RGX 501

Drug Profile

RGX 501

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

Latest Information Update: 11 Jan 2018

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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 Yes

Highest Development Phases

  • Phase I/II Hyperlipoproteinaemia type IIa

Most Recent Events

  • 04 Jan 2018 Adverse events data from a phase I/II trial in Hyperlipoproteinaemia type IIa released by REGENXBIO
  • 01 Mar 2017 REGENXBIO and University of Pennsylvania initiate enrolment in a phase I/II trial for Hyperlipoprotenaemia type IIa in USA (REGENXBIO, 10-K, March 2017)
  • 16 Feb 2017 ARIAD Pharmaceuticals has been acquired by Takeda
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