17 Sep 2024 Rocket Pharmaceuticals completes enrolment in a phase II trial in Glycogen storage disease type II (In children, In adolescents, In adults) in USA, UK, Italy and Germany (IV) (NCT06092034)

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