Research programme: NAV gene therapies - REGENXBIO

Drug Profile

Research programme: NAV gene therapies - REGENXBIO

Alternative Names: NAV® therapeutics - REGENXBIO; RGX 111; RGX-121; RGX-321

Latest Information Update: 17 Apr 2017

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At a glance

  • Originator ReGenX Biosciences
  • Developer REGENXBIO; University of Pennsylvania Medical Center
  • Class 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; Mucopolysaccharidosis I; Mucopolysaccharidosis II
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Preclinical Mucopolysaccharidosis I; Mucopolysaccharidosis II; Retinitis pigmentosa
  • No development reported Age-related macular degeneration; Alzheimer's disease; Amyotrophic lateral sclerosis; Gaucher's disease; Leber congenital amaurosis; Mucopolysaccharidosis VI; Parkinson's disease

Most Recent Events

  • 15 Mar 2017 REGENXBIO has patents pending for RGX 111 and RGX 121 in USA (REGENXBIO, 10-K, March 2017)
  • 16 Feb 2017 ARIAD Pharmaceuticals has been acquired by Takeda
  • 13 Sep 2016 Pharmacodynamics data from a preclinical study of RGX 111 in Mucopolysaccharidosis-I released by REGENXBIO
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