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RGX 501

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Drug Profile

RGX 501

Alternative Names: Adeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor

Latest Information Update: 07 Sep 2021

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At a glance

  • Originator REGENXBIO
  • Developer REGENXBIO; University of Pennsylvania
  • Class Antihyperlipidaemics; Gene therapies; Proteins
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    Yes - Hyperlipoproteinaemia type IIa
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Discontinued Hyperlipoproteinaemia type IIa

Most Recent Events

  • 07 Aug 2020 RGX 501 is available for licensing as of 07 Aug 2020. https://www.regenxbio.com/therapeutic-programs/
  • 07 Aug 2020 Discontinued - Phase-I/II for Hyperlipoproteinaemia type IIa in USA (IV)
  • 09 Jan 2020 Safety data from a phase I/II trial in Hyperlipoproteinaemia-type-IIa released by REGENXBIO
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