Research programme: neurological disorder therapeutics - AveXis

Drug Profile

Research programme: neurological disorder therapeutics - AveXis

Alternative Names: AVXS-201; AVXS-301

Latest Information Update: 17 May 2018

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At a glance

  • Originator AveXis
  • Class Gene therapies
  • Mechanism of Action Undefined mechanism
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Amyotrophic lateral sclerosis; Rett syndrome

Most Recent Events

  • 15 May 2018 AveXis has been acquired by Novartis
  • 27 Feb 2018 AveXis announces intention to submit IND application for AVXS 201 for Rett syndrome in late 2018 or early 2019
  • 27 Feb 2018 AveXis announces intention to submit IND application for AVXS 301 for Amyotrophic lateral sclerosis in late 2018 or early 2019
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