Research programme: NAV gene therapies - REGENXBIO

Drug Profile

Research programme: NAV gene therapies - REGENXBIO

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

Latest Information Update: 20 Jul 2018

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At a glance

  • Originator ReGenX Biosciences
  • Developer REGENXBIO
  • 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
  • New Molecular Entity No

Highest Development Phases

  • Discontinued Age-related macular degeneration; Alzheimer's disease; Amyotrophic lateral sclerosis; Gaucher's disease; Leber congenital amaurosis; Mucopolysaccharidosis VI; Parkinson's disease; Retinitis pigmentosa

Most Recent Events

  • 20 Jul 2018 Discontinued - Preclinical for Age-related macular degeneration in USA (Ophthalmic)
  • 20 Jul 2018 Discontinued - Preclinical for Alzheimer's disease in USA (Parenteral)
  • 20 Jul 2018 Discontinued - Preclinical for Amyotrophic lateral sclerosis in USA (Parenteral)
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