Research programme: NAV gene therapies - REGENXBIO

Drug Profile

Research programme: NAV gene therapies - REGENXBIO

Alternative Names: NAV® therapeutics - REGENXBIO; RGX 111; RGX-121; RGX-314; RGX-321

Latest Information Update: 13 Jan 2017

Price : $50

At a glance

  • Originator ReGenX Biosciences
  • Developer REGENXBIO; University of Pennsylvania Medical Center
  • Class Gene therapies; Proteins
  • Mechanism of Action Gene transference; Vascular endothelial growth factors inhibitors
  • Orphan Drug Status

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

    Yes - Mucopolysaccharidosis I; Mucopolysaccharidosis II; Hyperlipoproteinaemia type IIa
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Preclinical Mucopolysaccharidosis I; Mucopolysaccharidosis II; Retinitis pigmentosa; Wet age-related macular degeneration
  • 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

  • 06 Jan 2017 REGENXBIO files an IND application with the US FDA for Wet age-related macular degeneration
  • 06 Jan 2017 REGENXBIO completes a IND-enabling studies in Wet age-related macular degeneration
  • 06 Jan 2017 REGENXBIO plans a phase I trial for Wet age-related macular degeneration in USA
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top