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: 15 Nov 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

  • 08 Nov 2017 REGENXBIO announces intention to submit IND application to US FDA for RGX 121 for Mucopolysaccharidosis II in Q4 2017
  • 08 Nov 2017 US FDA approves IND application for RGX 111 in Mucopolysaccharidosis I
  • 08 Nov 2017 REGENXBIO plans a clinical trial for Mucopolysaccharidosis I in first half of 2018
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